How It Works
There is a mismatch between research ideas that patients prioritise (usually quality of life and symptom management), and those actually investigated in academia or industry (usually drug development or disease modification). We think patients and their families have important research ideas that should be addressed, so we partner with patient organisations to develop patient-driven research that’s important to you and your family.
Between 2015 and 2022, over 70% of our feasible proposals came from rare disease patient groups or charities. These groups are incredibly motivated but usually have minimal funding and limited research experience. Supporting this community requires resources and methods tailored to the challenges of rare disease research (including, for example, few clinical experts, limited baseline data, small numbers of eligible participants, large geographical spread, and the potential need to combine multiple, similar diseases). Rare disease is recognised as under-served in research, so in 2022, we decided to shift focus and exclusively support this patient community.
Who can submit an idea?
We focus on rare! Patients, carers, support groups, patient organisations and charities: if you or a loved one has experience living with a rare disease, we welcome your ideas. Each idea must be supported by a UK-based patient organisation or charity to help with study management and ensure a wide range of patients and families can be involved. Involving a patient network will strengthen the research proposal when applying for funding, and will benefit a study in many ways including improved design and recruitment. The PLRH requires a UK connection, but please do get in touch regardless of your geography: we’re open to international collaborations, and may be able to help you find a local network. We’ll also do our best to help if your research idea is about a common condition or overarching theme that affects the rare disease community.
Although our we cannot directly support proposals from small businesses, community organisations, or individual entrepreneurs, we encourage you to join the Rare Disease Research Network (RDRN). The RDRN is an online platform designed to encourage cross-sector collaboration and match-make members with similar research interests. You’ll be able to access resources and expert mentorship to help you develop your idea and build a research team.
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What ideas can you submit?
Any research idea, on any rare disease topic! Our ethos is to develop any project that is technically feasible.
Where do I start?
You can submit a simple idea or detailed project outline online, via email or through direct conversation. There is no set criteria but you may find our summary worksheet (here) helpful to complete (although it’s not required). We’ll discuss your idea with you to make sure we understand it thoroughly before beginning our feasibility assessment. You’re welcome to contact us anytime for more information about the PLRH or research process.
What to expect?
The first step will be to assess whether your proposal is feasible and practical. For feasible projects, we will work with you and field experts to develop a funding proposal and design the study. You become part of the study team and remain involved throughout the whole process, from initial development through reporting the results. We recognise that your ability to commit may fluctuate, so other members of your patient group can help with this role.
Setting up a clinical research project can take a long time, often years. Studies can require an extensive amount of background work, device development, and collaboration between organisations before data collection even commences. As the study progresses, funding external to the PLRH will be required. Although we endeavour to successfully secure a grant for your project, we can only assist in the application process and cannot guarantee funding will be achieved.
Often research ideas have already been studied or align closely with ongoing clinical trials. In these cases we do our best to connect you with the research group. Sometimes feasibility is uncertain and we need to do extra background work before deciding to progress the idea (literature reviews or prevalence surveys, for example). You’re welcome to help with this – we’ll provide training where possible. We can’t develop research ideas that are infeasible, but you’re welcome to submit another idea at any time. We do our best to keep all our communication open and transparent, and the Cambridge BRC PPI Oversight Group is always available to help.
Once we receive your idea we’ll send you our Terms of Reference document to help explain these steps in more detail. If your idea is feasible, we’ll send you another document: Co-producing Research with the PLRH. We’ll go through these documents together and answer any questions you may have. Throughout your project, we’ll also complete our Patient Involvement Reporting Matrix to make sure your involvement is perfectly tailored to your skills and expectations.
What if I don’t know anything about research?
Don’t worry! We don’t require you to have any knowledge of the research process. We provide the medical and research expertise, and you provide the idea and life experience. Both are equally important to developing clinical trials. Involving people with lived experience from the onset of research greatly improves the quality and credibility of research. You will be able to contribute invaluable knowledge to patient-centred design, communication, and outcomes, and ensure that the recommendations from your study reach all members of your patient organisation.