Industry Responds to Government Strategy to Improve Services for Rare Diseases

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The ABPI has responded to the UK government’s recently published plans on improvements to services for people with rare diseases

The Association of the British Pharmaceutical Industry has responded to the United Kingdom government’s recently published plans on improvements to services for people with rare diseases, in a Feb. 27, 2019 statement.

Within the National Genomic Healthcare Strategy, laid out by the UK government, are action points aimed at improving services. As reported on the government’s website, these action points include the following provisions: 

• Every person with a rare disease will have a dedicated person responsible for co-ordinating their care.

• Every patient with a rare disease will be given an ‘alert card’, including information about their condition, treatment regime, and contact details for the individual expert involved in their care.

• Every child with a rare condition will be transferred to appropriate adult services when they reach the age of 18, even if that adult service is not the commissioning responsibility of NHS England.


Additionally, the strategy has set out how the genomics community can work together to make the UK a global leader in genomic healthcare. Minister for Innovation, Nicola Blackwood, said, “Through the NHS Long Term Plan we want to lead the world in the use of data and technology to prevent illness-not just treat it. We want to diagnose conditions before symptoms occur. And we want to deliver personalized treatment, informed not just by our general understanding of disease but by our own personal, de-identified medical data-including our genetic make-up. In order to make this a reality, I am delighted to announce that we will be working with the National Genomics Board and the broader genomics community to develop a National Genomic Healthcare Strategy.”

Responding to the government’s announcement, Dr Sheuli Porkess, deputy chief scientific officer at the ABPI commented, “Three million people in the UK are affected by a rare disease at some point. However, 95% of rare conditions have no licensed treatment. Delivering on the government’s rare disease strategy will be an important part of changing this. By focusing on personalized medicine and genomics, pharmaceutical companies can play a leading role in improving the lives of people with rare diseases. We will continue to work with our partners in NHS England, NICE, MHRA and the Department of Health so that people with rare diseases can access the treatments they need.”

Sources: Gov.ukABPI