Drugs: Ultra-rare Diseases

George Freeman Excerpts

Tuesday 16th June 2015 Westminster Hall

[The Parliamentary Under-Secretary of State for Life Sciences (George Freeman)]

I start by thanking Mr Speaker and the Speaker’s Office for granting this debate and for allowing the Twitter debate, which has been a big step forward for public engagement. I thank the hon. Member for Leeds North West (Greg Mulholland) for bringing the debate to this forum this afternoon. I suspect that in the 14 minutes I have available, I will not be able to answer every question, but I have made a detailed log and, with permission, perhaps I can write all those present with answers if I run out of time.

I pay tribute to the parents and the patients, some of whom are here today, whom I have got to know over the past few months, particularly Sam Brown and his mother, Katy, Jagger Curtis and Archie Hill. Others have mentioned the MPS Society and Christine, and the many people in Action Duchenne and the Muscular Dystrophy UK group. This campaign raises some of the hardest issues at the heart of public health and the NHS, and is being driven hard by the parents and patients with active representation from all parties in the House. It is my job to respond as best as I can and to try to put in place a policy landscape, but I pay tribute to them for their work in raising difficult issues that need to be dealt with. I do not think anyone can fail to be moved by the situation that the parents and children find themselves in. I assure them, as I do everyone else in the Chamber, that I wish there were an easy solution.

It is absolutely right that every child and patient in this country should ask for and expect the very best from our NHS, but it is equally true that, as a taxpayer-funded, universal, free-at-the-point-of-use, comprehensive health service, we simply cannot afford to provide every single treatment. I will say some more about the pressures on the system, particularly in the field of rare diseases.

As several Members have been kind enough to point out, this is one of the issues on which I have worked most tirelessly since taking office as the first Minister for Life Sciences last year. I have had several meetings with the hon. Member for Leeds North West and campaigners. Indeed, the Prime Minister and I spent more time talking about this subject than any other in my first nine months in office. I continue to work with NHS England to help it develop a more appropriate mechanism for the transparency and timeliness of its processes in all the specialist services. I have met MPs from all parties, patient groups, drug companies, campaigners and children, and I will continue to be happy to do so.

These are some of the most complex, difficult and life-changing decisions that any Department has to deal with. It is in everyone’s interest that such decisions are taken not by politicians but by clinicians and healthcare professionals, whose job it is to make those decisions—indeed, they do it for us every day of every week of every year. I thank them for that.

I want to discuss the context in which the challenge of rare diseases is developing, and what the Government are doing about it. I also want to discuss the timetable for the specific drugs that have been mentioned. I will then deal with some of the questions that were asked. We are at the forefront of an extraordinary revolution in biomedicine that is increasing pressure on all healthcare systems throughout the developed world, and will continue to do so. There are currently more than 6,000 rare diseases, and it is estimated that one in 17 people will suffer from a rare disease at some point in their lives. Therefore, more than 3 million people have a rare disease in the UK. The NHS is attempting to put in place a fair mechanism for dealing with their needs as best as it can.

The term “ultra-orphan” has no formal or legal definition, but it is taken to mean a disease

“affecting fewer than 500 people in England”,

which means a prevalence of around one in 100,000 patients. Having come to the House after a career in biomedical research, I know well that rare-disease pressure is going to grow exponentially as the extraordinary advances in genomics and biomedical research mean that we discover that more and more diseases that we used to think of as a one-size-fits-all blockbuster are rarer diseases that require stratified, targeted and, ultimately, personalised therapies. I can assure the House that the Prime Minister, who has experienced first hand the huge pain of rare disease in a family, feels that personally. We have devoted time to trying to tackle it, and will continue to do so. Indeed, that is part of the reason why the Prime Minister created my role: part of my remit in government is to tackle some of these issues.

We are doing a number of important things on rare diseases. We have put £20 million into funding the National Institute for Health Research’s rare diseases translational research collaboration and £900,000 into funding to support the work led by Public Health England to establish the first UK rare diseases register. We are leading the work with other EU countries and key colleagues to develop a European reference network to support research. We have also launched the precision medicine initiative and the rare disease consortium, but we are going further.

Central to the mission of the new ministry of life sciences is dramatically accelerating UK leadership in the field, which is why we have established Genomics England. We are the first nation on earth to seek to sequence the full genome of 100,000 NHS patients and combine that with clinical data to form the world’s reference database for targeted and stratified diagnostics and treatments. That is why we have launched the stratified medicines initiative, and why I have launched the early access to medicines scheme.

A number of colleagues challenged me about whether we were getting a grip on this: I have launched a review of the accelerated access to innovative medicines and technologies for that reason. I can assure Members that the scope of the review has struck a chord around the world. We are looking at NICE and at the regulator, and the vision of the review—I have asked for first recommendations this autumn—is to look at how we can dramatically accelerate the timeline for innovative medicines to come into the NHS, dramatically shorten the timeline for patients, and unlock what is essentially the great win-win at the heart of the NHS. We can use its research potential—its genomic and clinical informatics potential—as the world’s only integrated healthcare system to drive research into new drugs and bring down the time and cost of developing them. That way we can get drugs tested and developed here, to the benefit of our patients, while putting this country at the forefront of the revolution again.

We need to remember that it typically costs a billion to a billion and a half pounds and takes 10 to 15 years to develop a new medicine. That is unsustainable for the industry and for us. We cannot afford to pay the premium price at the end of patent life that the industry requires. We are leading the global race to put in place a new landscape. I fear that the solution will not come in time to solve the particular funding issue that has been mentioned, either this month or this quarter, or even this year. Nevertheless, we are making rapid progress. We will look back with pride on the UK’s leadership in this field.

NHS England has in place very carefully worked out decision-making processes for making drugs for rare diseases available to patients, and I want briefly to outline how they work. Because of their rarity and the low patient populations, services for rare conditions are commissioned nationally by NHS England, as opposed to locally by clinical commissioning groups. These specialised services include 146 prescribed medical services set out in legislation and account for approximately 14% of the total NHS budget—£14 billion a year. It is worth remembering the price of the system. For just this one class—the Translarna drugs alone—we are talking about hundreds of millions of pounds over a lifetime. We have to reduce the cost. We simply will not be able to afford the price required by the companies for every single new class that comes on stream.

The NHS England specialised commissioning process has been set out very carefully. It starts with one of the 68 clinical reference groups in NHS England creating a commissioning policy, which is produced by clinicians and other medical professionals. The commissioning policy is referred to one of the care boards and then to a clinical panel, which assesses the draft policy against the known evidence, with particular regard to clinical effectiveness and cost-effectiveness. The supported policies are passed on to the clinical priorities advisory group, which ensures that due process has been followed and makes a recommendation to the specialised commissioning oversight group. It considers the appropriateness and relative priority of new and existing treatments. The final sign-off is by the specialised commissioning committee, an NHS board sub-committee. NHS England’s clinical priorities advisory group formulates recommendations on the basis of clinical advice. I stress to colleagues across the House that it is not in anyone’s interest for Ministers ever to attempt to intervene in clinical decisions.

I want to touch on the timetables for the drugs mentioned by a number of colleagues: Translarna and Vimizim. On Translarna, the clinical priorities advisory group developed the clinical commissioning policy for the treatment of the mutation, and the policy was out for consultation between 24 March and 23 April. The group is considering the draft commissioning policy today and tomorrow and will make a recommendation to the specialised commissioning oversight group very shortly. The oversight group will consider the recommendations on 24 June and make recommendations to the specialised commissioning committee. The committee will make recommendations on 30 June and then make a decision on whether to commission Translarna nationally until NICE releases final guidance.

Before purdah, I was delighted to refer Translarna as a topic for evaluation by NICE’s highly specialised technologies programme. It is unfortunate that the general election fell right in the middle of the consultation process; that explains some of the difficulty we had dealing with the correspondence, as the hon. Member for Leeds North West mentioned. Final guidance on Translarna is expected in February 2016; draft guidance will be complete by the end of this year.

Similarly, Vimizim is being considered by the clinical priorities advisory group today and tomorrow and a recommendation will go to the oversight group. That recommendation will be considered on 24 June, and the final recommendations will be made on 30 June for subsequent consideration. NICE’s highly specialised technologies programme will release final guidance on Vimizim in October 2015. It is important to point out that NICE has not yet issued its final guidance on Vimizim to the NHS. I encourage patients, the public, professionals and the manufacturer, BioMarin, to engage with the ongoing consultation.

I have several questions to answer with just under 120 seconds remaining. It would not be appropriate for me to try to spin through every one, so with colleagues’ permission, I will write with detailed answers to them all. Several Members from across the House asked whether we could do something to raise money more quickly to purchase these drugs. I am discussing with the Chancellor the whole issue of how we purchase specialist drugs and put in place a landscape so that we are not only bringing drugs more quickly into the NHS and unlocking its power as a research engine, but updating our commissioning structures.

The accelerated access review that I am leading does not just address how we light the runway in terms of regulations and NICE and the Medicines and Healthcare Products Regulatory Agency’s processes to bring drugs to proof of concept in the system more quickly; it specifically looks at how we can commission better. It also deals with the cancer drugs fund. I hear the comments from north of the border—I used to advise Scottish Enterprise on this whole field. We will look at whether we might put in place some kind of innovative medicines fund for rare diseases and specialist drugs to support testing medicines within the system in a research medicine setting, particularly for rare diseases.

In the next few years, Genomics England and our leadership of genomic insights into diagnostics and new drugs will bring on a range of potential new therapies. We need to ensure that England has a landscape for testing those drugs that is compatible with Scotland. That may well mean that we will not pay premium retail prices to manufacturers at the end of a traditional phase III or phase IV development process, but build a new model of commissioning based genuinely on evaluation, thus unlocking the power of the NHS as the world’s greatest research engine.

Motion lapsed (Standing Order No. 10(6)).