Validation of LifeArc’s inaugural Health Data Strategy: A robust and evidence-based process for strategy appraisal

In February 2026, LifeArc launched its inaugural Health Data Strategy to address the systemic barriers currently challenging innovation in the rare disease sector. Currently, an estimated 300 million people worldwide live with a rare disease, most of whom currently have little hope for effective treatment.

The development of health data assets in the UK is, understandably, driven by the needs of high-prevalence, high-cost conditions, where the greatest population-level impact and returns are expected. This focus means that the infrastructure, analytical tools, and data strategies are typically designed to serve common conditions, with less attention given to rare diseases. As a result, data assets and insights that support the identification and management of rare diseases are often underdeveloped and underutilised. Without deliberate efforts to design, govern, develop, and deploy these capabilities with rare diseases in mind, they risk remaining deprioritised. Specifically:

• Multi-modal data is essential – combining genomics, imaging, biomarkers, and detailed clinical history.
• Pathways frequently involve specialist or tertiary care, family history, and cross-specialty input requiring linkable data from multiple sources.
• Diagnosis often depends on subtle phenotypes, non-standard testing, and longitudinal tracking making depth and breadth of data imperative.

LifeArc has a clear and important role to play in shifting the trajectory in favour of rare diseases. With its unique perspective and positioning, LifeArc is well placed to identify where the design and evolution of health data assets may unintentionally overlook, or underinvest in, capabilities that are critical for rare disease identification, research, and care. Its position allows it to:

• Influence the design, priorities, and application of national datasets so they are better optimised for rare disease. For example, ensuring that rare disease can be and are correctly coded within national datasets.
• Support the development of analytical tools, ontologies, and models that are suited to low-prevalence, high-complexity conditions – where small samples, heterogeneous pathways, and noisy outcomes are standard.
• Work with policy stakeholders to ensure that data is not just available, but usable in the context of NICE, NHS commissioning, and regulatory decisions – supporting more informed judgement around uncertainty, long-term value, and equity of access.
• Strengthen and connect specialist rare disease datasets, improving coding practices, to support trial recruitment, and provide the evidence to make rare diseases more visible in the system.

Better data won’t solve everything alone, but it is one of the few levers that cuts across the commercial, clinical, and regulatory challenges in rare diseases. With the right design and investment, the UK could position itself as a leader in rare disease research and care.

Our robust and evidence-based process for strategy appraisal

Edge Health were commissioned, in partnership with Newmarket Strategy, to validate LifeArc’s inaugural Health Data Strategy. Our support included leading a health economic appraisal of key initiatives to support decision making and refinement of the strategy.

Core activities included:

• The development of a robust rubric for refining a long list of options into a shortlist, including key decision making metrics such as patient impact, time to benefit realisation, access to high-quality data to generate high quality outputs, external perceptions risk etc.
• The facilitation of interviews with internal and wider stakeholders to understand potential scenarios, from “do min” to “do max”, across core initiatives.
• The full health economic appraisal, including cost-benefit analysis, on shortlisted initiatives.
• The facilitation of three workshops with LifeArc stakeholders to validate findings and test assumptions.

Alongside our health economic support, our team also provided support with the drafting and refinement of the strategy.

“LifeArc is a self-funded medical research organisation focussed on transforming the lives of people living with rare diseases and drug-resistant infections. Our Health Data Strategy is a critical enabler of translation in these areas of high unmet need, where we can deliver significant positive change for patients. The support from Edge Health brought expertise and robust evidence to shape and validate our strategy to be targeted, partnership-led, and impact focussed.”
Rebecca Cosgriff
Scientific Director, Data Partnerships & Portfolio Strategy at LifeArc

The impact

Our robust appraisal enabled the strategy to recognise where targeted intervention by LifeArc can deliver the greatest value and set out where to invest accordingly. Specifically:

• Where early data is often neglected, particularly in paediatric rare diseases, the KidsRare initiative in collaboration with Great Ormond Street Hospital (GOSH), is improving how critical information is connected across specialist services. In rare diseases, where every data point counts, this strengthens the foundations for research, trials, and adoption.
• To support vital diagnosis and stratification, LifeArc is working to enable secure access to high-quality, multi-modal data to support the development of AIaMD/SaMD. Using rare dementias as the first focus area, the aim is to make faster and more accurate diagnoses by connecting previously unconnected data assets.
• Recognising the high cost and failure risk of trial recruitment, LifeArc is working alongside national infrastructure to build a proof-of-concept scalable model for identifying and enrolling rare disease patients more efficiently. This work is critical to making trials more viable and reducing delays caused by under-recruitment.
• In 2024, LifeArc made a strategic investment in health data as a new opportunity to deliver patient impact. To help harness the full potential of these investments, LifeArc’s in-house data science team is well placed to offer hands-on expertise to projects and partners across the portfolio, sharing what is learned in one part of the network so that the whole can benefit. The goal is to build and nurture strong partnerships that unite fragmented systems, break down barriers and advance scientific discoveries.

These initiatives are not standalone. Together, they form a coherent strategy to reduce friction across the rare disease translation pathway – ensuring that the UK’s health data environment becomes more inclusive, more usable, and ultimately more capable of supporting rare disease innovation.

Read the full strategy here.