Dr Rick Thompson
Patient organisations are formed by, and for, people whose lives are directly affected by a specific condition. However, with rare diseases, progress may be more challenging.
Change happens when those affected by a common challenge come together to make a difference. Shared experience unites people, driving them to solve complex problems. But, when it is hard to find another person affected by your rare disease, how do you change your future?
Challenges in rare disease patient groups
Patient organisations are the only real answer — groups that aim to connect people with shared experience and to drive change. Of course, rarity makes it a huge challenge to start these groups. Finding a person with the time, knowledge and energy required is the first hurdle.
From there, a whole range of complex, competing demands fall upon them: How do they help those affected? How do they change NHS services? How do they ensure a new drug reaches patients? And, of course, how do they get the work done? Rare disease patient groups are fundamentally resource-poor. Most are voluntary, running on the spare time of a few committed people and whatever donations and small grants they can find.
Rarity makes it a huge challenge to start these groups.
Support that patient groups need
Despite the lack of time, knowledge and money, they completely transform their rare condition — driving the development of new care pathways, better diagnoses and even new treatments. What patient groups need is more resources to build their operation and knowledge base. They need staff to collect data and to represent the views of their community.
Right now, pharma is one of the few sources of funding for this work. Although, as highlighted in a recent Observer article, such funding can raise concerns about independence. I feel that such complexities are, generally, well-managed by industry and patient groups alike. However, there is a need to fundamentally rethink the resourcing of rare disease patient organisations.
Solutions to help patient organisations
Either we invest in such patient organisations through the Government or pool pharma funding to be distributed,independent of drug development interest. Both approaches would give small, rare disease patient groups access to long-term, sustainable funding — independent of industry interest. Such a funding change needs coordinated action across the sector. We hope that you can help us drive this change.