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Rare disease patients should not be disadvantaged

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rare disease disadvantage

Rare Disease Day is major event across the globe. The main objective of the day is to raise awareness with policymakers and the public of rare diseases and their impact on the lives of patients.

Research is paramount for beating rare diseases

The theme for this year’s Rare Disease Day, for the second consecutive year, is research.

There are 6,000 known rare diseases and it is estimated that this number grows at a rate of five new conditions each week. For the majority, there are no effective treatments. If this is to change, research will be the key and patient involvement in research will be of fundamental importance.

It is critical the UK government ensures we remain able to collaborate with our EU neighbours.

Patients and patient organisations raise funds to support and promote research. They actively ensure research addresses their unmet needs and preferences, providing insights that improve research study design. Patients are the glue that binds together the complex ecosystem of researchers, drug developers, regulators, policymakers and professionals, which allows innovative and more effective models of care and treatment into practice.

The impact Brexit could have on research and collaboration

As the date of our withdrawal from the European Union grows closer, it is critical the UK government ensures we remain able to effectively collaborate with our EU neighbours. The number of patients with any given rare disease in any one country is small and so rare disease must reach across national borders.

EU regulatory incentives for rare disease medicines have helped deliver almost all of the licenced rare disease medicines in clinical use today. In terms of clinical care, the 24 European Reference Networks (ERNs) for rare diseases bring together a community of over 900 doctors working in centres of expertise, facilitating the sharing of knowledge and skills, expediting access to diagnosis and treatment. These things must not be lost.

England is paving the way for the UK rare disease plans

Just a few weeks ago, England became the final UK nation to publish its implementation plans for the UK Strategy for Rare Diseases.

The plans in our four nations aim to strengthen and incentivise research and improve diagnosis and advance the delivery and coordination of care. With these plans in hand and now more than ever, we must unite across borders, working together across the UK and internationally. We must make sure that patients affected by rare diseases are not disadvantaged.

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