BREXIT - EURORDIS STANDS UNITED WITH UK RARE DISEASE COMMUNITY
What remains certain however is the mission of EURORDIS to represent the voice of people living with a rare disease in Europe, not only those living in the European Union but in all 48 countries in Europe. We represent over 700 rare disease patient organisations in 63 countries. We continue to stand united with our UK members to work towards improving the lives of rare disease patients and their families in the UK. We are proud of the success of the recent European Conference on Rare Diseases & Orphan Products 2016, which took place in Edinburgh in May.
We will work closely with Rare Disease UK, Genetic Alliance UK and all EURORDIS members in the UK to advocate for UK rare disease patients and their families to continue to benefit from EU regulations and actions, which affect their quality of life and the availability of and access to treatments. At EURORDIS, we do not expect any negative impact on the work we do on behalf of our members and patients in ongoing EU-funded projects, research or joint rare disease actions with Member States.
We should keep in mind that some countries, such as Norway are close to the EU. We need to look at this evolution in the broader context of other non-EU countries strongly associated with the EU.
A European rare disease community
Emotions are high. The shock of the result on Friday 24 June is still felt; it is an attack on the principle of solidarity that binds the rare disease community together. It is not yet possible to determine the effect on our community, and in particular on our friends and colleagues in the UK, but there will be one. We must move 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.
The rare disease community has been thriving for years on exchange of experience and collaboration, surmounting physical and legal borders. The community overcomes small patient population numbers and isolation through joint efforts to find creative solutions that make the most of scarce and scattered resources and expertise. Step by step, the community has worked to convince all relevant civil and political players that working together, across European and international borders, is a win-win situation for all involved. This has taken time and great patience. In the current climate, we must resist the divisions that Brexit is likely to bring, and remain committed to this collaborative way of working.
We have seen milestones, such as the EU Regulation on orphan medicinal products (which has resulted in the availability of 120 orphan medicines on the European market) or more recently the EU Directive on patients’ rights in cross-border healthcare, some of the long term fruits of which are just starting to flourish (for example more than 20 applications for European Reference Networks were submitted just two days before the Brexit vote).
All stakeholders now recognise that rare diseases, because of their very nature, are an area with high European added value, in which the most effective strategies are cross-border and EU-wide.
From 1998 to 2013 we saw a tenfold increase in the European budget for collaborative research on rare diseases. The rare disease research community has been endlessly working across EU borders. The last few days we have heard colleagues recalling how science now transcends borders. Complex discussions on how the UK can participate in future European research and health projects such as Horizon 2020 are now starting to take place. While the UK remains an EU member state for ostensibly 2 years, the details of their participation in such projects as of 2018-2019 will need to be negotiated.
It is obvious that the loss of the UK contribution to the EU budget will have an impact on many programmes. It is difficult to determine the extent of this impact.
Brexit will likely have a direct impact on the participation of, and benefits received by, UK academic partners in such EU-funded research and policy projects. There is also concern for the loss of high level scientific expertise that comes from the UK.
European Reference Networks & Cross-Border Healthcare
While the applications from networks of healthcare providers and laboratories wanting to become European Reference Networks (ERNs) have only just been submitted, concerns that healthcare providers (HCPs) from the UK will be left out of ERNs are starting to appear. ERN clinical leads are required to be based in the EU so this could create a challenge in certain cases. But we will we contribute to finding a solution, for example, that UK HCPs could act as associative partners in ERNs.
The first successful ERNs are due to come to life in early 2017. Until then, and possibly for another year, it will be most likely be business as usual. We will advocate to ensure that the effects of Brexit will not hamper this ground-breaking initiative in its early stages of development.
It is also unknown what rights UK rare disease patients wishing to travel to other EU countries will hold and likewise for EU citizens coming to the UK to receive diagnosis or care. These rights are enshrined in EU legislation including the Cross-Border Healthcare Directive. EURORDIS will cooperate with other patient groups in Brussels to advocate for the preservation of patients’ rights to the greatest extent possible in the negotiations around cross-border healthcare.
The European Medicines Agency & orphan medicines
The European Medicines Agency (EMA) is based in London. EURORDIS has played a pioneering role in ensuring the patient voice is present at the EMA. A decision regarding the possible re-location of the EMA is yet to be taken. However, what is sure is that our work to represent the voice of rare disease patients across Europe, including those in the UK, at the EMA will continue irrespective of the location of the Agency’s offices. That being said, it is possible that the direct voice of UK regulators involved in EMA activity will be diminished.
It is also unsure yet of the effect that Brexit will have on clinical research and the marketing authorisation process for orphan medicines in the UK. The UK may miss out on opportunites brought about by the new Clinical Trials Regulation, and it is also possible that the UK’s Medicines and Healthcare Products Agency (MHRA) could assume responsibility for marketing authorisation of these medicines. This is yet to be determined.
Finally, EURORDIS will also continue to support cooperative initiatives between Member States in the field of Health Technology Assessment and in other areas such pricing and reimbursement of medicinal products. These voluntary initiatives will continue to attract only those countries willing and able to embark on a closer collaboration with peers and as such should not be halted by Brexit.
Our commitment to working towards European collaboration that improves the lives of people living with a rare disease is stronger than ever and our relationship with rare disease patient organisations, academic partners, regulators, policy makers and industry from the UK will not be undermined by Brexit.
Yann Le Cam, Chief Executive Officer, EURORDIS-Rare Diseases Europe