Our Work

The benefits of EU membership for our community

Last Reviewed 23/06/2016

Collaboration across borders

Collaboration in the rare and genetic disease community is fundamental to bringing change. While there are some 3.5 million people in the UK who will be affected by a rare disease at some point in their life, this number is made up of over 6000 individual conditions. Collaboration comes both from all these rare conditions uniting and from working together on a wider international scale.

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 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, we benefit 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.

European Reference Networks

The EU directive on cross-border healthcare provides incentives to Member States to develop European Reference Networks (ERN). ERNs provide a vehicle for Europe-wide collaboration, seeking to identify already established centres of expertise and to encourage voluntary participation with other centres of expertise across Europe. ERNs for rare diseases will serve as research and knowledge centres, updating and contributing to the latest scientific findings, treating patients from other Member States and ensuring the availability of subsequent treatment facilities where necessary.


Leaving the European Union may mean leaving behind the benefits that we gain from being part of the EU regulatory systems, and will mean forfeiting our influence over these systems. The UK currently benefits from regulation such as that on data sharing, clinical trials and medicines.

The regulation of data sharing in the EU means that UK based researchers can share data, including that pertaining to individual’s genetic material, within safe, secure and socially acceptable boundaries. Being a member of the EU also makes the UK a much more attractive place to host clinical trials. This is because the EU Clinical Trial Regulation harmonises and reduces the regulatory burden restricting the scope to deliver low volume international multi-centre clinical trials.

A good regulatory system means that clinical trials can produce effective treatments for patients of rare and genetic conditions in the UK. Being part of the EU means that the UK can attract clinical trials to take place here, often acting as an important source of treatments for those living in the UK. Recent examples of this include patients gaining access to the newest innovations in therapies for muscular dystrophy and metabolic disease.


Access to medicines is an issue high up the agenda for the rare and genetic disease community. The EU is an extremely important vehicle for incentivising the development of drugs, and for the UK to be either first or second (after the USA) on the list of markets that an innovator would seek to launch their products in.

The European Union’s regulation of medicines in Europe, overseen by the European Medicines Agency (EMA – based in London), creates the largest single regulatory environment for developed nations, with a population of 500 million. The European Union can leverage this critical mass to provide incentives for the development of orphan medicines and for advanced therapy medicinal products. It is important that we can influence the decisions made by the EMA (unlike Norway and Iceland) from inside the EU.

The orphan medicinal product regulation provides incentives as well as support for the development of treatments with indications with a prevalence of less than 1 in 2,000. To date this has supported the development of 114 treatments for patients affected by rare diseases.

The UK’s membership of the EU also benefits patients looking for other treatments outside medicines. For example our membership of the EU Tissue and Cells regulatory system increases the potential pool of donors for haemopoietic stem cell transplantation (HSCT). This has a significant benefit for our patient community as HSCT is one of very few effective treatments for genetic conditions. European regulation allows cross-border transfer of cells for clinical use, which is highly beneficial to the search for a matching donor.

The UK Strategy for Rare Diseases

Genetic Alliance UK runs a campaign called Rare Disease UK (RDUK). The RDUK campaign is an important part of our work; this is something that has been directly influence by our membership of the EU.

In 2008 the European Commission (EC) published a communication on rare diseases giving the momentum for RDUK to campaign to get a UK wide strategy for rare conditions and raising the profile of rare diseases to the benefit of the whole community. In 2013 all four nations of the UK published the UK strategy for rare diseases. It is the first time since the establishment of the NHS that patients and families affected by rare conditions have a clear and strong commitment from Government that their healthcare needs will be met, this is a direct result of the EC communication on the topic.

Effective implementation of the UK Strategy for Rare Diseases will improve the diagnosis and treatment of all patients affected by rare conditions. It will help to ensure that patients who are affected by rare conditions receive the care and treatment they require.

The Strategy includes strong commitments to cross boarder working, through specialist clinical centres, the “UK wants to support the sharing of information, data, knowledge and best practice in treatment nationally, across Europe and further afield”. In order to deliver this and improve the healthcare that patients receive, the strategy notes that “centres should have connections to others across the UK and in Europe”.


Some may argue that the points raised so far in this response would be, to some degree, possible without membership of the EU. We believe it would be to a small degree, but must concede this point to an extent.

However, we have been a member of the European Union for 42 years. It is not just the UK that benefits from our membership of the EU. The UK has had an enormous impact upon the EU’s outlook with respect to science, innovation and research.

The UK’s third sector’s influence on the Clinical Trial Regulation and the Data Protection Regulation (currently ongoing) has been seminal and powerful. Without our voices, the EU would undoubtedly be a worse place to do research, and cross border collaboration would certainly be much more difficult. The Medicines and Healthcare Regulatory Authority (MHRA) is a key opinion leader at the EMA and has played a significant role in creating the regulatory environment that we have in the EU today.

Leaving the EU would almost completely end our influence over policy development in the EU. The large consortium of countries on our doorstep with harmonised approaches to research and medicine regulation would begin to change significantly. The UK has a world leading approach to the regulation of innovative approaches in research and healthcare; this voice is crucial in the balance of attitudes on our continent.

An EU without the UK would eventually become an EU with which collaboration would have little value. Consequently, the argument that the UK can continue to benefit from the EU from outside will, at least in a health research and medical context, be shown to have been incorrect. The EU and the UK would both be diminished by a vote from the UK to leave the European Union.

On Thursday 23 June 2016 the population of the UK voted to leave the European Union. We continue to advocate for the best possible outcome for patients with rare, genetic and undiagnosed conditions, to see our most recent work on this issue please click here

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