What does the Brexit mean for rare disease care?

The world's eyes turned to the UK this week* as Britons voted in the EU referendum. The major decision for the UK to leave the EU is unprecedented for such a significant member of the union. Many changes will be implemented in the coming months in the areas of trade, immigration, and others, the effects of which are currently unclear. Nonetheless, rare disease patients in the UK and across the EU still require care. How will such a major political shift affect their care?

What's important to remember firstly is that while major changes are coming, patient care is unlikely to be affected immediately. Only until Article 50 of the Treaty of Lisbon - a short section discussing how countries might leave the EU - is invoked, and many months of negotiation thereafter have taken place, will any changes to the rare disease community be made known. The direct effects of the Brexit on medical care may not be seen for up to two years. However, the indirect effects of this move on pharmaceutical industries, clinical trials, and biomedical research will have far-reaching implications in the present and the distant future. While Britain's exit from the EU will free itself from various policies, including the Council Recommendation on rare diseases, the EU also stands to lose opportunities for industry collaborations with the UK's top research facilities and universities.

While the rare disease Council Recommendation advocates for providing comprehensive care to rare disease patients, EU member nations are left to create these policies for themselves. Britain's own Strategy for Rare Diseases outlines how the NHS (the UK's healthcare system) will change "to ensure that patients have access to high-quality care, including diagnostics, treatments, habilitation for those living with the disease and, if possible, effective orphan drugs." This six-year plan, culminating in 2020, is unlikely to be repealed as the EU Council Recommendation was published in 2009 and is non-binding, suggesting the UK's voluntary commitment to the cause. Additionally, the supposed financial freedom associated with the Brexit might also help fund rare disease care. While highly unlikely, some of the 5.7-7.3 billion pounds a year previously paid to EU membership fees may find its way to the NHS rare disease budget or other health related areas.

Unfortunately, one of the few clear effects of the Brexit will be a slower approval process for pharmaceuticals, including orphan drugs. While all countries have their own pharmaceutical regulatory body where drugs can be approved and regulated, EU members are also centrally regulated by the European Medicines Agency (EMA), and an EMA-approved drug can be distributed across all EU nations. After the Brexit, the only way new drugs can be approved for use in the UK will be through its own regulatory body, the Medicines & Healthcare Products Regulatory Agency. It is unclear how much this will restrict orphan drug availability in the UK, but the removal of the EMA approval process will slow down access to orphan drugs if new policies are not implemented. "The pharma industry argues that the UK is involved in about 40% of all adult rare diseases trials in the EU at present," says BBC Health Editor Hugh Pym, "but this would be undermined by a change of status." Clinical trials are likely to be affected in both the EU and the UK due to the Brexit, as the legal logistics will change as Britain no longer follows EU regulations. Both Britain and the EU are likely to suffer as a result, as UK clinical trials are cancelled due to the removal of EU legal frameworks. As such, the Brexit is likely to cause a loss of profits for pharmaceutical companies across the globe, which is also likely to result in fewer medications being actively developed in the next two to five years.

““The removal of the EMA approval process will slow down access to orphan drugs if new policies are not implemented.””

Long-term research into rare diseases is also likely to be affected by the Brexit. Due to the mystery surrounding the long-term effects of the secession, students and researchers alike may be hesitant towards studying or living in the UK. Depending on the Brexit's effect of the economy, research funding may also be decreased, especially for rare diseases, which only cater to a small population. Qualified students across the EU may be hesitant to study in the UK in the long term, especially with the as of yet unknown immigration law to replace EU policies. Additionally, it is likely the euro will fall in the long term due to the loss of such an industrially productive member, making it more difficult for socio-economically disadvantaged EU nationals from pursuing research opportunities in the UK. It is unclear if the pound will rise or fall in the long term, but if it continues to fall, graduate programs will attract brilliant students across the globe, but are unlikely to immigrate to the UK permanently. If the pound rises, UK students are likely to pursue graduate studies in the scientifically-comparable US but are likely to stay due to less competition and more opportunities.

The effects of the Brexit on rare disease drugs and care will be global, with the strongest and more direct effects on the UK and EU. It is too soon to say with any certainty as to the economic implications that affect orphan drugs, but the legal changes provide some insight on what to expect for the next five years. The EU remains a stronghold for rare disease policy and care, with or without the UK, but it is unclear how much the orphan drug landscape will change due to this significant event in world history.

*This article was originally published on June 24, 2016.

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Cite This Article:

Ho J. What does the Brexit mean for rare disease care? Illustrated by C. Suart. Rare Disease Review. July 2017. DOI:10.13140/RG.2.2.26796.05768.