Rare Disease Clinical Trials Programme

The Rare Disease Clinical Trials Programme has been set up to progress innovations that could change the lives of people living with rare diseases through the clinical development pathway.

LifeArc aims to translate promising research into life-changing innovations that address unmet needs in rare diseases within five years. To help deliver this ambition, the Rare Disease Clinical Trials Programme has been set up to help progress potentially transformative rare disease innovations through clinical development.

The programme will support academic institutions and industry to deliver high-quality clinical data through early-stage clinical trials. These will be primarily focused on proof of efficacy, with consideration of the route to patient impact within five years.

Interventions under investigation can include:

• Therapeutics (including repurposed drugs) and advanced therapies (including gene and cell therapies); or
• Medical technologies or devices; or
• Diagnostics that demonstrate potential to improve decision-making and outcomes.

Proposals should:

• Address an unmet rare disease medical need
• Be underpinned with a strong scientific rationale
• Be well developed with a clear clinical trial plan
• Have consideration of the future route to patient impact within five years
• Have an established manufacturing process for GMP trial supplies (or equivalent for non-therapeutic interventions)
• Include prior or planned patient and public involvement and engagement
• Include objectives to develop/validate biomarkers and enable targeted treatment
• Include a clear plan on the approach to access and recruitment of the rare disease population

Proposals can include:

• Co-funding with other partners
• Trials centred in the UK or abroad