Dr Jayne Spink, Chief Executive of Genetic Alliance UK 

According to the old adage, you never know what you have until it’s gone. This is certainly true of health and our NHS. As a result of Brexit, we are already suffering substantial losses and worse may be yet to come.  

If our predictions become reality, healthcare and research for rare diseases will be an early casualty of Brexit. Members of the rare disease community are already beginning to feel the impacts and to understand the imperative for protecting research and care. There are over 6,000 rare diseases affecting an estimated 3.5 million people in the UK; that’s 1 in 17 of us. If you don’t personally have a rare disease, then remember that your loved one, family member, friend, colleague, your future child or grandchild or your neighbour does. Everyone needs to care about this issue because it does, or will in the future, affect every single one of us.

Many rare conditions are severe and life-changing. Three quarters of rare diseases affect children. Delays in diagnosis, misdiagnosis and hurdles in accessing expert help and coordinated care are familiar experiences for many patients. Much of the progress in rare disease research and care is owed to the UK’s ability to strategically collaborate with countries across Europe. Brexit threatens every aspect of this collaboration.

We have already lost, as a direct result of Brexit, the UK’s leadership of 6 of the 24 European Reference Networks (ERNs). The ERNs draw on the expertise of doctors and researchers at 900 specialised healthcare units located in more than 300 hospitals across 26 EU and EEA countries.

Our self-expulsion from the EU’s medicines regulatory system, the European Medicines Agency, and the incentives of the centralised process for medicines approvals could be the difference between UK patients being able to access new treatments or not.  Rare disease medicines are trialled on a multi-national basis - there being too few patients in a single country in most instances to make single-nation trials viable. Being part of the EU has cemented the UK’s pole position for the launch of new medicines, being either first or second (after the USA).

The damage done to our ability to access funding for international collaboration with EU27 partners will impact negatively on the volume and quality of research outputs and participation opportunities. For most rare diseases there is no effective treatment. New medicines bring hope and, where proven effective, save and transform lives.

It’s not only access to experimental treatments we stand to lose. Brexit has threatened our medicines supply chain. Medicines to treat complex rare diseases, particularly biological and advanced products, will be amongst the first casualties.

Genetic Alliance UK was steadfastly unequivocal before the referendum that a vote for Brexit would damage our NHS and the prospects for the 3.5 million UK citizens affected by rare diseases and their families. Now, two years on, we see little cause for optimism as the various Brexit scenarios are being played out and we hurtle towards 29th March 2019.

In the debates and discussions about Brexit and health please remember the rare disease community. We believe there is still time to make a difference and hope that you will show your support for the rare disease community by signing up to our campaign to protect European Reference Networks.

Brexit is bad for the rare disease community, and bad for the UK's health. This is why we need a People's Vote.