As a member of the Rare Disease Forum, we welcome the latest report and its recognition of the role the pharmaceutical industry plays in tackling the challenges of Rare Diseases, from research to new medicines for patients. We also support the implementation of the UK Strategy for Rare Diseases as a core part of the Government’s broader strategy for Life Sciences.
Click here to read the report Delivering for patients with rare disease: Implementing a strategy
Dr Virginia Acha, Executive Director of Research, Medical & Innovation said,
"The pharmaceutical industry supports a national strategy on rare diseases which makes it easier for patients to access the medicines they need, and which helps companies overcome the unique regulatory barriers faced when developing new, innovative treatments for uncommon conditions. The strategy is not the end of the process, and we must now focus on getting it done.
"The UK has more than 6,000 rare diseases, which affect three million people and there a significant challenges in finding new medicines. This can't be solved by the pharmaceutical industry alone.
"We look forward to continued collaboration with healthcare, academia, charities and government partners in delivering medicines to support rare disease patients throughout England and the UK."
The report calls for three issues to be tackled - 'Consolidation' 'Collaboration' and Evaluation'. ABPI welcomes these core issues and the industry is ready to play its part in delivering the best outcomes for those living with rare diseases, both nationally and internationally.
While the report shows there has been good progress overall, there are still issues about the need for more clarity on implementation of the strategy. The lack of a strategy implementation plan for England also makes it difficult to link any improvements directly to the objectives of the UK Strategy, so there is more work to do.
We take the development of medicines for rare diseases extremely seriously. We need an approach that makes it easier for orphan medicines - products for diagnosis, prevention or treatment of life-threatening or debilitating rare diseases - to be developed, and these new medicines made more readily available to patients.
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About the ABPI
The ABPI represents innovative research-based biopharmaceutical companies, large, medium and small, leading an exciting new era of biosciences in the UK.
Our industry, a major contributor to the economy of the UK, brings life-saving and life-enhancing medicines to patients. We represent companies supplying around 90 per cent of all medicines used by the NHS, and are researching and developing the majority of the current medicines pipeline, ensuring that the UK remains at the forefront of helping patients prevent and overcome diseases.
The ABPI is recognised by government as the industry body negotiating on behalf of the branded pharmaceutical industry, for statutory consultation requirements including the pricing scheme for medicines in the UK.