Funding: Translational Rare Disease Centres

Through the Translational Rare Disease Centres we aim to strengthen the UK’s rare disease translational capabilities by:

• Increasing the volume of rare disease research delivered and catalysing translation of rare disease research
• Supporting early career researchers to establish their careers in rare disease translational research
• Increasing the understanding of the needs of rare disease patients with industry and policy makers
• Becoming a central point for engagement with the patient community

What are some of the main challenges?

For information on some of the identified challenges, please refer to the UK Rare Diseases Framework – which sets out a coherent, national vision on how the UK will improve the lives of those living with rare diseases.

How do we expect Centre bids to tackle these challenges?

We expect Centre bids to bring together critical mass to address a major challenge area and be designed around an identified, tractable opportunity in an area of rare disease translational research. Bids should be collaborative and include the diversity of skills required to bring about real change in an area of challenge. In every bid we expect to see:

• Inclusion of appropriate specialists across career stages, including early career researchers.
• Demonstration of harmonisation with existing resource/ capabilities in the area. We expect Centre applicants to have thoroughly scoped their area of focus and connected with appropriate key players. We will not fund duplicative activities and are looking for bids that either create new or add value to existing infrastructure/ resources. Between EoI and Full application stage we may work with applicants to refine applications and broker relationships between those proposing complementary activities.
• A well thought through and appropriately costed Patient and Public Involvement and Engagement strategy (PPIE). We will be including individuals with lived experience on our Funding Panel, and we wish to see a clearly articulated plan.
• Demonstration of feasibility, where appropriate, via engagement with regulators, ethicists and policy makers and other relevant stakeholders.
• Clear demonstration of unmet need and realistic prospect of success and impact.
• Sustainability: with this funding we are aiming to kickstart Centres that will go on to become established in the ecosystem. Applicants should demonstrate that they have given thought to the longevity of the Centre and how this will be achieved.
• Training: training more skilled, translational professionals has been identified as an opportunity for growth in the area. This will be a focus for the overarching Hub, but we would also like to see Centre bids contain an element of training and capacity building. We are happy for budget to be assigned to these activities.
• Governance: the overarching Hub will provide a co-ordinating resource, ensuring synergy, shared learnings and cohesiveness between Centres and the wider community. Each Centre will need a defined governance structure, to ensure the smooth running of day-to day activities, to co-ordinate for reporting requirements, to ensure that timelines and budgets are adhered to etc. LifeArc are happy to support the time of skilled translational project managers, and other appropriate staff, as part of Centre bids.

Areas of focus

We want the community to tell us about areas of unmet need that matter to them and, as such, have not planned to be prescriptive in narrowing focus.

From the scoping we have done to date, however, we think it is likely that we will see Centre bids focussed on either a particular modality, cluster of diseases that share common biological pathways, or development of enabling platform technologies/ methods (which may include clinical trial design, medicinal chemistry expertise, methods of diagnosis etc).

What is not in scope?

• We are not looking to fund basic, underpinning research through these Centres. The UK has a rich, rare disease research base and we are looking to build on this and facilitate translation with this funding.
• We are not looking for Centre bids that consist entirely of individual, discrete development projects. Therapeutic/ diagnostic development programmes are eligible as part of a Centre bid but we expect these to be synergistic, and part of a broader overall aim.
• While items of capital equipment may be eligible as part of a bid, if they are critical to the overarching aim and robustly justified, we are not looking to support bids consisting mainly of equipment/ physical infrastructure building.
• We are looking to focus our funding in areas that will have the biggest impact in rare disease translation. We expect Centres to make a transformational difference by adding considerable value to the current ecosystem. As such we are not looking to commit funding to already well-resourced activities.
• We welcome industrial partners as part of an academic-led consortium (where our T&Cs are met – please contact us via the form below to discuss terms if you have a commercial partner).

LifeArc is seeking to commit £40 million in total in this call, for an envisaged 4-5 Centres and the coordinating Hub (to be commissioned separately).

We fund direct costs (speak to your university financial administrator for further detail on direct costs).

Bids will be assessed for value for money and justification for all costs should be provided at the full application stage. Obviously over-or under-resourced bids will not be progressed through triage.

The opportunity is open to consortia led by an academic institution based in the UK.

SMEs and large industrial organisations may be included in consortia as co-investigators but may not request funding.

Proposals should be collaborative with at least two organisations involved. We are looking for wide reach into the rare disease translational community so please consider this when forming consortia.

Applicants will be required to demonstrate that the assembled team has the necessary experience, expertise and access to facilities to deliver the proposed Centre, or has a clear plan to obtain / build them.

One UK academic institution must act as the Lead Applicant and should have demonstrable experience in leading multi-investigator and multidisciplinary consortia or, at a minimum, should demonstrate their potential to lead and manage a large-scale collaborative project.

Each Centre bid should contain clearly articulated aims and measurable deliverables, progress against which will be assessed at regular intervals throughout the programme of work, and at the end of the five-year funding period. Any individual translational research programmes (see note in “What we are looking for” section above, under “What is not in scope”) that sit within the Centre should have a milestoned plan with SMART milestones.

Centres will be co-ordinated via a central Hub function, but we expect there to be governance structure in place for individual Centres. Centres should ensure that they are resourced appropriately to be able to fulfil timely reporting requirements, keep their spend on track and ensure that milestones are met.

Sustainability is mentioned in an earlier section. As explained, with this funding we are aiming to kickstart Centres that will go on to become established in the ecosystem. As such, applicants should demonstrate that they have given thought to the longevity of the Centre and how this will be achieved.