Pulmonary Hypertension (PH) population characterisation and epidemiological analysis
IQVIA Ltd · Commercial
Expired The latest version ended on 18 May 2024. The September 2026 register still lists the agreement, but its term has passed.
- Reference
- DARS-NIC-296034-T4Y4K
- Latest version
- v3.6
- Term of latest version
- 21 June 2023 to 18 May 2024
- Start date
- 19 May 2020
- Data controller
- Joint Data Controller
- Commercial purposes
- Yes
- Sublicensing
- No
- Files released to date
- 39
Data controllers
Why the data was released
Objective for processing
This Data Sharing Agreement permits IQVIA Ltd to continue to hold the data for one year and process it for the verification of published findings if required.
As part of an internal reorganisation of its group of companies, IQVIA Technology Services Limited transferred its business functions to IQVIA Ltd. on 1 January 2023. Any reference to IQVIA Technology Services Ltd within the purpose section under this application has been removed unless it is historical information that was accurate at the time.
IQVIA Technology Services Ltd will have no further involvement under this Data Sharing Agreement and are no longer a Controller.
Pulmonary Arterial Hypertension (PAH) (a type of Pulmonary Hypertension (PH)), is a progressive and rare disease caused by narrowing or tightening (constriction) of the pulmonary arteries. PAH primarily affects the small pulmonary arterioles through vascular proliferation and re-modelling, while resulting in a progressive rise in lung blood pressure and heart failure. PAH represents Group 1 (of 5) within the Pulmonary Hypertension World Health Organisation clinical classification system (Dana Point, 2008). In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. Furthermore, there are several types of PAH including Idiopathic PAH (iPAH) and Associated PAH related to a range of disease processes, including cirrhosis, connective tissue disease, congenital heart disease, HIV infection and sickle-cell disease
In addition, there are other types of PH, including Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Sarcoidosis-Associated Pulmonary Hypertension (SAPH). CTEPH is caused by complications of acute Pulmonary embolism (PE), leading to an obstruction of the large pulmonary arteries and distal small-vessel arteriopathy, with an estimated incidence of 3-5 cases per 100,000 patients. Sarcoidosis is a multisystem immune-mediated inflammatory disorder categorised by the presence of granulomas in tissues and is highly associated with respiratory tissue. Sarcoid patients may develop SAPH, which can lead to significant complications.
Due to the complexity, late diagnosis and absence of real world studies across all subtypes of PH (PAH, CTEPH and SAPH), there is currently a lack of understanding regarding the clinical pathway leading to diagnosis. In addition, there is also the need to further explore and study post-diagnosis pathways and patient related outcomes associated with these diseases in routine clinical practice.
As a result, IQVIA Ltd and IQVIA Technology Services Ltd carried out a retrospective real world data analysis of UK PAH, CTEPH and SAPH patients for Janssen-Cilage Limited. The study focused on describing and comparing PAH, CTEPH and SAPH populations (and define sub-populations) in terms of characteristics of patients, clinical pathways pre-diagnosis and diagnostic procedures, treatment patterns post diagnosis, clinical outcomes and Healthcare Resource Utilisation (HCRU). This presented an opportunity for both Janssen and the broader scientific/research community to improve the body of knowledge around these three sub-types and further enhance the possibility of developing better clinical pathways, improving treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL).
The Pulmonary Hypertension Association UK (PHA UK) is the UKs largest and foremost patient advocacy group for those suffering from this life threatening and shortening condition; PHA UK has some 4,340 members representing the views of a large part of the UK patient population.
The Pulmonary Hypertension Association UK, agreed that the study “Pulmonary Hypertension (PH) population characterisation and epidemiological analysis” would benefit the broader scientific community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL). This research study will form a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
There was an additional purpose of the study for the patients involved, namely that the study could potentially support the development of future novel drugs in clinical trials. It was determined that the combined analysis of HES data and the enhanced diagnostic clinical data jointly held by STHFT and the University of Sheffield (UoS) could be very beneficial to this study, hence the joint involvement of each in the development of this study.
The goal of the research is to:
• Generate real world evidence on the clinical and treatment management of patients diagnosed with PAH, CTEPH and SAPH
• Analyse the characteristics of diagnosed patients at the time of confirmed diagnosis, and the clinical pathways leading up to diagnosis, diagnostic procedures and the post-diagnostic treatment patterns
• Investigate various clinical outcomes associated with PH and its subtypes, and comparative outcomes analysis of PH treatments
• To explore the healthcare resource use (HCRU) and the costs associated with the diagnosis of PAH, CTEPH and SAPH
To achieve the above objectives, IQVIA Ltd built a joint dataset that has the required patient cohorts and clinical information. The database comprises of non-identified patient data derived from the STHFT deep clinical databases which collect data on all patients attending the Sheffield Pulmonary Vascular Disease Unit (SPVDU) and national level hospital interactions from HES data.
Parties involved in the research:
Each party in the collaboration had a different role during the research:
1. STHFT had responsibility for ethics approval for the study, provide expert clinical insight on the research findings, support on datasets de-identification and linkage of data in addition to supporting the publication of research findings
2. IQVIA Ltd and IQVIA Technology Services Ltd supported STHFT ethical approvals activities, co-developed the study protocol and SAP, conducted the transformation and data processing of non-identified data into analysable format and performed the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen) funded the research and supported the development of the study protocol and SAP and provide expert clinical insight on the findings.
4. IQVIA Ltd and IQVIA Technology Services Ltd had access to non-identifiable record level data and created aggregate outputs which were shared with both Janssen and STHFT. Janssen were not permitted to access record level HES data and will only ever have access to aggregated data with small number suppressed in line with the HES Analysis Guide.
5. STHFT and Janssen will provide clinical interpretation of the results.
Janssen funded the research to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. STHFT as one of England’s leading PH diagnostic and treatment centres benefits from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPDVU), in addition to the verification that their unique diagnostic process is beneficial to patients, allowing them to share their learning with other centres.
It was important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This allowed IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data was critical in providing greater insight on all the patients who had attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH were included. Furthermore the STHFT data also provided information on in-depth patient clinical characteristics that allowed IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care was requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data was crucial to understand the complete patient pathway. All 20 of the diagnosis codes were requested in order to understand the comorbidities associated with PAH. The OPCS codes were also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity were required in order to characterise the patients.
The study design was a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd analysed the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which allowed IQVIA Ltd and IQVIA Technology Services Ltd to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. The cohort was approximately 3000 patients. NHS numbers and dates of birth were provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis required a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd requested ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
If required IQVIA Ltd will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Research being undertaken is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested has helped achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
If required Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This is comprised of two representatives of each of STHFT the UoS and Janssen and with IQVIA Ltd having one representative who will chair the group.
The committee will perform the following functions:
1) Provide clinical interpretation of the results to support refinements of the analysis within the bounds of the protocol
2) Agree the dissemination / publication routes for research findings (e.g. conference posters vs peer review papers etc.) based on the nature and strength of findings. (Please see output section for further information)
No organisation on the clinical interpretation group or any of the data controllers will have the ability to suppress any of the findings or outputs of the analysis. The clinical interpretation group members do not have any access to record level data.
Processing activities
No additional data will be disseminated during this Agreement.
IQVIA Ltd have finalised all of the analysis, however, require an extension to hold data in order to finalise the study report and publish the results. This Agreement permits IQVIA Ltd and STHFT to only further process the data for the purpose of verifying published findings if required.
To ensure the minimum amount of patient identifiable data was used and handled by the fewest people outside of the direct care team the following process was implemented
1. STHFT shared with NHS Digital team, via a secure file transfer protocol, the NHS numbers and dates of birth of patients that had attended the SDPVU clinic since 2012, aligned to a generated study ID. The total number of this cohort was about 3000 patients
2. NHS Digital linked the identifiable patient cohort to the following data sets; Admitted Patient Care, Outpatient and Accident & Emergency data. NHS Digital will remove the NHS numbers and dates of birth and returning the non-identified extract (including study ID) to the STHFT informatics team.
3. Patient data received from STHFT was linked to the HES data via the generated study ID and will be done in compliance with all trust policies on patient data handling. This data will only be accessible by the patient management team. NHS Digital supplied STHFT with the HES Data with a non-identifiable ID linked to the Study ID, which allowed STHFT to link the HES data to the diagnostic clinical data held at STHFT. The linked non-identified data was then loaded to a second logical environment also located within STHFT.
The research conducted on this combined dataset was for the agreed research questions and was performed on non-identified patient information. The HES linked data will not leave STHFT. Only aggregated non-patient level outputs will be shared.
The analysis was conducted in order to generate real world evidence on the current clinical and treatment management of adult patients with a confirmed diagnosis of PAH, CTEPH or SAPH within the Sheffield EMR who have been treated with at least one PH-specific medical treatment.
1.Detailed analysis of the clinical characteristics (including diagnostic tests where available, functional class, presence of co-morbidities, previous pulmonary embolism for CTEPH patients) of diagnosed patients at the time of confirmed diagnosis between 2010 and 2019
2. Describe the pathways leading to diagnosis, including time to diagnosis from initial symptoms (PAH and CTEPH patients) or from sarcoidosis diagnosis (SAPH patients)
3.Describe the prescribed treatments after diagnosis and the treatment pathways, from the date of start of the first prescribed treatment, including use of co-medications (if available), persistence, discontinuation, switching and add-on therapy
4. Investigate various clinical outcomes associated with PAH, CTEPH and SAPH across different patient groups (e.g. categorised by comorbidity) as well as conducting comparative outcomes analysis of PH treatments. In addition, there is the aim to explore the healthcare resource use (HCRU) and the costs associated with the diagnosis.
Researchers who had access to the patient level HES data were logged on an access control register, ensuring that it was possible to identify everyone with access to patient level information. Each researcher from IQVIA Ltd. and /or IQVIA Technology Services Ltd that had access to record level data signed an Intra-Company Agreement that contained information on best practice and rules. These rules were adhered to as stated in specific section of the contract, including the prevention of exporting any data from the Sheffield (STHFT) server that contravenes the HES small numbers protocol.
All individuals with access to the record level data were substantive employees of IQVIA Ltd, and or/STH save researchers from other parts of the IQVIA group, who may have been required from time to time to provide expertise in analysis of the data. These individuals worked under an Intra-Company Agreement to IQVIA Ltd. All individuals accessing the data under an Intra-Company Agreement issued by IQVIA Ltd were a substantive employee of the IQVIA company group.
During the analysis process of the pseudonymised and aggregated data, there were regular sessions with Sheffield (STHFT) and Janssen clinical experts to provide clinical perspective and impact of the results generated.
Janssen-Cilag Limited did not carry out any processing of record level data under the previous version of this Agreement.
If required under this Data Sharing Agreement this environment will be remotely accessed within the STHFT “De-militarized Zone” (DMZ), which is a special local network configuration designed to improve security by segregating computers on each side of a firewall. This is an additional layer of security between the trusted internal network and an untrusted external network i.e. the internet. The research will be undertaken by trained researchers from IQVIA Ltd and specific members from the University of Sheffield who are under honorary contracts or hold Research Passports at the STHFT. Access is granted using strong two factor authentication based on USB keys which produce one time use passwords (more information can be found at https://www.yubico.com/).
There will be not attempt to re-identify any individuals
No data linkage will be undertaken with NHS England data provided under this Agreement except for the linkage already noted in the Agreement.
Expected output
IQVIA Ltd and IQVIA Technology Services Ltd conducted initial analysis of the clinical pathway leading up to diagnosis and the post-diagnostic treatment patterns of patients, including a comparative outcomes analysis of PH treatments that aimed to investigate different outcomes of interest such as, but not limited to, overall survival of the treated groups, liver toxicity, oedema/fluid retention, as well as the reason and number of hospitalisations. In addition, the study explored both the HCRU and the costs associated with the diagnosis. The study has now been finalised and the results will be published.
Dissemination plans are as follows:
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group in 2023.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results are expected be presented at appropriate conferences during 2023, and in other journals such as Pulmonary Circulation, International Journal of Cardiology, the European Journal of Health Economics and potential others.
• As of today one publication entitled "Healthcare resource utilisation, quality of life, and survival in patients with sarcoidosis-associated pulmonary hypertension" has been submitted to the Pulmonary Circulation journal and is waiting for peer-review
• Results will also be shared with other parties where appropriate. For example, sharing results with other NHS trusts that also manage PH patients or sharing with international centres that also diagnose and manage PH patients
All outputs will contain only data that is aggregated with small numbers suppressed in line with the HES Analysis Guide
Expected measurable benefits
There are likely benefits from this research for patients, the NHS, academia and life sciences companies. Overall, there are large gaps of knowledge within PH especially when looking at a subtype level (e.g. PAH, CTEPH, SAPH).
Understanding more about patient journeys through the secondary care system helped us identify ways of improving diagnosis and treatment, as well as providing evidence to support applications for novel therapies in this highly underserved disease area. This will allow patients to get access to new treatment options and provide health economic information to help design a more efficient care pathway for PH patients. This more efficient care pathway could potentially lessen the burden on patients by reducing repeat visits during patient’s diagnostic pathways and supporting earlier diagnosis to improve patient treatment outcomes. In heritable forms of the disease benefits may well subsequently advantage patient’s family members.
Patient pathway analysis:
• The healthcare community & academia will gain a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, provide earlier treatment and to improve quality of life for patients.
• Participants and non-participants will have increased access to information about their disease from the production of publications of study findings, which will be made available through the listed IQVIA Ltd website (noted on the posters at the STHFT), and potentially other channels e.g. PHA UK who support this research.
• The evidence produced will help inform research direction for novel treatment in this severely under-served disease.
• There is also the additional purpose of this study for the patients involved, as the study could potentially support the development of future novel drugs in clinical trials. The outcomes from this study could help provide vital insights into the PH (and its subtypes) population, and potential novel treatments that could be useful in the drug development process from its early stages through to post-authorisation.
Benefits reported so far
The main analysis and the study report have now been developed and IQVIA Ltd are currently developing the publication.
Datasets on the latest version
Legal basis for provision: Health and Social Care Act 2012 - s261 - 'Other dissemination of information'; National Health Service Act 2006 - s251 - 'Control of patient information'.
| Dataset | Type of data | Sensitivity | Frequency | Confidential data |
|---|---|---|---|---|
| Hospital Episode Statistics Accident and Emergency (HES A and E) | Anonymised - ICO Code Compliant | Non-Sensitive | One-Off | Section 251 NHS Act 2006 |
| Hospital Episode Statistics Admitted Patient Care (HES APC) | Anonymised - ICO Code Compliant | Non-Sensitive | One-Off | Section 251 NHS Act 2006 |
| Hospital Episode Statistics Outpatients (HES OP) | Anonymised - ICO Code Compliant | Non-Sensitive | One-Off | Section 251 NHS Act 2006 |
Files released
Files released counts only files released externally by DARS. Access granted in NHS England's own systems, such as its Secure Data Environment, is not included.
Patient opt-outs were applied to all 39 files released under this agreement, across every version. About opt-outs
No files recorded as released under the latest version. 39 were released under earlier versions, shown in the version history.
Version history
The register lists each renewal of this agreement as a separate row. This site has 4 versions.
DARS-NIC-296034-T4Y4K-v3.6 21 June 2023 to 18 May 2024
- Title
- Pulmonary Hypertension (PH) population characterisation and epidemiological analysis
- Commercial
- Yes
- Sublicensing
- No
- Datasets
- 3
- Files released
- 0
Datasets: Hospital Episode Statistics Accident and Emergency (HES A and E); Hospital Episode Statistics Admitted Patient Care (HES APC); Hospital Episode Statistics Outpatients (HES OP)
What changed from DARS-NIC-296034-T4Y4K-v2.4
Text removed is struck through; text added is underlined. Unchanged paragraphs are summarised rather than repeated.
| Field | Was | Became |
|---|---|---|
| Start date | 2023-06-21 | |
| End date | 2024-05-18 |
Data controllers:
− IQVIA TECHNOLOGY SERVICES LTD.
Objective for processing
This Data Sharing Agreement permits IQVIA Ltd to continue to hold the data for one year and process it for the verification of published findings if required.
As part of an internal reorganisation of its group of companies, IQVIA Technology Services Limited transferred its business functions to IQVIA Ltd. on 1 January 2023. Any reference to IQVIA Technology Services Ltd within the purpose section under this application has been removed unless it is historical information that was accurate at the time.
IQVIA Technology Services Ltd will have no further involvement under this Data Sharing Agreement and are no longer a Controller.
[3 paragraphs unchanged]
As a result, IQVIA Ltd and IQVIA Technology Services
Ltd, are carrying
Ltd carried
out a retrospective real world data analysis of UK PAH, CTEPH and SAPH patients for Janssen-Cilage Limited. The study
will focus
focused
on describing and comparing PAH, CTEPH and SAPH populations (and define sub-populations)
[12 words unchanged]
treatment patterns post diagnosis, clinical outcomes and Healthcare Resource Utilisation (HCRU). This
presents a great
presented an
opportunity for both Janssen and the broader scientific/research community to improve the
[21 words unchanged]
informing clinical guidelines to improve patient’s health and Quality of Life (QoL).
[1 paragraph unchanged]
The Pulmonary Hypertension Association UK,
agree
agreed
that the study “Pulmonary Hypertension (PH) population characterisation and epidemiological analysis”
will
would
benefit the broader scientific community by improving the body of knowledge around
[20 words unchanged]
to improve patient’s health and Quality of Life (QoL). This research study
represents for the members
will form
a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
There
is
was
an additional purpose of
this
the
study for the patients involved, namely that the study could potentially support
[42 words unchanged]
hence the joint involvement of each in the development of this study.
IQVIA Ltd and IQVIA Technology Services Ltd. will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd and IQVIA Technology Services Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd and IQVIA Technology Services Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Janssen-Cilag Limited will be processing the data in line with their research objectives as part of their legitimate interests. Janssen-Cilag Limited is a pharmaceutical company with interest in the pulmonary hypertension therapy area. It is funding this study to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. Janssen-Cilag Ltd. is particularly interested in understanding more about patient journeys through the secondary care system which can help identify ways of improving diagnosis and treatment, as well as potentially providing evidence to support applications for novel therapies in this highly under-served disease area. The reported results of this analysis may potentially assist in allowing patients to get access to new treatment options and provide health economic information to help design a more efficient care pathway for PH patients
Research being undertaken by is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
The use of this pseudonymised data will allow the data controllers and data processors to provide the research outputs to Sheffield Teaching Hospitals assist in enabling them to deliver better healthcare. This includes analysis and interpretation which provide understanding of care pathways, understanding use of medicines, and supporting healthcare commissioners and providers in achieving data quality, all these examples are with the aim of improving overall healthcare. The data processors also utilise this pseudonymised data to provide services to Janssen-Cilag Limited to improve healthcare and assist the efficiency of the National Health Service in the interest of public health. The service supports the development of innovative solutions and service improvement and helps to track outcomes and provide real-world evidence as required by the NHS, NICE, and NHS England with the aim of improving patient care and support enhanced access to improved services and innovative solutions. Individuals have got the right to opt out of the use of the HES data. All data removed from the storage location is small number suppressed as per HES Analytics guide.
Research overview
[5 paragraphs unchanged]
To achieve the above objectives, IQVIA Ltd
and IQVIA Technology Services Ltd propose to build
built
a joint dataset that has the required patient cohorts and clinical information. The database
will comprise
comprises
of non-identified patient data derived from the STHFT deep clinical databases which
[9 words unchanged]
Vascular Disease Unit (SPVDU) and national level hospital interactions from HES data.
[1 paragraph unchanged]
Each party in the collaboration
will have
had
a different role during the research:
1. STHFT
will take
had
responsibility for ethics approval for the study, provide expert clinical insight on
[8 words unchanged]
linkage of data in addition to supporting the publication of research findings
2. IQVIA Ltd
will support
and IQVIA Technology Services Ltd supported
STHFT ethical approvals activities,
co-develop
co-developed
the study protocol and SAP,
conduct
conducted
the transformation and data processing of non-identified data into
analyzable
analysable
format and
perform
performed
the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen)
will be funding
funded
the research and
will support
supported
the development of the study protocol and SAP and provide expert clinical insight on the findings.
4. IQVIA Ltd and IQVIA Technology Services Ltd
will each have
had
access to non-identifiable record level data and
create
created
aggregate outputs which
will be
were
shared with both Janssen and STHFT. Janssen
will
were
not
be
permitted to access record level HES data and
shall
will
only ever have access to aggregated data with small number suppressed in line with the HES Analysis Guide.
[1 paragraph unchanged]
Janssen
is funding
funded
the research to further the body of knowledge of PAH, CTEPH and
[60 words unchanged]
beneficial to patients, allowing them to share their learning with other centres.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This shall be comprised of two representatives of each ofSTHFT the UoS and Janssen and with IQVIA Ltd and IQVIA Technology Services Ltd having one representative who will chair the group.
It was important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This allowed IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data was critical in providing greater insight on all the patients who had attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH were included. Furthermore the STHFT data also provided information on in-depth patient clinical characteristics that allowed IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care was requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data was crucial to understand the complete patient pathway. All 20 of the diagnosis codes were requested in order to understand the comorbidities associated with PAH. The OPCS codes were also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity were required in order to characterise the patients.
The study design was a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd analysed the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which allowed IQVIA Ltd and IQVIA Technology Services Ltd to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. The cohort was approximately 3000 patients. NHS numbers and dates of birth were provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis required a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd requested ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
If required IQVIA Ltd will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Research being undertaken is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested has helped achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
If required Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This is comprised of two representatives of each of STHFT the UoS and Janssen and with IQVIA Ltd having one representative who will chair the group.
[4 paragraphs unchanged]
The studies chief investigator from STHFT will oversee the research and offer clinical insight on the findings.
The dissemination of findings has been pre-agreed with the committee and outlined in the outputs section.
It is important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This will allow IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data will be critical as it will provide great insight on all the patients who have attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH will be included. Furthermore, STHFT data will also provide information on in-depth patient clinical characteristics that will allow IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care is being requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data will be crucial to understand the complete patient pathway. All 20 of the diagnosis codes are being requested in order to understand the comorbidities associated with PAH. The OPCS codes are also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity are needed in order to characterise the patients. This data will allow the aims discussed in previous sections of this application to be achieved.
The study design is a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd are requesting the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which will allow IQVIA Ltd and IQVIA Technology Services Ltd. to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. It is estimated that this cohort will be around 3000 patients. NHS numbers and dates of birth will be provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis requires a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd and IQVIA Technology Services Ltd. are requesting ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd and IQVIA Technology Services Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
Processing activities
[1 paragraph unchanged]
To ensure the minimum amount of patient identifiable data will be used and handled by the fewest people outside of the direct care team the following process is currently proposed
IQVIA Ltd have finalised all of the analysis, however, require an extension to hold data in order to finalise the study report and publish the results. This Agreement permits IQVIA Ltd and STHFT to only further process the data for the purpose of verifying published findings if required.
1. STHFT will share with NHS Digital team, via a secure file transfer protocol, the NHS numbers and dates of birth of patients that have attended the SDPVU clinic since 2012, aligned to a generated study ID. The total number of this cohort is about 3000 patients
To ensure the minimum amount of patient identifiable data was used and handled by the fewest people outside of the direct care team the following process was implemented
2. NHS Digital will link the identifiable patient cohort to the following data sets; Admitted Patient Care, Outpatient and Accident & Emergency data. NHS Digital will remove the NHS numbers and dates of birth and returning the non-identified extract (including study ID) to the STHFT informatics team.
1. STHFT shared with NHS Digital team, via a secure file transfer protocol, the NHS numbers and dates of birth of patients that had attended the SDPVU clinic since 2012, aligned to a generated study ID. The total number of this cohort was about 3000 patients
3. Patient data received from STHFT will be linked to the HES data via the generated study ID and will be done in compliance with all trust policies on patient data handling. This data will only be accessible by the patient management team. NHS Digital will supply STHFT with the HES Data with a non-identifiable ID linked to the Study ID, which will allow STHFT to link the HES data to the diagnostic clinical data held at STHFT. The linked non-identified data will then be loaded to a second logical environment also located within STHFT.
2. NHS Digital linked the identifiable patient cohort to the following data sets; Admitted Patient Care, Outpatient and Accident & Emergency data. NHS Digital will remove the NHS numbers and dates of birth and returning the non-identified extract (including study ID) to the STHFT informatics team.
4. This environment will be remotely accessed within the STHFT “De-militarized Zone” (DMZ), which is a special local network configuration designed to improve security by segregating computers on each side of a firewall. This is an additional layer of security between the trusted internal network and an untrusted external network i.e. the internet. The research will be undertaken by trained researchers from IQVIA Ltd and IQVIA Technology Services Ltd and specific members from the University of Sheffield who are under honorary contracts or hold Research Passports at the STHFT. Access is granted using strong two factor authentication based on USB keys which produce one time use passwords (more information can be found at https://www.yubico.com/).
3. Patient data received from STHFT was linked to the HES data via the generated study ID and will be done in compliance with all trust policies on patient data handling. This data will only be accessible by the patient management team. NHS Digital supplied STHFT with the HES Data with a non-identifiable ID linked to the Study ID, which allowed STHFT to link the HES data to the diagnostic clinical data held at STHFT. The linked non-identified data was then loaded to a second logical environment also located within STHFT.
The research conducted on this combined dataset
will be
was
for the agreed research questions and
will be
was
performed on non-identified patient information. The HES linked data will not leave STHFT. Only aggregated non-patient level outputs will be shared.
The applicant expects to conduct the following analysis with the data, in order to generate real world evidence on the current clinical and treatment management of adult patients with a confirmed diagnosis of PAH, CTEPH or SAPH within the Sheffield EMR who have been treated with at least one PH-specific medical treatment.
The analysis was conducted in order to generate real world evidence on the current clinical and treatment management of adult patients with a confirmed diagnosis of PAH, CTEPH or SAPH within the Sheffield EMR who have been treated with at least one PH-specific medical treatment.
[4 paragraphs unchanged]
IQVIA Ltd have finalised all of the analysis, however, require a couple of months more to finalise the study report. The data will be retained at present as there is a plan to publish the results.
Researchers who had access to the patient level HES data were logged on an access control register, ensuring that it was possible to identify everyone with access to patient level information. Each researcher from IQVIA Ltd. and /or IQVIA Technology Services Ltd that had access to record level data signed an Intra-Company Agreement that contained information on best practice and rules. These rules were adhered to as stated in specific section of the contract, including the prevention of exporting any data from the Sheffield (STHFT) server that contravenes the HES small numbers protocol.
Researchers who will access the patient level HES data will be logged on an access control register, ensuring that it is possible to identify everyone with access to patient level information. Each researcher from IQVIA Ltd. and /or IQVIA Technology Services Ltd that will have access to record level data will sign an Intra-Company Agreement that will contain information on best practice and rules. These rules must be adhered to as stated in specific section of the contract, including the prevention of exporting any data from the Sheffield (STHFT) server that contravenes the HES small numbers protocol.
All individuals with access to the record level data were substantive employees of IQVIA Ltd, and or/STH save researchers from other parts of the IQVIA group, who may have been required from time to time to provide expertise in analysis of the data. These individuals worked under an Intra-Company Agreement to IQVIA Ltd. All individuals accessing the data under an Intra-Company Agreement issued by IQVIA Ltd were a substantive employee of the IQVIA company group.
All individuals with access to the record level data must be substantive employees of IQVIA Ltd, and/or IQVIA Technology Services Ltd, and or/STH save researchers from other parts of the IQVIA group, who may be required from time to time to provide expertise in analysis of the data. These individuals will work under an Intra-Company Agreement to IQVIA Ltd. All individuals accessing the data under an Intra-Company Agreement issued by IQVIA Ltd will be a substantive employee of the IQVIA company group. IQVIA Limited is not permitted under this Agreement to enter into an Intra-Company Agreement with any individual, who is not substantively employed by an IQVIA group company.
During the analysis process of the pseudonymised and aggregated data, there were regular sessions with Sheffield (STHFT) and Janssen clinical experts to provide clinical perspective and impact of the results generated.
During the analysis process of the pseudonymised and aggregated data, there will be regular sessions with Sheffield (STHFT) and Janssen clinical experts to provide clinical perspective and impact of the results generated.
Janssen-Cilag Limited did not carry out any processing of record level data under the previous version of this Agreement.
Janssen-Cilag Limited will not carry out any processing of data under this Agreement.
If required under this Data Sharing Agreement this environment will be remotely accessed within the STHFT “De-militarized Zone” (DMZ), which is a special local network configuration designed to improve security by segregating computers on each side of a firewall. This is an additional layer of security between the trusted internal network and an untrusted external network i.e. the internet. The research will be undertaken by trained researchers from IQVIA Ltd and specific members from the University of Sheffield who are under honorary contracts or hold Research Passports at the STHFT. Access is granted using strong two factor authentication based on USB keys which produce one time use passwords (more information can be found at https://www.yubico.com/).
[1 paragraph unchanged]
No data linkage will be undertaken with NHS
Digital
England
data provided under this Agreement except for the linkage already noted in the Agreement.
All organisations party to this agreement must comply with the Data Sharing Framework Contract requirements, including those regarding the use (and purposes of that use) by "Personnel" (as defined within the Data Sharing Framework Contract i.e. employees, agents and contractors of the Data Recipient who may have access to that data).
Expected output
IQVIA Ltd and IQVIA Technology Services Ltd
will conduct
conducted
initial analysis of the clinical pathway leading up to diagnosis and the post-diagnostic treatment patterns of patients, including a comparative outcomes analysis of PH treatments that
will aim
aimed
to investigate different outcomes of interest such as, but not limited to,
[11 words unchanged]
well as the reason and number of hospitalisations. In addition, the study
will explore
explored
both the HCRU and the costs associated with the diagnosis.
The study has now been finalised and the results will be published.
An amendment of the Data Sharing Agreement will be required in the future to support any supplementary questions arising from publications, in addition to supporting further publications to contribute to the body of knowledge on PH.
Dissemination plans are as follows:
In addition to the publication, additional dissemination plans are as follows:
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group in 2023.
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group at the beginning of the year 2022.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results are expected be presented at appropriate conferences during 2023, and in other journals such as Pulmonary Circulation, International Journal of Cardiology, the European Journal of Health Economics and potential others.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results will be presented at the European Society of Cardiology in August 2022, and in other journals such as the International Journal of Cardiology, the European Journal of Health Economics and potential others.
• As of today one publication entitled "Healthcare resource utilisation, quality of life, and survival in patients with sarcoidosis-associated pulmonary hypertension" has been submitted to the Pulmonary Circulation journal and is waiting for peer-review
[1 paragraph unchanged]
Note:
All outputs will contain only data that is aggregated with small numbers suppressed in line with the HES Analysis
Guide.
Guide
Expected measurable benefits
[1 paragraph unchanged]
Understanding more about patient journeys through the secondary care system
can help
helped us
identify ways of improving diagnosis and treatment, as well as
potentially
providing evidence to support applications for novel therapies in this highly underserved disease area. This
could potentially
will
allow patients to get access to new treatment options and provide health
[44 words unchanged]
forms of the disease benefits may well subsequently advantage patient’s family members.
[5 paragraphs unchanged]
Benefits reported
The main analysis
has
and the study report have
now been developed and IQVIA Ltd are currently developing the
study report. The publication for the end of 2021, or early 2022.
publication.
DARS-NIC-296034-T4Y4K-v2.4 19 May 2021 to 18 May 2022
- Title
- Pulmonary Hypertension (PH) population characterisation and epidemiological analysis
- Commercial
- Yes
- Sublicensing
- No
- Datasets
- 3
- Files released
- 0
Datasets: Hospital Episode Statistics Accident and Emergency (HES A and E); Hospital Episode Statistics Admitted Patient Care (HES APC); Hospital Episode Statistics Outpatients (HES OP)
What changed from DARS-NIC-296034-T4Y4K-v1.1
Text removed is struck through; text added is underlined. Unchanged paragraphs are summarised rather than repeated.
| Field | Was | Became |
|---|---|---|
| Start date | 2021-05-19 | |
| End date | 2022-05-18 | |
| Hospital Episode Statistics Accident and Emergency (HES A and E): legal basis | Health and Social Care Act 2012 - s261 - 'Other dissemination of information'; National Health Service Act 2006 - s251 - 'Control of patient information'. | |
| Hospital Episode Statistics Admitted Patient Care (HES APC): legal basis | Health and Social Care Act 2012 - s261 - 'Other dissemination of information'; National Health Service Act 2006 - s251 - 'Control of patient information'. | |
| Hospital Episode Statistics Outpatients (HES OP): legal basis | Health and Social Care Act 2012 - s261 - 'Other dissemination of information'; National Health Service Act 2006 - s251 - 'Control of patient information'. |
Objective for processing
[5 paragraphs unchanged]
The Pulmonary Hypertension Association UK, agree that the study “Pulmonary Hypertension
The Pulmonary Hypertension Association UK, agree that the study “Pulmonary Hypertension (PH) population characterisation and epidemiological analysis” will benefit the broader scientific community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL). This research study represents for the members a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
(PH) population characterisation and epidemiological analysis” will benefit the broader scientific
community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informingclinical guidelines to improve patient’s health and Quality of Life (QoL). This research study represents for the members a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
[4 paragraphs unchanged]
Reseacrh veung
Research being
undertaken by is covered under the GDPR Article 6(1)(f) - This work
[72 words unchanged]
improvement of clinical services as well as potential support for drug development.
[13 paragraphs unchanged]
2. IQVIA Ltd will support STHFT ethical approvals activities, co-develop the study protocol and SAP, conduct the transformation and data processing of non-identified data into
analysable
analyzable
format and perform the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen) will be funding the research and
willvsupport
will support
the development of the study protocol and SAP and provide expert clinical insight on the findings.
[19 paragraphs unchanged]
Processing activities
No additional data will be disseminated during this Agreement.
[10 paragraphs unchanged]
These analyses are expected to be completed 9 to 12 months after HES data has been provided.
IQVIA Ltd have finalised all of the analysis, however, require a couple of months more to finalise the study report. The data will be retained at present as there is a plan to publish the results.
[1 paragraph unchanged]
All individuals with access to the record level data must be substantive
[43 words unchanged]
Agreement to IQVIA Ltd. All individuals accessing the data under an Intra-Company
vAgreement
Agreement
issued by IQVIA Ltd will be a substantive employee of the IQVIA company group. IQVIA Limited is not permitted under this
agreement
Agreement
to enter into an Intra-Company Agreement with any individual, who is not substantively employed by an IQVIA group company.
[5 paragraphs unchanged]
Expected output
[3 paragraphs unchanged]
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group at the beginning of the year
2021.
2022.
• Furthermore, the abstracts and links to publications will be hosted on
[8 words unchanged]
Results will be presented at the European Society of Cardiology in August
2021,
2022,
and in other journals such as the International Journal of Cardiology, the European Journal of Health Economics and potential others.
[2 paragraphs unchanged]
Benefits reported
Not stated in the previous version; added here.
The main analysis has now been developed and IQVIA Ltd are currently developing the study report. The publication for the end of 2021, or early 2022.
Unchanged: Expected measurable benefits.
Objective for processing
Pulmonary Arterial Hypertension (PAH) (a type of Pulmonary Hypertension (PH)), is a progressive and rare disease caused by narrowing or tightening (constriction) of the pulmonary arteries. PAH primarily affects the small pulmonary arterioles through vascular proliferation and re-modelling, while resulting in a progressive rise in lung blood pressure and heart failure. PAH represents Group 1 (of 5) within the Pulmonary Hypertension World Health Organisation clinical classification system (Dana Point, 2008). In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. Furthermore, there are several types of PAH including Idiopathic PAH (iPAH) and Associated PAH related to a range of disease processes, including cirrhosis, connective tissue disease, congenital heart disease, HIV infection and sickle-cell disease
In addition, there are other types of PH, including Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Sarcoidosis-Associated Pulmonary Hypertension (SAPH). CTEPH is caused by complications of acute Pulmonary embolism (PE), leading to an obstruction of the large pulmonary arteries and distal small-vessel arteriopathy, with an estimated incidence of 3-5 cases per 100,000 patients. Sarcoidosis is a multisystem immune-mediated inflammatory disorder categorised by the presence of granulomas in tissues and is highly associated with respiratory tissue. Sarcoid patients may develop SAPH, which can lead to significant complications.
Due to the complexity, late diagnosis and absence of real world studies across all subtypes of PH (PAH, CTEPH and SAPH), there is currently a lack of understanding regarding the clinical pathway leading to diagnosis. In addition, there is also the need to further explore and study post-diagnosis pathways and patient related outcomes associated with these diseases in routine clinical practice.
As a result, IQVIA Ltd and IQVIA Technology Services Ltd, are carrying out a retrospective real world data analysis of UK PAH, CTEPH and SAPH patients for Janssen-Cilage Limited. The study will focus on describing and comparing PAH, CTEPH and SAPH populations (and define sub-populations) in terms of characteristics of patients, clinical pathways pre-diagnosis and diagnostic procedures, treatment patterns post diagnosis, clinical outcomes and Healthcare Resource Utilisation (HCRU). This presents a great opportunity for both Janssen and the broader scientific/research community to improve the body of knowledge around these three sub-types and further enhance the possibility of developing better clinical pathways, improving treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL).
The Pulmonary Hypertension Association UK (PHA UK) is the UKs largest and foremost patient advocacy group for those suffering from this life threatening and shortening condition; PHA UK has some 4,340 members representing the views of a large part of the UK patient population.
The Pulmonary Hypertension Association UK, agree that the study “Pulmonary Hypertension (PH) population characterisation and epidemiological analysis” will benefit the broader scientific community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL). This research study represents for the members a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
There is an additional purpose of this study for the patients involved, namely that the study could potentially support the development of future novel drugs in clinical trials. It was determined that the combined analysis of HES data and the enhanced diagnostic clinical data jointly held by STHFT and the University of Sheffield (UoS) could be very beneficial to this study, hence the joint involvement of each in the development of this study.
IQVIA Ltd and IQVIA Technology Services Ltd. will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd and IQVIA Technology Services Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd and IQVIA Technology Services Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Janssen-Cilag Limited will be processing the data in line with their research objectives as part of their legitimate interests. Janssen-Cilag Limited is a pharmaceutical company with interest in the pulmonary hypertension therapy area. It is funding this study to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. Janssen-Cilag Ltd. is particularly interested in understanding more about patient journeys through the secondary care system which can help identify ways of improving diagnosis and treatment, as well as potentially providing evidence to support applications for novel therapies in this highly under-served disease area. The reported results of this analysis may potentially assist in allowing patients to get access to new treatment options and provide health economic information to help design a more efficient care pathway for PH patients
Research being undertaken by is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
The use of this pseudonymised data will allow the data controllers and data processors to provide the research outputs to Sheffield Teaching Hospitals assist in enabling them to deliver better healthcare. This includes analysis and interpretation which provide understanding of care pathways, understanding use of medicines, and supporting healthcare commissioners and providers in achieving data quality, all these examples are with the aim of improving overall healthcare. The data processors also utilise this pseudonymised data to provide services to Janssen-Cilag Limited to improve healthcare and assist the efficiency of the National Health Service in the interest of public health. The service supports the development of innovative solutions and service improvement and helps to track outcomes and provide real-world evidence as required by the NHS, NICE, and NHS England with the aim of improving patient care and support enhanced access to improved services and innovative solutions. Individuals have got the right to opt out of the use of the HES data. All data removed from the storage location is small number suppressed as per HES Analytics guide.
Research overview
The goal of the research is to:
• Generate real world evidence on the clinical and treatment management of patients diagnosed with PAH, CTEPH and SAPH
• Analyse the characteristics of diagnosed patients at the time of confirmed diagnosis, and the clinical pathways leading up to diagnosis, diagnostic procedures and the post-diagnostic treatment patterns
• Investigate various clinical outcomes associated with PH and its subtypes, and comparative outcomes analysis of PH treatments
• To explore the healthcare resource use (HCRU) and the costs associated with the diagnosis of PAH, CTEPH and SAPH
To achieve the above objectives, IQVIA Ltd and IQVIA Technology Services Ltd propose to build a joint dataset that has the required patient cohorts and clinical information. The database will comprise of non-identified patient data derived from the STHFT deep clinical databases which collect data on all patients attending the Sheffield Pulmonary Vascular Disease Unit (SPVDU) and national level hospital interactions from HES data.
Parties involved in the research:
Each party in the collaboration will have a different role during the research:
1. STHFT will take responsibility for ethics approval for the study, provide expert clinical insight on the research findings, support on datasets de-identification and linkage of data in addition to supporting the publication of research findings
2. IQVIA Ltd will support STHFT ethical approvals activities, co-develop the study protocol and SAP, conduct the transformation and data processing of non-identified data into analyzable format and perform the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen) will be funding the research and will support the development of the study protocol and SAP and provide expert clinical insight on the findings.
4. IQVIA Ltd and IQVIA Technology Services Ltd will each have access to non-identifiable record level data and create aggregate outputs which will be shared with both Janssen and STHFT. Janssen will not be permitted to access record level HES data and shall only ever have access to aggregated data with small number suppressed in line with the HES Analysis Guide.
5. STHFT and Janssen will provide clinical interpretation of the results.
Janssen is funding the research to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. STHFT as one of England’s leading PH diagnostic and treatment centres benefits from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPDVU), in addition to the verification that their unique diagnostic process is beneficial to patients, allowing them to share their learning with other centres.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This shall be comprised of two representatives of each ofSTHFT the UoS and Janssen and with IQVIA Ltd and IQVIA Technology Services Ltd having one representative who will chair the group.
The committee will perform the following functions:
1) Provide clinical interpretation of the results to support refinements of the analysis within the bounds of the protocol
2) Agree the dissemination / publication routes for research findings (e.g. conference posters vs peer review papers etc.) based on the nature and strength of findings. (Please see output section for further information)
No organisation on the clinical interpretation group or any of the data controllers will have the ability to suppress any of the findings or outputs of the analysis. The clinical interpretation group members do not have any access to record level data.
The studies chief investigator from STHFT will oversee the research and offer clinical insight on the findings.
The dissemination of findings has been pre-agreed with the committee and outlined in the outputs section.
It is important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This will allow IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data will be critical as it will provide great insight on all the patients who have attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH will be included. Furthermore, STHFT data will also provide information on in-depth patient clinical characteristics that will allow IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care is being requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data will be crucial to understand the complete patient pathway. All 20 of the diagnosis codes are being requested in order to understand the comorbidities associated with PAH. The OPCS codes are also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity are needed in order to characterise the patients. This data will allow the aims discussed in previous sections of this application to be achieved.
The study design is a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd are requesting the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which will allow IQVIA Ltd and IQVIA Technology Services Ltd. to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. It is estimated that this cohort will be around 3000 patients. NHS numbers and dates of birth will be provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis requires a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd and IQVIA Technology Services Ltd. are requesting ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd and IQVIA Technology Services Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
Expected output
IQVIA Ltd and IQVIA Technology Services Ltd will conduct initial analysis of the clinical pathway leading up to diagnosis and the post-diagnostic treatment patterns of patients, including a comparative outcomes analysis of PH treatments that will aim to investigate different outcomes of interest such as, but not limited to, overall survival of the treated groups, liver toxicity, oedema/fluid retention, as well as the reason and number of hospitalisations. In addition, the study will explore both the HCRU and the costs associated with the diagnosis.
An amendment of the Data Sharing Agreement will be required in the future to support any supplementary questions arising from publications, in addition to supporting further publications to contribute to the body of knowledge on PH.
In addition to the publication, additional dissemination plans are as follows:
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group at the beginning of the year 2022.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results will be presented at the European Society of Cardiology in August 2022, and in other journals such as the International Journal of Cardiology, the European Journal of Health Economics and potential others.
• Results will also be shared with other parties where appropriate. For example, sharing results with other NHS trusts that also manage PH patients or sharing with international centres that also diagnose and manage PH patients
Note: All outputs will contain only data that is aggregated with small numbers suppressed in line with the HES Analysis Guide.
Benefits reported
The main analysis has now been developed and IQVIA Ltd are currently developing the study report. The publication for the end of 2021, or early 2022.
DARS-NIC-296034-T4Y4K-v1.1 20 August 2020 to 18 May 2021
- Title
- Pulmonary Hypertension (PH) population characterisation and epidemiological analysis
- Commercial
- Yes
- Sublicensing
- No
- Datasets
- 3
- Files released
- 1
Datasets: Hospital Episode Statistics Accident and Emergency (HES A and E); Hospital Episode Statistics Admitted Patient Care (HES APC); Hospital Episode Statistics Outpatients (HES OP)
What changed from DARS-NIC-296034-T4Y4K-v0.16
Text removed is struck through; text added is underlined. Unchanged paragraphs are summarised rather than repeated.
| Field | Was | Became |
|---|---|---|
| Start date | 2020-08-20 |
Benefits reported
Stated in the previous version and removed here.
Yielded Benefits is not a requirement for new applications.
Unchanged: Objective for processing, Processing activities, Expected output, Expected measurable benefits.
Objective for processing
Pulmonary Arterial Hypertension (PAH) (a type of Pulmonary Hypertension (PH)), is a progressive and rare disease caused by narrowing or tightening (constriction) of the pulmonary arteries. PAH primarily affects the small pulmonary arterioles through vascular proliferation and re-modelling, while resulting in a progressive rise in lung blood pressure and heart failure. PAH represents Group 1 (of 5) within the Pulmonary Hypertension World Health Organisation clinical classification system (Dana Point, 2008). In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. Furthermore, there are several types of PAH including Idiopathic PAH (iPAH) and Associated PAH related to a range of disease processes, including cirrhosis, connective tissue disease, congenital heart disease, HIV infection and sickle-cell disease
In addition, there are other types of PH, including Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Sarcoidosis-Associated Pulmonary Hypertension (SAPH). CTEPH is caused by complications of acute Pulmonary embolism (PE), leading to an obstruction of the large pulmonary arteries and distal small-vessel arteriopathy, with an estimated incidence of 3-5 cases per 100,000 patients. Sarcoidosis is a multisystem immune-mediated inflammatory disorder categorised by the presence of granulomas in tissues and is highly associated with respiratory tissue. Sarcoid patients may develop SAPH, which can lead to significant complications.
Due to the complexity, late diagnosis and absence of real world studies across all subtypes of PH (PAH, CTEPH and SAPH), there is currently a lack of understanding regarding the clinical pathway leading to diagnosis. In addition, there is also the need to further explore and study post-diagnosis pathways and patient related outcomes associated with these diseases in routine clinical practice.
As a result, IQVIA Ltd and IQVIA Technology Services Ltd, are carrying out a retrospective real world data analysis of UK PAH, CTEPH and SAPH patients for Janssen-Cilage Limited. The study will focus on describing and comparing PAH, CTEPH and SAPH populations (and define sub-populations) in terms of characteristics of patients, clinical pathways pre-diagnosis and diagnostic procedures, treatment patterns post diagnosis, clinical outcomes and Healthcare Resource Utilisation (HCRU). This presents a great opportunity for both Janssen and the broader scientific/research community to improve the body of knowledge around these three sub-types and further enhance the possibility of developing better clinical pathways, improving treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL).
The Pulmonary Hypertension Association UK (PHA UK) is the UKs largest and foremost patient advocacy group for those suffering from this life threatening and shortening condition; PHA UK has some 4,340 members representing the views of a large part of the UK patient population.
The Pulmonary Hypertension Association UK, agree that the study “Pulmonary Hypertension
(PH) population characterisation and epidemiological analysis” will benefit the broader scientific
community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informingclinical guidelines to improve patient’s health and Quality of Life (QoL). This research study represents for the members a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
There is an additional purpose of this study for the patients involved, namely that the study could potentially support the development of future novel drugs in clinical trials. It was determined that the combined analysis of HES data and the enhanced diagnostic clinical data jointly held by STHFT and the University of Sheffield (UoS) could be very beneficial to this study, hence the joint involvement of each in the development of this study.
IQVIA Ltd and IQVIA Technology Services Ltd. will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd and IQVIA Technology Services Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd and IQVIA Technology Services Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Janssen-Cilag Limited will be processing the data in line with their research objectives as part of their legitimate interests. Janssen-Cilag Limited is a pharmaceutical company with interest in the pulmonary hypertension therapy area. It is funding this study to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. Janssen-Cilag Ltd. is particularly interested in understanding more about patient journeys through the secondary care system which can help identify ways of improving diagnosis and treatment, as well as potentially providing evidence to support applications for novel therapies in this highly under-served disease area. The reported results of this analysis may potentially assist in allowing patients to get access to new treatment options and provide health economic information to help design a more efficient care pathway for PH patients
Reseacrh veung undertaken by is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
The use of this pseudonymised data will allow the data controllers and data processors to provide the research outputs to Sheffield Teaching Hospitals assist in enabling them to deliver better healthcare. This includes analysis and interpretation which provide understanding of care pathways, understanding use of medicines, and supporting healthcare commissioners and providers in achieving data quality, all these examples are with the aim of improving overall healthcare. The data processors also utilise this pseudonymised data to provide services to Janssen-Cilag Limited to improve healthcare and assist the efficiency of the National Health Service in the interest of public health. The service supports the development of innovative solutions and service improvement and helps to track outcomes and provide real-world evidence as required by the NHS, NICE, and NHS England with the aim of improving patient care and support enhanced access to improved services and innovative solutions. Individuals have got the right to opt out of the use of the HES data. All data removed from the storage location is small number suppressed as per HES Analytics guide.
Research overview
The goal of the research is to:
• Generate real world evidence on the clinical and treatment management of patients diagnosed with PAH, CTEPH and SAPH
• Analyse the characteristics of diagnosed patients at the time of confirmed diagnosis, and the clinical pathways leading up to diagnosis, diagnostic procedures and the post-diagnostic treatment patterns
• Investigate various clinical outcomes associated with PH and its subtypes, and comparative outcomes analysis of PH treatments
• To explore the healthcare resource use (HCRU) and the costs associated with the diagnosis of PAH, CTEPH and SAPH
To achieve the above objectives, IQVIA Ltd and IQVIA Technology Services Ltd propose to build a joint dataset that has the required patient cohorts and clinical information. The database will comprise of non-identified patient data derived from the STHFT deep clinical databases which collect data on all patients attending the Sheffield Pulmonary Vascular Disease Unit (SPVDU) and national level hospital interactions from HES data.
Parties involved in the research:
Each party in the collaboration will have a different role during the research:
1. STHFT will take responsibility for ethics approval for the study, provide expert clinical insight on the research findings, support on datasets de-identification and linkage of data in addition to supporting the publication of research findings
2. IQVIA Ltd will support STHFT ethical approvals activities, co-develop the study protocol and SAP, conduct the transformation and data processing of non-identified data into analysable format and perform the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen) will be funding the research and willvsupport the development of the study protocol and SAP and provide expert clinical insight on the findings.
4. IQVIA Ltd and IQVIA Technology Services Ltd will each have access to non-identifiable record level data and create aggregate outputs which will be shared with both Janssen and STHFT. Janssen will not be permitted to access record level HES data and shall only ever have access to aggregated data with small number suppressed in line with the HES Analysis Guide.
5. STHFT and Janssen will provide clinical interpretation of the results.
Janssen is funding the research to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. STHFT as one of England’s leading PH diagnostic and treatment centres benefits from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPDVU), in addition to the verification that their unique diagnostic process is beneficial to patients, allowing them to share their learning with other centres.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This shall be comprised of two representatives of each ofSTHFT the UoS and Janssen and with IQVIA Ltd and IQVIA Technology Services Ltd having one representative who will chair the group.
The committee will perform the following functions:
1) Provide clinical interpretation of the results to support refinements of the analysis within the bounds of the protocol
2) Agree the dissemination / publication routes for research findings (e.g. conference posters vs peer review papers etc.) based on the nature and strength of findings. (Please see output section for further information)
No organisation on the clinical interpretation group or any of the data controllers will have the ability to suppress any of the findings or outputs of the analysis. The clinical interpretation group members do not have any access to record level data.
The studies chief investigator from STHFT will oversee the research and offer clinical insight on the findings.
The dissemination of findings has been pre-agreed with the committee and outlined in the outputs section.
It is important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This will allow IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data will be critical as it will provide great insight on all the patients who have attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH will be included. Furthermore, STHFT data will also provide information on in-depth patient clinical characteristics that will allow IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care is being requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data will be crucial to understand the complete patient pathway. All 20 of the diagnosis codes are being requested in order to understand the comorbidities associated with PAH. The OPCS codes are also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity are needed in order to characterise the patients. This data will allow the aims discussed in previous sections of this application to be achieved.
The study design is a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd are requesting the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which will allow IQVIA Ltd and IQVIA Technology Services Ltd. to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. It is estimated that this cohort will be around 3000 patients. NHS numbers and dates of birth will be provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis requires a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd and IQVIA Technology Services Ltd. are requesting ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd and IQVIA Technology Services Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
Expected output
IQVIA Ltd and IQVIA Technology Services Ltd will conduct initial analysis of the clinical pathway leading up to diagnosis and the post-diagnostic treatment patterns of patients, including a comparative outcomes analysis of PH treatments that will aim to investigate different outcomes of interest such as, but not limited to, overall survival of the treated groups, liver toxicity, oedema/fluid retention, as well as the reason and number of hospitalisations. In addition, the study will explore both the HCRU and the costs associated with the diagnosis.
An amendment of the Data Sharing Agreement will be required in the future to support any supplementary questions arising from publications, in addition to supporting further publications to contribute to the body of knowledge on PH.
In addition to the publication, additional dissemination plans are as follows:
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group at the beginning of the year 2021.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results will be presented at the European Society of Cardiology in August 2021, and in other journals such as the International Journal of Cardiology, the European Journal of Health Economics and potential others.
• Results will also be shared with other parties where appropriate. For example, sharing results with other NHS trusts that also manage PH patients or sharing with international centres that also diagnose and manage PH patients
Note: All outputs will contain only data that is aggregated with small numbers suppressed in line with the HES Analysis Guide.
DARS-NIC-296034-T4Y4K-v0.16 19 May 2020 to 18 May 2021
- Title
- Pulmonary Hypertension (PH) population characterisation and epidemiological analysis
- Commercial
- Yes
- Sublicensing
- No
- Datasets
- 3
- Files released
- 38
Datasets: Hospital Episode Statistics Accident and Emergency (HES A and E); Hospital Episode Statistics Admitted Patient Care (HES APC); Hospital Episode Statistics Outpatients (HES OP)
Objective for processing
Pulmonary Arterial Hypertension (PAH) (a type of Pulmonary Hypertension (PH)), is a progressive and rare disease caused by narrowing or tightening (constriction) of the pulmonary arteries. PAH primarily affects the small pulmonary arterioles through vascular proliferation and re-modelling, while resulting in a progressive rise in lung blood pressure and heart failure. PAH represents Group 1 (of 5) within the Pulmonary Hypertension World Health Organisation clinical classification system (Dana Point, 2008). In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. Furthermore, there are several types of PAH including Idiopathic PAH (iPAH) and Associated PAH related to a range of disease processes, including cirrhosis, connective tissue disease, congenital heart disease, HIV infection and sickle-cell disease
In addition, there are other types of PH, including Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Sarcoidosis-Associated Pulmonary Hypertension (SAPH). CTEPH is caused by complications of acute Pulmonary embolism (PE), leading to an obstruction of the large pulmonary arteries and distal small-vessel arteriopathy, with an estimated incidence of 3-5 cases per 100,000 patients. Sarcoidosis is a multisystem immune-mediated inflammatory disorder categorised by the presence of granulomas in tissues and is highly associated with respiratory tissue. Sarcoid patients may develop SAPH, which can lead to significant complications.
Due to the complexity, late diagnosis and absence of real world studies across all subtypes of PH (PAH, CTEPH and SAPH), there is currently a lack of understanding regarding the clinical pathway leading to diagnosis. In addition, there is also the need to further explore and study post-diagnosis pathways and patient related outcomes associated with these diseases in routine clinical practice.
As a result, IQVIA Ltd and IQVIA Technology Services Ltd, are carrying out a retrospective real world data analysis of UK PAH, CTEPH and SAPH patients for Janssen-Cilage Limited. The study will focus on describing and comparing PAH, CTEPH and SAPH populations (and define sub-populations) in terms of characteristics of patients, clinical pathways pre-diagnosis and diagnostic procedures, treatment patterns post diagnosis, clinical outcomes and Healthcare Resource Utilisation (HCRU). This presents a great opportunity for both Janssen and the broader scientific/research community to improve the body of knowledge around these three sub-types and further enhance the possibility of developing better clinical pathways, improving treatment patterns and informing clinical guidelines to improve patient’s health and Quality of Life (QoL).
The Pulmonary Hypertension Association UK (PHA UK) is the UKs largest and foremost patient advocacy group for those suffering from this life threatening and shortening condition; PHA UK has some 4,340 members representing the views of a large part of the UK patient population.
The Pulmonary Hypertension Association UK, agree that the study “Pulmonary Hypertension
(PH) population characterisation and epidemiological analysis” will benefit the broader scientific
community by improving the body of knowledge around PH and its subtypes, while enhancing the possibility of developing better clinical pathways, advancing treatment patterns and informingclinical guidelines to improve patient’s health and Quality of Life (QoL). This research study represents for the members a crucial part of PHA UK's collective endeavours to find new and more efficacious therapies for those with PH.
There is an additional purpose of this study for the patients involved, namely that the study could potentially support the development of future novel drugs in clinical trials. It was determined that the combined analysis of HES data and the enhanced diagnostic clinical data jointly held by STHFT and the University of Sheffield (UoS) could be very beneficial to this study, hence the joint involvement of each in the development of this study.
IQVIA Ltd and IQVIA Technology Services Ltd. will be processing the data in line with their related to the research objectives, as part of their legitimate interests. IQVIA Ltd and IQVIA Technology Services Ltd are affiliated healthcare technology companies with interest in medical research which typically establishes relationships between pharma companies and healthcare providers to further this interest. IQVIA Ltd and IQVIA Technology Services Ltd considers this study will provide the healthcare community and academia with a better understanding of the diagnosis and treatment of PH patients in England, providing opportunities to identify areas to improve services, improve the patient journey, as well as opportunities to provide earlier treatment and to improve quality of life for patients. It is anticipated that patients and other healthcare stakeholders will have increased access to information about the disease given the planned production of publications of study findings. The evidence produced upon completion of the study could potentially help inform research direction for novel treatment in this severely under-served disease.
Covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Janssen-Cilag Limited will be processing the data in line with their research objectives as part of their legitimate interests. Janssen-Cilag Limited is a pharmaceutical company with interest in the pulmonary hypertension therapy area. It is funding this study to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. Janssen-Cilag Ltd. is particularly interested in understanding more about patient journeys through the secondary care system which can help identify ways of improving diagnosis and treatment, as well as potentially providing evidence to support applications for novel therapies in this highly under-served disease area. The reported results of this analysis may potentially assist in allowing patients to get access to new treatment options and provide health economic information to help design a more efficient care pathway for PH patients
Reseacrh veung undertaken by is covered under the GDPR Article 6(1)(f) - This work is necessary for the purposes of the legitimate interests pursued by the controller except where such interests are overridden by the interests or fundamental rights and freedoms of the data subject which require protection of personal data, in particular where the data subject is a child. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
Sheffield Teaching Hospitals NHS Foundation Trust will be processing the data in line with its goals to carry out the task in the public interest. STHFT one of England’s leading PH diagnostic and treatment centres considers that this analysis may provide it with information to better support their patients through the full care pathway. They consider that their patients will benefit from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPVDU) – leading to a more coordinated approach to care - in addition to understanding the outcomes of its unique diagnosis process and allowing it to share its learning with other centres.
Research being undertaken by Sheffield Teaching Hospitals NHS Foundation Trust is covered under the GDPR Article 6(1)(e) - Processing is necessary for the performance of a task carried out in the public interest or in the exercise of official authority vested in the controller. The data requested is to help achieve the following: pre- and post-diagnosis pathway analysis, health economics/burden of illness studies, treatment pathway analysis, diagnostic pathway assessment and improvement of clinical services as well as potential support for drug development.
The use of this pseudonymised data will allow the data controllers and data processors to provide the research outputs to Sheffield Teaching Hospitals assist in enabling them to deliver better healthcare. This includes analysis and interpretation which provide understanding of care pathways, understanding use of medicines, and supporting healthcare commissioners and providers in achieving data quality, all these examples are with the aim of improving overall healthcare. The data processors also utilise this pseudonymised data to provide services to Janssen-Cilag Limited to improve healthcare and assist the efficiency of the National Health Service in the interest of public health. The service supports the development of innovative solutions and service improvement and helps to track outcomes and provide real-world evidence as required by the NHS, NICE, and NHS England with the aim of improving patient care and support enhanced access to improved services and innovative solutions. Individuals have got the right to opt out of the use of the HES data. All data removed from the storage location is small number suppressed as per HES Analytics guide.
Research overview
The goal of the research is to:
• Generate real world evidence on the clinical and treatment management of patients diagnosed with PAH, CTEPH and SAPH
• Analyse the characteristics of diagnosed patients at the time of confirmed diagnosis, and the clinical pathways leading up to diagnosis, diagnostic procedures and the post-diagnostic treatment patterns
• Investigate various clinical outcomes associated with PH and its subtypes, and comparative outcomes analysis of PH treatments
• To explore the healthcare resource use (HCRU) and the costs associated with the diagnosis of PAH, CTEPH and SAPH
To achieve the above objectives, IQVIA Ltd and IQVIA Technology Services Ltd propose to build a joint dataset that has the required patient cohorts and clinical information. The database will comprise of non-identified patient data derived from the STHFT deep clinical databases which collect data on all patients attending the Sheffield Pulmonary Vascular Disease Unit (SPVDU) and national level hospital interactions from HES data.
Parties involved in the research:
Each party in the collaboration will have a different role during the research:
1. STHFT will take responsibility for ethics approval for the study, provide expert clinical insight on the research findings, support on datasets de-identification and linkage of data in addition to supporting the publication of research findings
2. IQVIA Ltd will support STHFT ethical approvals activities, co-develop the study protocol and SAP, conduct the transformation and data processing of non-identified data into analysable format and perform the analysis to meet the research objectives
3. Janssen- Clilag Ltd (Janssen) will be funding the research and willvsupport the development of the study protocol and SAP and provide expert clinical insight on the findings.
4. IQVIA Ltd and IQVIA Technology Services Ltd will each have access to non-identifiable record level data and create aggregate outputs which will be shared with both Janssen and STHFT. Janssen will not be permitted to access record level HES data and shall only ever have access to aggregated data with small number suppressed in line with the HES Analysis Guide.
5. STHFT and Janssen will provide clinical interpretation of the results.
Janssen is funding the research to further the body of knowledge of PAH, CTEPH and SAPH populations in terms of patient characteristics, clinical pathways pre-diagnosis, treatment patterns post diagnosis, clinical outcomes and HCRU. STHFT as one of England’s leading PH diagnostic and treatment centres benefits from the research by furthering their understanding of patient journeys outside of the Sheffield Pulmonary Vascular Disease Unit (SPDVU), in addition to the verification that their unique diagnostic process is beneficial to patients, allowing them to share their learning with other centres.
To ensure findings are published fairly and not suppressed there will be a clinical interpretation committee in place. This shall be comprised of two representatives of each ofSTHFT the UoS and Janssen and with IQVIA Ltd and IQVIA Technology Services Ltd having one representative who will chair the group.
The committee will perform the following functions:
1) Provide clinical interpretation of the results to support refinements of the analysis within the bounds of the protocol
2) Agree the dissemination / publication routes for research findings (e.g. conference posters vs peer review papers etc.) based on the nature and strength of findings. (Please see output section for further information)
No organisation on the clinical interpretation group or any of the data controllers will have the ability to suppress any of the findings or outputs of the analysis. The clinical interpretation group members do not have any access to record level data.
The studies chief investigator from STHFT will oversee the research and offer clinical insight on the findings.
The dissemination of findings has been pre-agreed with the committee and outlined in the outputs section.
It is important when linking HES data with the STHFT dataset to utilise the confirmed and sub-typed (PAH, CTEPH and SAPH) patient diagnoses present in the STHFT dataset, where the patient PH classification has been confirmed by world leading clinical experts. This will allow IQVIA Ltd and IQVIA Technology Services Ltd to identify patients with confirmed PAH, CTEPH and SAPH diagnoses within the HES data for investigation and analysis with high certainty. Current ICD-10 coding available in HES (the International classification system for coding of disease types, maintained by the World Health Organisation) does not have a specific code for the different PH sub-types (PAH, CTEPH and SAPH), instead multiple different pulmonary diseases coded under the same ICD-10 4-level character code. In addition, coding is not consistently applied across centres, meaning that PH patients in HES are coded across many different ICD-10 codes and therefore confirmation of disease and subtype in HES alone is not possible with complete certainty.
Access to STHFT data will be critical as it will provide great insight on all the patients who have attended SPDVU since 2012, to gain a better understanding of both the diagnostic pathway and process at SPDVU. Patients diagnosed with PAH, CTEPH or SAPH will be included. Furthermore, STHFT data will also provide information on in-depth patient clinical characteristics that will allow IQVIA Ltd and IQVIA Technology Services Ltd to characterise these patients at different points of the patient journey and develop matched cohorts for conducting comparative outcomes analysis across different PH treatments, and further investigate the variation in clinical outcomes of patients that have other comorbidities. Data from Hospital Episode Statistics Accident and Emergency, Hospital Episode Statistics Outpatients and Hospital Episode Statistics Admitted Patient Care is being requested in order to understand the complete patient pathway. Often, PAH patients may be admitted to A&E and may be taken as inpatients in relation to their PAH diagnosis, therefore this data will be crucial to understand the complete patient pathway. All 20 of the diagnosis codes are being requested in order to understand the comorbidities associated with PAH. The OPCS codes are also required in order to understand the procedures that a patient may have undergone as a results of their PAH diagnosis. Other data requests such as age, gender and ethnicity are needed in order to characterise the patients. This data will allow the aims discussed in previous sections of this application to be achieved.
The study design is a non-interventional retrospective database analysis of data collected on patients with PH (including its subtypes; PAH, CTEPH and SAPH) based on data from the Pulmonary Vascular Disease Unit at Sheffield Teaching Hospitals NHS Foundation Trust (STHFT) and HES data from NHS Digital.
To facilitate this project, IQVIA Ltd and IQVIA Technology Services Ltd are requesting the following patient cohort from NHS Digital; Cohort: Patients with PH who have been managed at the SPDVU since 2012, which will allow IQVIA Ltd and IQVIA Technology Services Ltd. to confirm the patient diagnosis (and subtype) in HES data and understand the diagnostic pathway before and at the SPDVU along with the post-diagnosis pathway. It is estimated that this cohort will be around 3000 patients. NHS numbers and dates of birth will be provided by STHFT to NHS Digital with the defined cohort of patients for linking to HES data.
Due to the complicated disease area and goals of the research the patient pathway analysis requires a long period of data for the following reasons:
• Having sufficient time to understand patient activity from onset of symptoms to diagnosis
IQVIA Ltd and IQVIA Technology Services Ltd. are requesting ~12-year historical extract of data for the study to cover the requested cohort (patients with PH who have been managed at the SPDVU since 2012).
The reason for the extended time period of the extract is that PH patient populations (especially at subtype level) are very small and the diagnosis pathway is a long multi-year processes and often complex. By analysing a longer period of data, it is more likely that IQVIA Ltd and IQVIA Technology Services Ltd will be able to create cohorts of patients with a higher number which will contribute to a more robust analysis and avoid some of the predicaments and biases of small patient number.
In Europe, PAH prevalence and incidence are in the range of 15–60 subjects per million population and 5–10 cases per million per year, respectively. One of the most common forms, idiopathic PAH, has an annual incidence of 1-2 cases per million. The other subtypes of PH are equally rare, with CTEPH having estimated full incidence of 3-5 cases per 100,000, and SAPH having an estimated incidence of 10-35 cases per 100,000. Because these datasets have relatively small numbers of patients with a rare disease, this request is for a ~10-year historical extract to ensure that a sufficient number of patients in order to ensure the results show statistical significance.
Expected output
IQVIA Ltd and IQVIA Technology Services Ltd will conduct initial analysis of the clinical pathway leading up to diagnosis and the post-diagnostic treatment patterns of patients, including a comparative outcomes analysis of PH treatments that will aim to investigate different outcomes of interest such as, but not limited to, overall survival of the treated groups, liver toxicity, oedema/fluid retention, as well as the reason and number of hospitalisations. In addition, the study will explore both the HCRU and the costs associated with the diagnosis.
An amendment of the Data Sharing Agreement will be required in the future to support any supplementary questions arising from publications, in addition to supporting further publications to contribute to the body of knowledge on PH.
In addition to the publication, additional dissemination plans are as follows:
• Published results will be shared with the Pulmonary Hypertension Association UKPHA UK (PAH UK) patient advocacy group at the beginning of the year 2021.
• Furthermore, the abstracts and links to publications will be hosted on IQVIA Ltd's online bibliography that is publicly available. Results will be presented at the European Society of Cardiology in August 2021, and in other journals such as the International Journal of Cardiology, the European Journal of Health Economics and potential others.
• Results will also be shared with other parties where appropriate. For example, sharing results with other NHS trusts that also manage PH patients or sharing with international centres that also diagnose and manage PH patients
Note: All outputs will contain only data that is aggregated with small numbers suppressed in line with the HES Analysis Guide.
Benefits reported
Yielded Benefits is not a requirement for new applications.
Register history
When this agreement appeared in, or was edited in, each monthly edition of the register. Built by comparing every edition this site holds, the earliest of which is July 2021.
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July 2021 —
already listed in the earliest edition this site holds, so it may be older. 2 versions: DARS-NIC-296034-T4Y4K-v0.16, DARS-NIC-296034-T4Y4K-v1.1
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December 2021
1 version added: DARS-NIC-296034-T4Y4K-v2.4
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December 2022
Register-wide edit DARS-NIC-296034-T4Y4K-v0.16, DARS-NIC-296034-T4Y4K-v1.1 — Datasets: legal basis: “
s261(1) and” taken out. Made to 639 agreements in this edition, so it is reported once, on the changes page, and not counted as an amendment of this agreement. -
September 2023
1 version added: DARS-NIC-296034-T4Y4K-v3.6
Cite this page
NHS England (2026) Data Uses Register, September 2026 edition, agreement DARS-NIC-296034-T4Y4K, “Pulmonary Hypertension (PH) population characterisation and epidemiological analysis”. Read via NHS Data Access Explorer (unofficial), https://healthdatauses.uk/agreements/dars-nic-296034-t4y4k/ (accessed [date]).
This address stays the same, but the page is rebuilt with each monthly edition, so the citation names the edition it shows. Every edition's data is kept in the facts store.
Source: datausesregister_september2026.xlsx, September 2026 edition of the NHS England Data Uses Register. Search that workbook for DARS-NIC-296034-T4Y4K to see the original rows.