BLOG: Rare disease patients in limbo from Brexit negotiations
The Brexit Health Alliance believes that patients across the European Union, including the UK, currently benefit enormously from the existing close collaboration between medical researchers who investigate, develop and test new treatments on an EU-wide basis. This is perhaps most clearly seen in the collaborative EU work on rare diseases.
Rare diseases affect an estimated 3.5 million people in the UK and 30 million people in the EU. It is currently possible to identify more than 6,000 different rare diseases. The move towards personalised genetic medicine (an NHS priority) means that there will be an increased trend of research conducted into understanding subtypes of diseases which have fewer numbers of patients. In effect, this means there will be more rare diseases.
Importance of shared EU regulatory frameworks
Shared regulatory frameworks at EU level have been crucial in providing patients with faster access to treatments. In the global market, separate national systems of regulation of health technologies can slow down treatments for patients. This applies particularly to smaller patient groups, which is why regulatory oversight across the EU brings particular added value for children and rare disease patients.
The EU regulation on orphan medicines has incentivised the development of products intended for the diagnosis, prevention or treatment of life-threatening or very serious rare conditions. The value of a single market can be clearly seen in the rare disease sector where medicines for rare diseases and medical technologies get a single authorisation to be used in the EU, providing for cost-effective licensing and distribution of medicines for small populations.
The Brexit Health Alliance is calling for maximum co-operation and harmonisation of frameworks governing regulation of medical research, medicines and medical devices.
European Reference Networks - the future of diagnosis and treatment of EU rare disease patients
Established in Spring 2017 to improve research into and diagnosis and treatment of rare or low prevalence complex diseases, 24 thematic European Reference Networks (gathering over 900 highly specialised healthcare units from 26 countries) are the most advanced and innovative form of cross-border co-operation between healthcare providers across the EU.
Since 2017 the European Commission has been assisting the hospitals involved in the networks to build an appropriate IT platform to enable clinicians to communicate and cooperate on diagnosing and treating patients. The pilot phase of this IT platform, called the Clinical Patients Management System, started in November 2017. This means the first patients have benefited from the expertise and knowledge of a multi-speciality clinical team from across Europe, without having to travel from their home country. Throughout the trial phase, 15 European Reference Networks will start using the system to diagnose and treat real patients, with the aim of testing and validating it, with a view to a full roll-out in Spring 2018.
This is a very important development which has the potential to revolutionise the delivery of EU cross-border healthcare for the benefit of patients, clinicians and healthcare providers alike. But these networks and both EU and UK patients are in a state of limbo because of Brexit.
The Brexit Health Alliance is calling for continued UK participation in European Reference Networks for rare and complex diseases, to benefit patients in the whole of Europe.
Niall Dickson, who co-chairs the Brexit Health Alliance says: “We need to make sure UK and EU patients do not lose out as the UK leaves the EU. With little more than a year before Britain leaves the European Union, we need to settle this uncertainty as soon as possible"