Collaboration across Europe in the rare and genetic disease community is fundamental to bringing change. By their very nature it is likely that for rare conditions there may be too few patients within any one Member State to advance treatment and research alone. Collecting and analysing large amounts of patient data from across Europe and the world makes it possible to make meaningful progress in understanding a condition and the effectiveness of new treatments. For example, as a world leader in genome sequencing research, the UK benefits greatly from being able to share information internationally with the Netherlands (our major peer in Europe in this field). It is not possible to validate a genetic sequence for a suspected impact on health without further examples of its occurrence.
The UK’s decision to exit the European Union has the potential to severely impact the research and technology community throughout the country. Recent inquiry into the potential ramifications of Brexit by the Science and Technology committee has highlighted some potential restrictions that a leaving the EU may impart:
Future Brexit negotiations must pay careful consideration to maintaining the UK as an attractive research centre and must act to protect the privileges that existing as an EU member state had afforded us.
For rare diseases, being able to participate in European wide projects is vital. As individual nations are likely to have small subject populations of people with a specific rare disease it is necessary that there is collaboration of institutions across borders, so that clinical trials for rare disease treatments can achieve an ample sample size and the results of the trial can have significance.
The impact of the Brexit vote risks dismantling confidence in the UK as potential research partner, which will diminish the willingness of EU nations to collaborate with the UK. If this is to happen, fewer clinical trials for rare disease treatments are likely to occur in the UK, and as clinical trials are a means by which patients can gain accelerated access to new and innovative treatments, the leave vote risks seriously affecting those with rare disease.
At present the potential effects of Brexit are broad and undetermined prior to instigation of comprehensive leave negotiations. Thus, there is an important need to examine how existing as an EU member state has acted to benefit and hinder the rare disease community in the UK, to ensure that post-Brexit rare disease patients and their families are supported and are not disproportionately affected following the exit of the EU.
We'll be working to make sure that as the negotiations move forward, the voice of the rare, genetic and undiagnosed community is heard. We'll be bringing the voice of patients, researchers and scientists to Parliament, and investigating the potential impact on our community through the All Party Parliamentary Group on Rare, Genetic and Uniagnosed Conditions.