Although 3.5 million people in the UK live with a rare disease, many experience isolation. This includes isolation from other patients, from healthcare specialists, from specialist centres, and from researchers. GPs and other medical professionals are largely unfamiliar with rare conditions, meaning 30% of rare disease patients have received three or more misdiagnoses, and patients often have to become the expert in their own disease.
Not having sufficient support in place, while going through disease-related pain, stress and anxiety, can be incredibly demanding. Patient groups are an important source of support and advice for patients, and they are often determined to fund and fill the research gap themselves. However, only around half of rare diseases have a specific group established. Those that do exist are often set up by patients and/or their families, meaning they frequently lack the knowledge of the third-sector, healthcare and biochemistry that they need to progress.
Findacure is a UK charity building the rare disease community, to drive research and develop treatments. We aim to empower patient groups, so that they can increase their support capacity and drive treatment research. We run a series of innovative programmes to communicate with patient group representatives and provide them with the knowledge and skills needed to reach their own goals. Together, these projects allow groups to learn from each other and from other experts, which negates the need for each to ‘reinvent the wheel’. Programmes include:
We currently run four training workshops per year, which are free for patients and advocates to attend. Having been organised in partnership with expert speakers and successful case study representatives, these workshops aim to provide patient groups with a range of information and skills. Valuable discussion and networking opportunities are also worked into each session.
Past topics have included effective fundraising, patient group management, developing clinical trials, and engaging with the pharmaceutical industry.
Our first peer mentoring programme, which selected sixteen small patient groups and matched them with rare disease/third-sector experts based on their needs, began in September 2014 and ended in January 2016. We are proud that all patient groups made excellent progress towards their identified goals, and that feedback of the programme was immensely positive.
Participation in the scheme is free. We are currently planning our second round of peer mentoring, which we hope to run from mid-2016 to late-2017.
Our online portal gives patients and advocates access to the tools and resources they need to run their patient communities. Following this link to sign-up, patients and advocates can access free information, connect through moderated forums, and contribute to increasing the collective knowledge of the rare disease community. In 2016, we intend to develop the portal with interactive training modules and videos.
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Drug repurposing takes existing generic drugs licensed for human use, and tries to find other illnesses that they could treat. It is faster and less expensive than normal drug discovery, because we already understand the drug’s behaviour and side effects in humans; we already know about dosing and delivery of the drug; and we understand the drug’s mode of action. It is therefore an excellent option for rare diseases, where there is a lack of research funding and few available treatments.
Following identification of promising repurposing projects by patient groups, clinicians and researchers, Findacure’s own drug repurposing project will fund ten repurposing trials for rare diseases using a social impact bond (SIB) financial model. SIBs are financial tools designed to allow the government to pay third parties, like charities, for providing services to a specific group of people, based on the success of that service.
While the rare disease community is currently quite disparate and divided, with different stakeholders pursuing their own interests, we have experienced for ourselves the great things that can be achieved when individuals and their strengths are put together in a facilitating environment. Combining the motivation of patients and patient groups, the ability of academics and the pharma industry, the responsibility of healthcare professionals, with the skills and experiences of advanced patient groups, gives so much potential for great goals to be achieved. We truly believe collaboration between these stakeholders is necessary for the rare disease community to move forward and confront its most pressing challenges.
Findacure regularly produces an environment that welcomes all stakeholders and facilitates collaboration between them. We host two networking events, a scientific conference, and a student essay competition each year to unite a diverse audience around common goals.
Below are some quotes from patients and patient group representatives about accessing Findacure’s services.