George Freeman responds to debate on access to drugs for ultra-rare diseases

George Freeman to meet with NHS England, NICE and manufacturers to attempt to broker deal to secure access to drugs for ultra-rare diseases.

The Parliamentary Under-Secretary of State for Business, Innovation and Skills (George Freeman): I congratulate the hon. Member for Leeds North West (Greg Mulholland) on his tireless work on this issue, and colleagues across the House, including the hon. Member for Blaydon (Mr Anderson), my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) and others here today.

I thank the hon. Member for Leeds North West for his kind words about the work that I have been trying to do for him, and about the Prime Minister’s signal of support. The issues are incredibly complex and do not lend themselves to an easy waving of a ministerial wand, but we are committed to finding a solution.

The hon. Gentleman has been tireless in his support of one of his constituents, six-year old Sam Brown from Otley, who has the very rare Morquio syndrome. A new treatment is now available called Vimizim, from which Sam has already benefited as part of a clinical trial. I wish to state my support for Sam and his family, and for all those who suffer from the disease, including those in the trial who have access to the drug when others currently do not. I also pay tribute to the hon. Gentleman’s support for the family of another young boy, Archie, who has Duchenne muscular dystrophy, a very rare form of muscular dystrophy that affects only boys. Archie’s family want him to be treated with a new medicine, Translarna.

I will say a little about the background to the diseases and what we are trying to do about them. Both conditions are very rare—there are about 80 children living with Morquio syndrome in England, and about 140 boys with Duchenne muscular dystrophy—so we are talking about a very small number of children with those life-limiting conditions. However, rare diseases are not rare: there are between 5,000 and 10,000 known types of rare disease, and an estimated one in 17 people will be affected by a rare disease in their lifetime, amounting to some 3 million suffers in the UK alone.

The truth is that the more we know about the human genome and the behaviour of genes in disease development, the more we understand its complexity. In cancer particularly, we know that the tumour itself mutates at different stages of the disease. The more we know about genetics, the more we discover that diseases that we thought yesterday were one disease in fact break down into different bundles of rare disease. New knowledge, technology and advances in biomedicine are a wonderful thing, but that does not detract from the fact that the NHS operates with finite resources and that difficult funding decisions must be made daily.

I was delighted to meet Sam’s mother and Archie’s family early in December, along with the hon. Gentleman and representatives of the Society for Mucopolysaccharide Diseases, to whose work I pay tribute, and of the Muscular Dystrophy Campaign. As the hon. Gentleman mentioned, we had a number of meetings over the Christmas period. I was delighted to meet patient groups and the manufacturers of Vimizim and ataluren just before Christmas. In that meeting, I asked the patient groups and companies to set out their proposals, which they have now done. I am grateful to them, and I have passed on that information to NHS England.

This morning, I met NHS England’s clinical director of specialised services, James Palmer, and its director of specialised commissioning, Richard Jeavons, and I will convene a further meeting shortly to pursue the issues that the hon. Gentleman has raised this morning. Since he first made me aware of this issue, I have been absolutely determined to bring as much ministerial focus to it as I can. I am also grateful for his acknowledgement of the Prime Minister’s support. The Prime Minister and I are both determined to ensure, without compromising due process, that the case for these children and their families is properly heard, and that the system works as it is supposed to.

I am acutely aware of the urgency behind the hon. Gentleman’s comments today and that is why I have taken the unusual step of trying to broker an agreement on what we might do to help children affected by these diseases, but I must stress that it is for NHS England, which in the end is the responsible commissioner, to make any decisions about making funding available so that the treatments are available on the NHS. It will act on the best clinical advice from the UK’s specialist body, the National Institute for Health and Care Excellence.

I will say a little more about the options for accelerating that process in a moment, but first I will talk about our approach to improving access to treatments for rare diseases generally, because I know that this debate is being watched closely by others who have an interest in a number of other drugs and conditions, in the commissioning process, and in NHS England’s prioritisation and decision-making framework. In setting the scene, I remind right hon. and hon. Members of the pressures that the NHS faces, particularly on budgets for rare diseases. The emergence of new treatments, the increasing personalisation of medicines, the end of the one-size-fits-all model and the possibilities offered by the rapid advances that we are making in genomic medicine and diagnosis are all putting immense pressure on NHS England’s resources for the commissioning of services for rare conditions.

Ideally, of course, we would want to fund all the treatments that are shown to benefit patients in any way, but we have to make difficult decisions about how we spend the money that we have available. That is why we have put clinicians in charge of the process, so that they can make decisions based on patient benefit and on the best health economic assessments that we can make. The painful truth is that with finite resources, when we make a decision in one case to accept a drug, we will make a decision elsewhere to reject, and we have a duty to all to ensure that we make those decisions fairly.

For people with rare conditions, their families, carers and clinicians, having access to the latest and most effective treatments is obviously critical, and I am absolutely committed to ensuring that patients with rare diseases have access to the latest and most effective treatments that represent value to the NHS and the taxpayer, as well as delivering benefits to patients. That is why we recently introduced the early access to medicines scheme, which aims to give patients with life-threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation or licence where there is clear unmet medical need. I am delighted that initial products have been brought forward in the last six months under that scheme.

More generally, our strategy for life sciences sets out an ambitious longer-term plan to improve the wider environment for health and life sciences companies in this country. Recently, I launched a major review of the landscape in the UK for bringing innovative medicines and medical technologies to patients much more quickly, and I will soon announce the chair, the terms of reference, the scope and the timetable of that review.

We are not in any way complacent. The truth is that the challenges in this sector, which are being driven by the pace of technological change, demand that in our policy-making framework, in the Department of Health and in NHS England, we adapt the way in which we handle these processes. Because of their rarity and the low patient populations, services for rare conditions are directly commissioned nationally by NHS England as specialised services. They account for approximately 14% of the total NHS budget and represent spending of about £14 billion a year. Both Morquio syndrome and Duchenne muscular dystrophy fall within these national specialist commissioning arrangements.

As right hon. and hon. Members are aware, NHS England is considering draft clinical commissioning policies for both Vimizim and ataluren. I understand that they are being considered as part of NHS England’s wider prioritisation process for funding in 2015-16. NHS England’s clinical priorities advisory group formulates recommendations on the commissioning of new treatments for rare diseases in England. It is made up of clinicians, patient representatives and commissioners of health services.

In summer 2014, a decision-making aid for the prioritisation of new interventions and treatments was developed by a partnership of stakeholders, including more than 250 patient representatives. It was due to be used for the first time in early December 2014, but on 28 November 2014 NHS England decided to postpone its introduction, in response to concerns that some patients affected by rare diseases might be disadvantaged by its application. The legal process about that must now run its course. I understand that NHS England is, rightly, reviewing the appropriate approach to prioritising new treatments and interventions within specialised commissioning in response to those concerns. A 90-day consultation on the prioritisation framework and decision-making process for commissioning decisions on new treatments will be launched by NHS England shortly. This morning, I again raised the importance and urgency of that consultation process.

I know that patients and their families are understandably concerned that it may take a long time for a decision to be made by NHS England on whether it will fund the drugs, and that in the interim the children affected will not receive them. However, I am delighted to say that NHS England has assured me that the consultation will have no impact on the decision-making timetable for commissioning NHS services from April 2015 onwards. In addition, it has assured me that existing treatments will continue to be commissioned, ensuring that support for patients is maintained. NHS England understands that the manufacturer, Bio Marin, is providing Vimizim under an expanded access arrangement to those patients who are on the clinical trial until an NHS England policy decision has been made.

Since April 2013, NICE, which is responsible for the evaluation of selected high-cost low-volume drugs under its highly specialised technologies programme, has been playing an important role in ensuring that commissioning decisions are based on a robust and thorough assessment of the available evidence. NICE has recently been asked to evaluate Vimazim under this programme, and it is also considering whether to develop guidance on Translarna. That is a very positive step, and I look forward to receiving NICE’s proposals on future topics that will be considered. I know that NICE will also be keen to learn lessons from its recent experiences with the new highly specialised technologies process, to make that process as efficient and effective as possible.

For my part, I am absolutely determined to continue playing the active role that I have taken on in the last few months, to drive this process and give it the focus that it requires. I am delighted to have confirmed with NHS England that it will continue to meet the treatment costs. I have signalled, and will continue to signal, to NICE, without compromising its processes, the strength of the case that has been made by Members and patient groups to put Translarna on the list, and to consider whether it can expedite its process in any way, but I do not want to compromise that process in any way. I will also ask NICE to ensure that it uses its review of the experience of the HST programme to explore how we can speed up both this process and others in due course.

Finally, I am committed to continuing to work with the companies to see whether I might be able to help broker some kind of planning arrangement that might encourage NICE to make the decision that I know everyone in Westminster Hall today would like to hear.

Mrs Gillan: I am grateful to the Minister and I congratulate him on taking up the cudgels on this issue and trying to move it forward. The Muscular Dystrophy Campaign has asked whether the individual funding requests from patients would be a route to secure access to Translarna while the Minister is waiting for due process to take its course, because I am afraid that muscular dystrophy waits for no man and no process.

George Freeman: I understand; my right hon. Friend makes an important point. In fact, I raised it this morning in my meeting with NHS England. My understanding is that NHS England will continue to consider individual applications for Translarna through its individual funding request process from patients who may be exceptional. However, my understanding is that such cases really do have to be exceptional. In reality, the members of the whole group that we are considering are more or less suffering from the same condition and therefore they may not qualify under those criteria. I merely share that with my right hon. Friend because I myself raised that point this morning with NHS England.

Greg Mulholland: I stress to my hon. Friend the Minister that we are discussing two conditions and two drugs, Translarna and Vimazim. I also have to say to him again that we understand that NHS England has to put a process in place; of course it does, because the process it had put in place was not fit for purpose. Does he accept that NHS England has a legal, as well as a moral, responsibility in this regard? It certainly has a moral responsibility. Having said that the decision will be made on 15 December, NHS England cannot now hide behind saying, “There needs to be a new process,” when this situation is their fault in the first place. We are now a month on from that initial deadline, and there needs to be an interim solution to somehow allow these 138 children to access the two drugs in the meantime, and before that process is complete.

George Freeman: I certainly accept the moral case; I think that everyone would accept that there is a moral duty to get this matter right and to try to make these decisions on the right basis and on the basis of the right evidence. The legal position, given the legal challenge, is more complicated, and it has triggered a formal process of reappraisal. As I have said, I will meet NHS England officials to urge them to try to expedite that process as best they can. However, I must stress that I do not want to get into a situation where we compromise due process and inadvertently undermine a case. What I want to see is a NICE decision being made as quickly as possible, and I will urge NICE to expedite that process in every way it can, so that we get the right decision that we all want.

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