UK Rare Diseases Framework

Part of the debate – in Westminster Hall at 5:03 pm on 24th March 2021.

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Photo of Andrew Lewer Andrew Lewer Conservative, Northampton South 5:03 pm, 24th March 2021

It is a pleasure to serve under your chairmanship, Mrs Miller. I congratulate Liz Twist on securing this important debate.

Motor neurone disease is a devastating and rapidly progressing neurological condition that leaves individuals unable to walk, talk, eat and, ultimately, breathe. It is a rare disease in this sense, because sadly one third of people die within one year of receiving their diagnosis, but at present one in 300 people will develop it in their lifetime.

There is currently no cure, but as chairman of the all-party parliamentary group on motor neurone disease, I have had the privilege of hearing about the pioneering research that is under way to find effective treatments. Huge progress has already been made, particularly in terms of understanding which genes cause the disease and of subsequent pioneering gene therapy trials such as that conducted by Professor Chris McDermott at the University of Sheffield.

It would be disappointing, when scientific advancements are at their most promising, to see Government funding for MND research plateauing. Although charities have picked up the shortfall, this source of funding is under more pressure than ever because of covid-19. In this context, the Government’s recent announcement of the rare diseases framework provides a welcome and much needed opportunity. It is encouraging that one of the framework’s key priorities is to improve access to specialist care, treatment and drugs, and that one of its underpinning themes is to encourage and support pioneering research into rare diseases. Key to successfully delivering this will, in part, be the completion of ongoing NICE methods and a process review changing how we access new medicines. It will also come from close partnership between the devolved Administrations and the voluntary sector, which is already working to support pioneering research.

In the same vein, MND Scotland and My Name’5 Doddie Foundation are asking the Government to consider investing £50 million over five years to establish a virtual MND research institute. It would be designed to create a world-leading drug discovery and development programme, to establish a sustainable MND trials platform and to implement a rigorous clinical research programme. The institute and the funding would help the national and local delivery of this new framework’s key aims of improving the lives of those living with MND and embedding personalised care in the UK healthcare system. I thank the Government for the support they have already shown, and I look to them for more regarding the research institute.