Duchenne Muscular Dystrophy: Givinostat

Baroness Blake of Leeds Excerpts

Tuesday 22nd July 2025 Lords Chamber

[Baroness Kramer]

To ask His Majesty’s Government what progress has been made in making givinostat available to boys living with Duchenne muscular dystrophy to slow down degeneration, particularly becoming unable to walk.

[Baroness in Waiting/Government Whip (Baroness Blake of Leeds) (Lab)]

NICE is currently evaluating givinostat for the treatment of Duchenne muscular dystrophy to determine whether it should be funded by the NHS, based on the evidence of its costs and benefits. Its independent appraisal committee met on 10 July, and I understand that NICE will issue draft guidance shortly. I hope noble Lords will appreciate that it would not be appropriate for me to speculate on NICE’s draft guidance or to seek to influence the outcome.

[Baroness Kramer (LD)]

My Lords, boys M and H are friends aged 14 and 11, and both have Duchenne muscular dystrophy. Boy M is receiving givinostat, provided by his NHS trust, and expects to preserve his ability to walk and to be eligible for new drugs in the pipeline. Boy H’s NHS trust is not providing him with givinostat. His family fear he will lose his ability to walk before September, and he is unlikely to be eligible for future drugs. Givinostat is cost-free from the manufacturer under the early access programme, but delivery and monitoring costs fall on the NHS. What can the Minister say to boy H and the 400 other boys like him who are losing in the postcode lottery? Will she meet with some of the families?

[Baroness Blake of Leeds (Lab)]

The noble Baroness raises a very interesting point. The drug companies will provide the drugs for free, but it is down to individual trusts to pay the administrative costs. She highlights that one boy in one trust is being supported, while another boy is not. It is a difficult situation; I completely understand that, and my heart goes out to all the young people who suffer from this dreadful disease. It is critical that the trusts work together and look at other practice. It is not for the Government to intervene and force trusts to do as the noble Baroness proposes. However, if they choose to do so, companies can provide funding to enable the clinical administration of their products in advance of the NICE decision. Perhaps the noble Baroness could go back and recommend that as a course of action.

[Lord Kamall (Con)]

My Lords, we heard from the noble Baroness, Lady Kramer, that there is an early access programme for a new treatment of Duchenne muscular dystrophy in the UK. Can the Minister share any intermediate observations or results from the early access programme? As the noble Baroness, Lady Kramer, asked, are there any plans to extend or expand it?

--- Later in debate ---

[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.

[Lord Evans of Rainow (Con)]

My Lords, I witnessed a close family member suffer with this dreadful disease, and the Minister is right to say that she cannot comment on the actual drug and NICE’s decision. However, before this drug is administered, you require blood-clotting platelets so that the drug can have the best effect. However, there is a big shortage of platelet donors within the NHS, as the noble Baroness may be aware. Waiting for NICE’s decision may be part of the administration cost of administering this drug, but one thing it may use to say it cannot do it is a shortage of donors, so perhaps we can proactively ask the NHS to look into how we can get more donors for platelets.

[Baroness Blake of Leeds (Lab)]

The noble Lord touches on a very sensitive matter that could be replicated around a variety of issues. Looking for more people to come forward is a perpetual challenge, and I am sure that everything should be done to encourage people to do so. I am sure that many people do not know that this is an issue, and some promotion could be helpful.

[Lord Kakkar (CB)]

I draw the House’s attention to my registered interest as chairman of King’s Health Partners. The 10-year plan recently published by His Majesty’s Government, with regard to the future of the NHS, puts at its very centre the adoption of innovation at scale and pace to transform the health service and secure its sustainability. What arrangements are in place, and what assessment is being made of those mechanisms? In particular, do His Majesty’s Government have a view about advancing the opportunity for early adoption of innovation in such a way that the kinds of problems we have heard about in this short Question are not repeated on several occasions, and the ambition to transform the health service with innovation is not frustrated?

[Baroness Blake of Leeds (Lab)]

The noble Lord raises a crucial point: there is no point having all the work going into innovation if we cannot implement it. It is at the centre of the 10-year plan. A process is being worked up to look at how we can bring the best of our innovation and technological advancement into clinical practice. I am grateful for his input into this area, and I know that this will be taken very seriously as we move forward.

[Baroness Blackwood of North Oxford (Con)]

My Lords, I declare my interest as chair of Genomics England and Oxford University Innovation. This product is a classic example of one that should be eligible for the innovative medicines fund. However, concerns have been raised about the clarity of entry criteria, transparency and scale of ambition. What steps are being taken to tackle those challenges in order to address the concerns raised by the noble Lord, Lord Kakkar?

[Baroness Blake of Leeds (Lab)]

I am not sure that I can add a great deal more, other than to refer the noble Baroness to the 10-year plan, where innovation is absolutely at the core and the centre. We are where we are, and her point is about how we move from here to where we need to be. We need to make sure that there is a real focus on moving all that expertise into actual practice to bring benefit to as many patients as possible.

[Baroness Burt of Solihull (LD)]

My Lords, currently only 70 of the 500 children with Duchenne are accessing the trial of givinostat. We are hoping for a decision on 18 December from NICE, but my worry is about how long the rollout will take for sufferers and their parents. They want to know how long it will take for those who are not included in the fortunate 70 currently receiving givinostat, and for others in categories that may also be helped.

[Baroness Blake of Leeds (Lab)]

I recognise the noble Baroness’s concern and in-depth knowledge about this situation. I need to repeat at this point that it would be wrong of me to speculate on the outcome. I hope that the decision will be earlier than the date she mentioned. When the guidance is published, we will be in a much better position to assess next steps.