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Experts have warned that a no-deal Brexit will lead to the UK's exclusion from the 24 European Reference Networks (ERN) set up to improve care for patients who bear the lifelong burden of a rare disease and require highly specialized diagnosis and treatment.
One in 17 British citizens lives with a rare disease, which is defined as a disease that affects less than one in 2,000 people in the general population. A group of experts wrote to The Lancet highlighting their concern about the adverse effects of a no-deal Brexit on these people.
"Rare diseases are rare and experts are even rarer," said Dr. Marc Tischowitz from Cambridge University who coordinated the letter. "European reference networks were created because no country has the expertise or the resources to cover all known rare diseases, of which there are thousands. They have a critical role in harnessing the collective knowledge across the continent and in developing one Health care played a sustainable role in treating those affected. "
The UK has led the way in creating and developing these virtual networks involving healthcare providers across Europe. As a result, the experts write, it "took advantage of closer collaboration with experts and patient representatives across Europe".
The ERNs have made it much easier to develop guidelines, create disease registries, build research collaborations, and create new education and training programs. Crucially, they have directly improved patient care by setting up a pan-European platform where international experts come together to advise on complex patient-specific problems and therapeutic options that are lacking in expertise in one country alone.
Dr. Tischowitz added: "If we leave the EU without an agreement on UK participation in the networks, we may be able to write off years of advances made by UK clinicians, researchers and patient advocates while limiting access to clinical trials and funding. Most importantly, our ability will affect the ability to provide the best possible care to millions of children and adults with rare and complex diseases in the future. "
The letter has a total of 73 signatories, including 19 signatories each representing a patient support group and 54 signatories from senior clinicians and researchers who are currently members of a European Reference Network and will be removed from the networks as of January 1st if no agreement is reached is reached.
Allison Watson co-founded Ring20, a charity that helps people with Ring 20 syndrome, an extremely rare disease that affects their young adult son. She is also co-head of EpiCARE ERN for rare and complex epilepsy.
"I am very encouraged by the change being part of an ERN can bring to people like my son and many others with extremely rare diseases," said Watson. "I think we couldn't have done this with just British rare disease organizations."
Initiatives already carried out through the EpiCARE ERN include increased awareness of rare epilepsy (including ring chromosome 20 syndrome) in the 28 EpiCARE centers, long overdue Orphanet updates, improved information and education for doctors and patient families in the form of brochures and patient trips. plus updated guidelines for clinical practice aiming to simplify and expedite diagnosis and improve care by understanding unmet needs.
Watson added, "With thousands of rare diseases, many of which are extremely rare and affect only a handful of people in the UK, it is inexpensive or even possible for the UK to provide effective services and research to these people alone I believe Only by working with our European partners and others around the world can we truly meet the needs of those affected and ultimately improve their outcomes and quality of life. "
Beverley Power, chairman of CDH UK, the congenital diaphragmatic hernia charity, says one of the main barriers to rare disease research is access to patients and patient records.
"Since joining the ERNICA European Reference Network, access to patients and data has become broader for the UK and the rest of Europe," she said. "It has enabled charities such as CDH UK to better understand other health care facilities and to steer newly diagnosed parents and patients with ongoing medical needs in a much better direction. It has also introduced new and innovative ways to work together to be more effective." Outcomes and quality of life for patients and their families, which may ultimately affect the economic impact of rare disease treatment in the UK and overseas. "
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Tischowitz, M et al. A no-deal Brexit will adversely affect people with rare diseases. Lancet, December 12, 2020. DOI: 10.1016 / S0140-6736 (20) 32631-3
No deal Brexit could adversely affect people in the UK living with a rare disease (2020, December 11th).
accessed on December 11, 2020
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