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New report by LifeArc Hub’s Policy Working Group: More trials, better data, faster access

We’re delighted to share a new report that Genetic Alliance UK has developed on behalf of the Policy Working Group of the LifeArc Translational Centres for Rare Disease Hub: The new report is ‘More trials, better data, faster access: Opportunities to innovate in translational research for rare conditions.’

The last year has seen many promising developments in UK health policy.

This includes an announcement that the UK’s regulations for clinical trials will change from April 2026, and a bigger focus on the role of artificial intelligence (AI) and data in healthcare by UK decision-makers.

However, if you are a person living with a rare condition in the UK, access to innovative diagnostic tests and treatments continues to be limited for a number of reasons.

One of these reasons is that many researchers into rare conditions report challenges in helping move (translate) promising discoveries in the lab to being made available via the NHS for people who need them most.

To explore this in more detail, the LifeArc Hub’s Policy Working Group has identified three areas of opportunity to ‘get policy right’ and make the biggest difference to those working on research into rare conditions:

  • New types of clinical trial design that are intended to be more flexible, ethical and inclusive to account for the unique challenges seen in research for rare conditions.
  • Artificial intelligence to help make research into rare conditions more efficient and support researchers make better use of small amounts of patient data. 
  • Multi-omics and multimodal data (the linking of different types of data) to help make research findings more precise and insightful. 

Our report brings together the voices of centres leading translational research across the UK. They are delivering exciting progress towards treatments for rare conditions, and maintaining that momentum is one of the most compelling arguments for continuing the UK Rare Disease Framework. The centres' important recommendations to address barriers and grasp opportunities will ensure the centres can meet their potential to address the unmet needs of people living with rare conditions.

Nick Meade, CEO, Genetic Alliance UK

The report also builds on the ten recommendations outlined in the 2025 report by LifeArc and Genetic Alliance UK called ‘Accelerating R&D for rare disease in the UK’. This includes a recommendation that all four nations commit to refreshing the UK Rare Diseases Framework when it expires in early 2027. A renewed Framework would also help us deliver the ambitions set out in the UK Government’s Life Sciences Sector Plan to ensure the UK remains a global leader in innovation.

Through coordinated national efforts and continued partnership, we can ensure that the progress made in research translates into transformative change for people whose lives are impacted by rare conditions. LifeArc is committed to helping realise this future.

Dr Alessandra Gaeta, Head of Rare Disease, LifeArc

LifeArc doesn't want anyone to miss out on innovation because of complexity, cost, or commercial risk. And we firmly believe that we go further, and faster, when we work together.

Sam Barrell, CEO, LifeArc

We hope the report is a useful contribution to ongoing discussions about these topics. Learn more about the LifeArc Hub’s Policy Working Group and how Genetic Alliance UK collaborates with LifeArc.

Genetic Alliance UK is also running a campaign to renew the UK Rare Diseases Framework. Click to read more about our Future for Rare Campaign.

For any questions about the report, please email: [email protected]