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.
About the programme
This programme is an exciting opportunity for researchers working on any rare disease to apply for funding, alongside advice and support from our internal teams, to progress their innovations through clinical trials.
We are looking to fund early-stage clinical trials for rare diseases that will deliver high-quality efficacy and safety data, with a view to driving patient impact within the next 3–5 years.
If you believe you have a suitable project, early engagement is encouraged. You can contact us via the form below to learn more and enquire about submitting an expression of interest.
Funding availability and process
The programme is now open and accepting proposals for consideration. Proposals will be assessed and progressed on a case-by-case basis.
How long the programme stays open will depend on demand. Early engagement highly recommended.
Successful proposals may be awarded up to a maximum of £5 million each.
Overview
LifeArc aims to translate promising research into life-changing innovations that address unmet needs in rare diseases within 5 years. To help deliver this ambition, our 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 5 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.
Eligibility
Applications are open to not-for-profit institutions (including academic institutes, healthcare organisations, public sector research establishments etc) and SMEs, either in the UK or abroad.
Scope
What is in scope?
We welcome well-developed clinical-stage projects, particularly those that are ready to progress to the next stage of clinical development within the next 12 months. Those which align with one of our Translational Challenges (rare neurodegenerative, rare respiratory, childhood cancer), are especially encouraged.
Proposals should:
- adress 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 5 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.
What is not in scope?
Proposals that focus solely on non-clinical development or manufacture will not be considered.
Proposals focused on running clinical studies and not clinical trials, such as clinical studies focused only on biomarker discovery / validation or projects focused on natural history studies or equivalent types of clinical studies, will not be considered.
Proposals that are not sufficiently developed for rapid progression to clinical development (e.g. do not have sufficient non-clinical evidence, manufacturing or clinical trial plans in place) will not be progressed.
Available funding
Applicants can request up to £5 million per proposal. Lead applicants can only submit one application as the lead.
Submitting an expression of interest
Applicants must engage with LifeArc before applying.
If you believe you have a project within scope and would like to learn more, please contact us below to arrange a follow-up meeting.
Contact us
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