Mary Rose Roberts
Chief Operating Officer, Beacon for Rare Diseases
Rare diseases do not recognise geography. Yet, the infrastructure, funders and healthcare services supporting people affected by them are increasingly regional.
For instance, recent changes to The National Lottery Community Fund’s Reaching Communities grant mean it no longer supports national work. Consequently, patient groups face a growing challenge: supporting communities spread across the country while navigating systems designed for local delivery.
Rare disease patient groups are national by necessity. For most patients, only one organisation exists for their condition, if any at all. They connect people who may otherwise have no one who understands their experience, creating a collective patient perspective that would not exist without them.
we know the value that patient groups bring to the whole community
Essential to the ecosystem
Despite their importance, patient groups are often viewed as an additional layer of support rather than an essential part of the rare disease infrastructure.
Without patient groups, the rare disease ecosystem loses one of the few mechanisms capable of consolidating dispersed knowledge, lived experience and clinical insight. By combining the expertise of patients, clinicians, researchers and policymakers, they build a far richer understanding of a condition, helping shape services, treatment and care around the realities of living with a rare disease.
These groups cannot operate within local structures without leaving people behind. If funding, infrastructure support and healthcare systems prioritise local delivery, there is a growing risk that organisations providing national support will fall between the gaps — along with the patients they serve.
Supporting the future
At Beacon, we know the value that patient groups bring to the whole community. Our pioneering Rare Insights Study is exploring the experiences, challenges and opportunities facing patient groups to better understand what they need to thrive and how they can be strengthened for the future.
Patient groups are a foundation on which the rare disease landscape depends. Future rare disease policy, healthcare planning and funding decisions must ensure these organisations can be sustained nationally. Doing so will help reduce geographical inequalities by ensuring that people affected by rare diseases can access expertise, representation and community wherever they live — not just where local structures are available.
