UK Rare Diseases Framework

Part of the debate – in Westminster Hall at 5:22 pm on 24th March 2021.

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Photo of Daniel Zeichner Daniel Zeichner Shadow Minister (Environment, Food and Rural Affairs) 5:22 pm, 24th March 2021

I, too, congratulate my hon. Friend Liz Twist on securing the debate. I chair the all-party parliamentary group on life sciences and want to raise a couple of wider points. I am grateful to the Association of the British Pharmaceutical Industry and the UK Bioindustry Association for advice.

We are all familiar with the figure of Sir John Bell on our television screens, but in the current circumstances would the Minister tell us a little about the status of the life sciences industrial strategy and sector deal? It seems that the industrial strategy has gone by the wayside. What is going on? Will she also tell us how the various initiatives, including the NICE methods review, the innovative medicines fund and the recently published commercial medicines framework will work together to support access to medicines for rare diseases?

There has been a long-running issue around the NICE process review in relation to the single technology appraisal programme, which many feel is not very suited to the specialised medicines world. It led to the highly specialised technologies programme, but there is a continuing gap between the two, and perhaps she can shed some light on that.

Professor Lucy Raymond was professor of medical genetics and neurodevelopment at the department of medical genetics in Cambridge, and has welcomed me on Rare Disease Day in previous years. She made a couple of important points, saying that despite the welcome work done by the 100,000 genomes project at such places as the Wellcome Sanger Institute, there are still long delays, and testing is limited by resources and NHS England funding. Secondly, there is the question of making sure there is sufficient access to clinical trials. Professor Raymond suggests that as we have a limited number of nationally funded genomic laboratories, it could be possible to introduce a statutory obligation to notify, which would produce a bigger group for clinical trials.

Finally, on the question of our relationship with the European Union, what progress has been made on developing a rare disease trial protocol?