Duchenne Muscular Dystrophy: Givinostat

Lord Sharkey Excerpts

Tuesday 22nd July 2025 Lords Chamber

[Baroness Blake of Leeds (Lab)]

I cannot comment on this case as the decision has not been made public yet; that would be completely inappropriate. If NICE does give its approval, the Secretary of State has committed to bringing it in. We are looking at ways in which we can get the medicines for rare diseases to which NICE gives its approval to where they are most needed as speedily as possible.

[Lord Sharkey (LD)]

My Lords, I declare an interest as a trustee of Muscular Dystrophy UK. Following the previous question, can the Minister say whether the early access programme really works? How many other drugs have been licensed under the programme, how many were eventually approved by NICE and how many were declined? If the application for NICE approval of givinostat is unsuccessful, what will happen to the patients currently undergoing treatment with the drug? Will that depend partly or entirely on the pharmaceutical company that makes the drug?

[Baroness Blake of Leeds (Lab)]

I cannot give the detailed answer on the numbers that the noble Lord is after, but I am very happy to write to him. We have this experience in a whole range of other medicines, but I appreciate the noble Lord’s particular interest in this. This is a very difficult area. It is for NICE to look at the best way forward on providing medicines that give the best results and value for money and achieve the right outcomes. This is an ongoing situation, and I am happy to write to the noble Lord on the detail.