Our exit from the European Union will likely be the single most significant political change many of us will witness during our lifetime. We don’t yet know the shape of our future relationship with Europe or how discussions on ‘big ticket items’ such as trade will ultimately play out. But of this we are certain; in formulating a negotiating position on the terms of our exit, it would be all too easy for the interests of rare disease patients to go quietly unrecognised in the maelstrom of activity.
Being invited to give oral evidence to the Health Select Committee was not only a great privilege but also an incredible opportunity. It’s encouraging that the Committee is aware of and actively considering the interests of rare disease patients. For me on a personal level, being broadcast live on Parliament TV was certainly nerve-wracking and I was grateful to be seated alongside Aisling Bernand MBE from the Association of Medical Research Charities and Dr Beth Thompson MBE from Wellcome – both powerful and passionate advocates for medical research.
I see three clear take take-home messages from the evidence session on 12 December.
Firstly, in terms of access to clinical trials and the ability to both influence and benefit from the development of innovative medicines, our membership of the EU has proven enormously beneficial for UK rare disease patients. These factors were at the forefront in our written response to the Health Select Committee Brexit inquiry. Being part of the EU has enabled researchers in the UK to collaborate with researchers in other member states and to apply for European funding to support their work.
Secondly, the regulatory environment for medicines has incentivised and driven forward the development of orphan medicinal products – being part of the EU has enabled UK researchers to run, and UK patients to participate in, multi-state European clinical trials. For the vast majority of rare diseases there will be too few patients within the UK to support a meaningful clinical trial.
Thirdly, the UK boasts world-class clinical expertise and is a recognized leader in rare disease research. It’s important that there is continued provision for UK involvement with and leadership of the European Reference Networks. Unlike some other EU countries, the UK has no specific measures in place to encourage the development of orphan medicinal products. Building clinical services for rare diseases and promoting uptake of innovation in rare disease care and treatment could help the UK remain competitive. Should specific incentives be introduced in a post Brexit UK, it would remain unrealistic to expect rare disease clinical trials to take place here in isolation, regardless of how competitive an environment can be created.
As the rare disease community has long known, our greatest strength is in collaboration – if we lose our ability to work in partnership the impacts will be felt by rare disease patients first and hardest. The interest shown by the Health Select Committee gives hope; we have clear political recognition of the potential impacts of Brexit on rare disease patients and this a step in the right direction.