Brexit: what does it mean for rare disease patients
The United Kingdom (UK) has voted to leave the European Union (EU) and is poised to begin the proceedings to invoke Article 50 of the 2007 Lisbon Treaty, which would set in motion negotiations on how to leave the bloc.
Although little is known at this stage about exactly what the exit of the United Kingdom would entail, many have expressed concern that the collaboration and cooperation which rare disease stakeholders have struggled for and achieved over the years, could be jeopardised,. Rare disease patient organisations have released statements attempting to allay concerns, as well as asking for solidarity in this moment of uncertainty. Genetic Alliance has released a statement expressing disappointment, but also asserted that they “will be a strong and focused presence, speaking truth to power in government, in parliament, in research, health care and in society so the voice of individuals and families is heard in the settlement of the outcomes of this referendum." A letter from the President of the European Association for Haemophilia and Allied Disorders (EAHAD) and the European Haemophilia Consortium (EHC) states that they will "remain committed to adequately representing patients in the EU and UK, improving patients’ lives, and furthering scientific and medical research.”
Patient organisations from countries neighbouring the UK, have also weighed in as it greatly impacts their situation as well. DEBRA Ireland has expressed deep concern over the referendum’s impact on European Reference Networks (ERN), which aim to bring together the best clinical specialists and to improve care for rare diseases. They believe that this decision may mean that the “UK rare disease patients may not now have easy access to clinical expertise in other EU countries and also for Irish patients, for whom UK centres of expertise are often the first port of call.” Other than delayed access for UK patients and possible loss of funding for UK research bodies, they have said that “the implications go far beyond British research groups and will be damaging to the many rare disease research networks that are reliant on valuable UK partners.”
EURORDIS has confirmed that they will continue to actively work with Rare Disease UK, Genetic Alliance and underscore the importance of “mov(ing) forward to analyse the impact and to start a dialogue to deal with the fallout so that people living with a rare disease in the UK do not suffer the consequences of Brexit.” EURORDIS also recognises that whether initiatives such as the ERNs and cross-border healthcare may be affected in the future, especially after 2017 when the “first successful ERNs are due to come to life”, remains unknown. They also indicate the possibility of the European Medicines Agency moving to another European country. The EMA has released a statement acknowledging that they are in unfamiliar territory and expressed openness to move as well. They have also added that the “European Regulatory Network as a whole is a very strong and flexible system that is able to adapt to changes without jeopardising the quality and effectiveness of its work.”
The hardest hit area could be rare disease research in the UK. Although a minority of scientists are sanguine about Brexit, for many the future of international collaborations and the freedom of students and researchers to move across the channel once the United Kingdom ceases to be an EU Member State are key worries. This could affect young European researchers, who are largely funded on ’soft money’, which will in-turn affect rare disease research. However, most acknowledge that nothing will change in the immediate future for EU funded teams. The RD-Connect team at the University of Newcastle has released a statement expressing that although they feel passionately about the vote, “the outcome of the British EU referendum will not affect (their) accomplishments and it goes without saying that we will continue to work together across borders.” UK researchers attracted 17% (around €570 million) of the EU's Seventh Framework Programme's (2007-2013) entire budget. It is possible that the European funds for the UK, such as funds from Horizon 2020 could be curtailed to some extent, even if it becomes an associate member. Complex negotiations between the EU and UK will ultimately determine the full impact on UK research.
RD-ACTION will of course continue its work to promote the implementation of European recommendations in member states for the benefit of rare diseases patients. UK partners are major and committed collaborators in this project. Orphanet activities will continue in the UK. The work lead by the University of Newcastle contributing to the development and establishment of ERNs, and to the promotion of policies for rare diseases, will also continue.
Since the news of the referendum outcome broke, the furore has calmed down a little. The news cycle has turned. Although Brussels is waiting for the UK to begin formal negotiations, many political factors have complicated the present scenario. Rare disease patients and all other stakeholders will have to wait for negotiations between the EU and the UK to begin to determine the full consequence of the referendum.