Sickle Cell Disease (SCD) Unmet Patient Needs, Standards of Care & National Clinically-Established Treatment Costing Project

Sanius Health requires access to NHS England data for the purpose of the following project:

Sickle Cell Disease (SCD) Unmet Patient Needs, Standards of Care & National Clinically-Established Treatment Costing Project

The following is a summary of the aims of the research project provided by Sanius Health:

Sickle Cell Disease (SCD) is a lifelong, inherited condition with high healthcare utilisation and substantial unmet needs. Patients often experience repeated pain episodes, chronic complications, and long-term organ damage, yet there is currently no nationally published, standardised framework for costing SCD care pathways in England.

This project seeks to fill that gap by establishing a national costing index for SCD, using five key NHS datasets (HES APC, OP, CC, ECDS, and NHSBSA prescribing data). The goal is to describe real-world care utilisation and associated costs for SCD patients across urgent and elective settings, stratified by demographic and deprivation indicators. The project will also characterise treatment patterns, particularly relating to high-cost drugs, transfusions, and complications requiring intensive support. These insights will support NHS England, commissioners, and industry stakeholders in understanding variation in SCD care and the economic implications of service redesign or therapy adoption.

Sanius Health is a commercial UK-based health data and technology company that generates real-world evidence to support healthcare decision-making. It partners with patients, clinicians, and life sciences companies to deliver research, patient support programmes, and digital health solutions. Sanius generates revenue primarily by providing real-world data extracts to pharmaceutical companies using its own ethically consented patient database, which includes prospective data such as app-reported outcomes, wearable data, and 10-year retrospective clinical histories. These data are used to support observational studies, algorithm development, and RWE for scientific publications

Sanius has developed an application (app) that allows patients with SCD to track symptoms and access non-clinical support.

Sanius are not permitted to link NHS England data with any patient data collected via their app or to use NHS England data for any purposes connected with maintaining, evaluating or improving their app.

To supplement this work, a brief standalone survey may be deployed by Sanius to a separate group of SCD patients to understand impacts on families, work, and quality of life, but this will not be linked to NHS data and will be held outside the SDE environment.

This work aligns with Sanius' broader mission to improve understanding, awareness, access, innovation, and outcomes for people living with SCD, a population that has long experienced clinical and systemic inequalities.

The purpose of this project is to develop a fair, nationally applicable costing index to support the NHS and wider system in understanding the burden of SCD. While the company has a broader interest in improving outcomes for people with SCD, this specific project is focused solely on transparent cost benchmarking and is not structured to generate commercial return.

This project has received support from senior NHS clinicians with national expertise in haemoglobinopathies, including consultants at University Hospitals Bristol and Weston NHS Foundation Trust, London North West University Healthcare NHS Trust, and Barts Health NHS Trust.

The Data will be used to generate a national, peer-reviewed publication on the treatment costs and patterns of care for Sickle Cell Disease (SCD) in England. Insights from this publication will support improved understanding of real-world service use, inform policy discussions, and guide the evaluation of new treatment options.

The following NHS England Data will be accessed:

- Hospital Episode Statistics (HES)

o Admitted Patient Care (APC): Required to understand inpatient hospital utilisation and associated care patterns for people living with SCD. Inpatient admissions represent a major cost driver in the condition, particularly due to the frequency and severity of VOCs, infections, and complications such as acute chest syndrome or stroke. This dataset will allow Sanius Health to calculate the number, length, nature, and clinical coding of admissions for SCD and to cost these appropriately using HRG codes and procedure-based detail. It is central to achieving the project aim of producing a validated national reference for care costs in SCD.

o Accident & Emergency (A&E) and Emergency Care Data Set (ECDS): Required to provide an important granular view of emergency attendances, capturing fields such as triage time, care professional seen, and clinical observations. For people with SCD, emergency care is a core element of the disease burden due to the frequency of acute pain crises and complications requiring unscheduled visits. ECDS and HES A&E will allow Sanius Health to characterise and cost the full range of emergency attendances, including attendances that do not result in admission, which may be undercounted in inpatient-only datasets. It also allows identification of repeat attendances and timings, which are relevant for pathway design and cost variation modelling.

o Critical Care (CC): SCD patients can require escalation to high-dependency or intensive care units for serious complications such as sepsis, acute chest syndrome, or multi-organ failure. These episodes incur disproportionately high costs, and as such, HES CC will provide structured data on these types of admission, including organ support types and duration. Including this dataset is necessary to capture and cost these high-acuity, high-cost events accurately, which would otherwise be underrepresented in APC alone.

o Outpatients (OP): Outpatient care for SCD varies significantly across the UK and often includes multidisciplinary clinics, routine haematology follow-up, transfusion appointments, and counselling. HES OP will allow Sanius Health to quantify and cost these patterns of care, including visit frequency, specialty involvement, and variation in practice. Understanding outpatient activity is essential to provide a complete economic picture of current service provision, and to map comparisons with treatment models that may reduce emergency visits or admissions.

- Medicines dispensed in Primary Care (NHSBSA data): Required to capture the real-world prescribing of key therapies relevant to SCD care pathways, including hydroxyurea, opioids, antibiotics, folic acid, and iron chelation treatments. It will also allow Sanius Health to calculate costs associated with ongoing primary care prescribing, which is not visible in HES datasets. This is vital for a complete costing model and to understand variation in prescribing behaviour across geographies and populations. It also supports consideration of the broader burden of disease management, including medication usage and primary care engagement.

These data are necessary to understand clinical burden, treatment patterns, disease severity and to quantify direct healthcare costs and model care pathways.

Demographic data within these datasets is necessary to provide accurate cohort identification, demographic stratification and regional analysis.

The level of the Data will be

• Pseudonymised

The Data will be minimised as follows:

• Limited to data between 2015-latest available data

• Limited to the following geographic area: England

The focus population is any individual with SCD (approximately 17,000 in England).

Sanius Health will filter the data in SDE and will only analyse the data for those individuals with SCD. Sanius Health will conduct this filtering because SCD is frequently miscoded or clustered with sickle cell trait in administrative datasets, and the team has condition-specific expertise and published methods to accurately define the cohort without requiring multiple rounds of filtering requests to NHS England.

Sanius Health is the controller as the organisation responsible for ensuring that the Data will only be processed for the purpose described above.

Article 6(1)(f) – processing is necessary for the purposes of the legitimate interests pursued by the controller or by a third party.

Sanius Health has determined that the processing is necessary for its legitimate interests in supporting healthcare improvement through the development of tools, research, and analytics that enhance understanding of patient outcomes, disease progression, and treatment effectiveness, particularly in under-served or rare disease populations.

Article 9(2)(j) – processing is necessary for scientific research purposes in accordance with Article 89(1), based on UK law, and subject to safeguards that protect the rights and freedoms of data subjects.

This processing is in the public interest as it enables the generation of robust, real-world evidence to address long-standing gaps in the understanding, treatment, and economic burden of SCD. The programme aligns with the UK Policy Framework for Health and Social Care Research and aims to improve outcomes for underserved patient populations, particularly Black and ethnic minority communities, by informing equitable commissioning, earlier diagnosis, and more effective service design.

Insights generated will be shared through peer-reviewed publications and used to support national policy and pathway reform in SCD. The project supports system-level improvements while protecting individual rights.

The funding is provided by Vertex Pharmaceuticals. The funding is specifically for the project described.

Data will be accessed by substantive employees of Sanius Health.

Patient groups will be actively involved in the project – including the Sickle Cell Society, Sicklekan, Cianna’s Smile, Sickle Cell Care Manchester, OSCAR Birmingham, My Friend Jen, Africa Sickle Aid – as well as a diverse group of patients from multiple treatment sites and geographic regions, to ensure that the findings are comprehensive and reflective of the real-world experiences of individuals living with SCD. Sanius’ existing ecosystem and patient community will enable the project to engage with a wide network of patients, carers, and community members to gather valuable insights into the personal and economic impacts of SCD.

These patient groups will participate in ensuring an inclusive approach ensures that the patient perspective remains integral to the project's outputs. By involving patient groups throughout the project, we aim to highlight the lived experiences and unmet needs of the SCD community, thereby supporting the adoption of new treatments and improving overall care pathways. The commitment to patient engagement also underscores the project's focus on enhancing patient access to innovative therapies and ensuring that regulatory and healthcare decisions are informed by robust, patient-centred evidence.

This project is funded through a non-commissioned, hands-off grant provided by Vertex Pharmaceuticals to support the development of an open-access, non-commercial SCD Costing Index. The aim is to create a standardised national evidence base for real-world SCD treatment costs that can be used by all stakeholders, including the NHS, NICE, and the wider industry, to improve service commissioning, technology appraisal, and regulatory submissions.

Vertex has not commissioned the project, will not influence its outputs or dissemination, and does not hold any decision-making role within the study. The funding agreement explicitly states that all programme content and activities are to be solely managed by Sanius Health, with no promotion of any Vertex product or product candidate permitted. There is no exclusive access to data or findings by the funder, and all outputs will be publicly shared and available.

The funding provided is non-contingent and solely supports the costs of analysis required to construct a consistent and transparent reference framework for NHS service utilisation and costing in SCD. No specific product, drug, or therapy—whether owned by Vertex or any other entity—is being assessed or promoted. Sanius Health retains full independence over all analytical approaches, reporting decisions, and dissemination activities.

The scope of the project and all analyses are determined independently by Sanius Health and its clinical advisors. The outputs are intended to benefit the broader SCD community by supporting improved health outcomes and resource planning across the NHS, without preference to any individual therapeutic intervention. These outputs will be made available at no cost, and access will be open and non-exclusive through public channels including peer-reviewed publications, commissioner briefings, and digital dashboards.

This work aligns with Sanius Health’s broader mission to reduce health inequalities and support better NHS commissioning in underserved populations, particularly within rare and chronic disease communities.

NHS England will grant access to the Data via the Secure Data Environment (SDE). The SDE is a secure data and research analysis platform. It allows approved researchers with approved projects access to pseudonymised data and industry-leading analytics tools.

NHS England will provide access to the relevant records from the datasets listed in this agreement to Sanius Health via NHS England Secure Data Environment (SDE). The Data will contain no direct identifying data items. The Data will be pseudonymised and individuals cannot be reidentified through linkage with other data in the possession of the recipient.

SDE users can request exportation of aggregated analysis results (suppressed and summarised according to the NHSE SDE Disclosure Control rules) subject to review and approval by the NHS England SDE Output Checking team. The SDE Output Checking team will ensure that no output contains information which could be used either on its own or in conjunction with other data to breach an individual's privacy.

Users must identify themselves via a multi-factor authentication mechanism and are only able to access the datasets detailed within this DSA. The access and use of the system is fully auditable, and all users must comply with the use of the Data as specified in this DSA.

Users are only authorised to access the Data specified in this DSA and can utilise a variety of analytical tools available within the SDE platform. Users are not permitted to export record-level data from the SDE.

The Data will be stored on servers at NHS England.

The Data will be accessed by authorised personnel via remote access.

The Controller(s) must confirm and provide evidence upon audit by NHS England that access via any remote device complies with the data security obligations within this DSA and the Data Sharing Framework Contract.

For remote access:

- Remote access will only be from secure locations situated within the territory of use (as further restricted elsewhere within the DSA if so done) stated within this DSA;

- Access controls granting users the minimum level of access required are in place;

- Remote access is only via secure connections (e.g., VPNs or secure protocols) to protect data;

- Multifactor authentication (MFA) is required for remote access;

- Device security, including up-to-date software and operating systems, antivirus software, and enabled firewalls are utilised for the remote access;

- All remote access is undertaken within the scope of the organisation’s DSPT (or other security arrangements as per this DSA) and complies with the organisation’s remote access policy.

The above applies in addition to any condition set out elsewhere within the DSA (e.g. who may carry out processing, and for what purpose).

Remote processing will be from secure locations within the UK. The data will not leave the UK at any time.

Access is restricted to employees of Sanius Health who have authorisation from Sanius’ Data Custodian and Chief Technology Officer, who also serves as the named Data Controller and is responsible for the internal governance of data access within the project.

All personnel accessing the Data have been appropriately trained in data protection and confidentiality.

There will be no requirement and no attempt to reidentify individuals when using the Data.

The project does not involve the use of artificial intelligence (AI) or machine learning methods and use of such is not permitted when processing the Data under this DSA.

The expected outputs of the processing will be:

• A peer-reviewed publication detailing national real-world costs of SCD care in the UK. This will include clinically validated, activity-based cost estimates for A&E attendances, inpatient stays, outpatient appointments, and common treatment interventions. The aim is to support a more transparent and standardised understanding of current care costs for use in policy, commissioning, and economic evaluation.

• A series of summary reports, derived from the main publication, tailored for NHS stakeholders, including commissioners, Integrated Care Boards, and national bodies such as NICE. These will highlight key findings and provide suggested reference points to inform local planning and resource allocation.

• A cost reference pack, made available free of charge, to support future health economic models for SCD. This will consist of structured tables of unit costs (e.g. average cost per admission type or procedure) along with explanatory notes and worked examples to help analysts reuse the data accurately in local or national planning.

• Scientific presentations at major conferences including the British Society for Haematology (BSH), European Hematology Association (EHA), and American Society of Hematology (ASH), to disseminate insights to clinical and research audiences.

• A public report hosted on the Sanius Health website that clearly outlines the project’s aims, methodology, findings, and conclusions, ensuring transparency and accessibility for patients, carers, researchers, and healthcare providers.

The outputs will not contain NHS England Data and will only contain aggregated information with small numbers suppressed as appropriate in line with the relevant disclosure rules for the dataset(s) from which this information was derived.

The outputs will be communicated to relevant recipients through the following dissemination channels:

• Journals (e.g., submission to peer-reviewed open-access health economics or haematology journals)

• Presentations at appropriate conferences including ASH, EHA, and BSH

• Public reports and dashboards hosted on the Sanius Health website

• Direct bilateral engagement with NHS commissioners, NICE, and academic partners

• Briefing documents provided to regulatory and HTA stakeholders

• Public promotion of the research via the Sanius website and social media channels

• Press/media engagement in collaboration with clinical advisors and patient representatives

Outputs are expected to be achieved in mid-late 2025.

Tailored summaries and reports derived from the final publication may also be shared with relevant stakeholders, including:

• Department for Health and Social Care (DHSC)

• The Care Quality Commission (CQC)

• NHS Trusts

• Mental Health Trusts

• Community Provider Trusts

• Commissioning Support Units (CSUs)

• Integrated Care Systems (ICSs)

• Life science industry

The proposed work is intended to address a long-standing gap in national understanding of the true cost burden of SCD on the NHS. The data outputs will be used to:

• Advance understanding of real-world health and care utilisation in SCD across emergency, inpatient, and outpatient settings.

• Inform consistent, equitable, and evidence-based health technology assessments (HTAs) for new SCD therapies submitted to NICE and other regulatory bodies.

• Support more accurate planning of SCD services, helping local systems allocate resources and funding based on real patient need and use patterns.

• Reduce duplication and uncertainty in economic modelling across the NHS and industry by providing a gold standard benchmark cost index, developed independently and available for all stakeholders.

• Contribute to wider national efforts to reduce health inequalities faced by the predominantly Black SCD patient population through better service design and policy decisions underpinned by real-world data.

Patients are expected to benefit from improved access to innovative therapies and better organised NHS services through:

• Faster and more equitable assessment of new treatments by NICE and commissioners, supported by robust cost-effectiveness data.

• More efficient and tailored service commissioning at local level, reducing unplanned emergency care reliance and enabling proactive management.

• Increased clinical awareness and adoption of consistent national care standards, reducing variability in patient experience across different NHS trusts.

By producing a transparent and peer-reviewed costing framework for SCD, Sanius Health will provide NHS stakeholders, regulators, and industry with a single authoritative source of economic evidence. These outputs will be disseminated across national NHS bodies, NICE, and local commissioners to inform policy and funding decisions. The consistent, clinically validated data will remove a key barrier to access for SCD therapies and help improve long-term service planning.

The findings will also be published and shared through scientific conferences and policy-facing materials, increasing uptake and use of the data in decision-making processes.

• Peer-reviewed journal publication alongside presentation of findings, which will take place at relevant scientific meetings including BSH, EHA, ASH, and ASCAT.

• The final costings report and price index will be made publicly available on the Sanius Health website and disseminated through clinical networks and professional bodies.

• Briefing packs will be shared directly with commissioners, NHS England decision-makers, and NICE reviewers.

• A targeted communications plan will include engagement with relevant third sector organisations (e.g., the Sickle Cell Society) to promote awareness and use of the findings across the community.

• Public summaries will also be developed to ensure patients and carers are aware of the value and intended impact of the work on future care access and service models.

Patients

This project aims to address inequitable access to care and innovation in SCD by creating a transparent and clinically validated foundation for economic assessments. It ensures future evaluations of new therapies and services are based on real-world data, helping unlock access to effective treatments and improving care services. By aligning stakeholders around a common cost framework, it addresses unmet needs and puts patient experience at the centre of decision-making.

Clinicians and the NHS

For clinicians, this project will provide a peer-reviewed benchmark for evaluating the true cost of care delivery, aiding in clinical service design and resource allocation. It will highlight areas of concentrated demand and patient groups at risk, supporting advocacy for new service models and funding. For the NHS, it will create a national reference index for SCD care, enabling consistent decision-making and reducing the burden of bespoke cost models, streamlining engagement with regulatory bodies.

Industry

For life sciences and therapeutic developers, this project will offer a publicly available benchmark for health economic modelling in SCD. Developed independently and validated by clinical leaders, it will provide a neutral reference point for submissions to NICE and commissioners, reducing delays and inconsistencies. It will offer strategic insights into real-world SCD care patterns and costs, refining trial designs, pricing strategies, and access programmes, ultimately facilitating faster and more equitable market access for effective therapies.

Yielded Benefits is not a requirement for new applications.