Brexit and rare diseases: big risk, bigger opportunity?

Abstract

The UK's planned exit from the EU will leave its national health sector in a very dangerous position. It will also have profound consequences for domestic UK law. The impact may be particularly drastic for patients for whom EU law protects the right to treatment. At a particular risk are patients with rare, ‘orphan’, diseases whose treatments are uniquely enabled at the EU level. We examine the potential effects of Brexit on the orphan sector and identify an opportunity to solve long-standing and intensifying difficulties, especially the pricing of orphan drugs.

Keywords:

• Orphan drug
• rare disease
• legislation
• Brexit
• access

Transparency

Declaration of funding

This editorial was not funded.

Declaration of financial/other relationships

HI Hyry and JCP Roos have attended symposia and received hospitality or funding towards attendance or related research from the UK Gaucher Association, Susan Lewis Memorial Fund, Helen Manuel Foundation and the European Working Group on Gaucher disease. JCP Roos has also received support from the National Institute for Health Research. TM Cox has received support from the UK Medical Research Council and the National Institute for Health Research as well as unrestricted research grants from Genzyme and Shire. TM Cox also advises pharmaceutical companies engaged in the orphan disease setting, including Actelion, Genzyme, Shire, Amicus Therapeutics, and Protalix Biotherapeutics, receiving speakers? fees and travel costs. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.