George Freeman commends patient-led innovation in accelerating the commissioning of rare disease treatments

8th December 2015

George Freeman outlines opportunities for the UK to use our leadership in genomics and informatics to better understand patients’ genetic predisposition to different diseases and to respond to different drugs. He commends the work of the Cystic Fibrosis Trust and other charities who are at the forefront of patient-led innovation that is greatly contributing to the work on accelerating the commissioning of rare disease treatments which will benefit their patients.

The Parliamentary Under-Secretary of State for Life Sciences (George Freeman): It is a pleasure to serve under your chairmanship, Sir Edward. I congratulate the hon. Member for Dudley North (Ian Austin) and other hon. Members from across the House who have spoken. This timely debate has been incredibly powerful—not that there has been much disagreement in it. It has been an opportunity to raise important issues that I am dealing with, and I am grateful to colleagues for acknowledging that.

The debate is particularly timely because I am convenor of a major summit today on accelerated access for faster cures. There is a precision medicine summit in London and the Association of Medical Research Charities has just held its annual conference, at which I exhorted members to come to my table with ideas about how to accelerate novel treatments and give the charities more of a voice. A powerful and helpful debate is going on.

I pay tribute to the work of the Cystic Fibrosis Trust, which is among a number of charities that lead the debate on innovative treatments and medicines. Its leader Ed Owen in particular plays an important role in that; but so do Carly, Lorraine, Michael, Kelly and the other people who have been mentioned. Many of the charities do extraordinary work to articulate the experience of patients who suffer from disease and bring it to the policy table in a powerful way. It is a change in policy making that I am keen to accelerate.

The debate goes to the heart of the challenge and opportunity that precision medicines represent for our system and the landscape of assessment, testing, approvals and reimbursement, as well as the growing role of charities and the patient voice. Those things are passions of mine and I want to discuss why, in the next few months and the years ahead, there will be dramatic progress.

The Government and I wholeheartedly support the cystic fibrosis campaign’s central aim of ensuring that as many people with CF as possible will have access to personalised medicines by 2020. That sets an inspiring and clear goal and I relish the attempt to deliver it. I want to make some remarks about the condition, about what NHS England and the NHS in Scotland and Northern Ireland are doing about treatment today, about the rare diseases and precision medicine landscape, and about the reforms that I am pushing to try to deal with the issues that have been raised.

I have had a career in biomedical research, so it is an extraordinary privilege to have been given my role by the Prime Minister, who has personal experience of the tragic consequences of genetic disorders affecting children. I am delighted to share with the House the fact that my passion to lead in this field, and unleash the power of the NHS and our research expertise in a new landscape for accelerated access, is exceeded only by the Prime Minister’s.

As hon. Members know, cystic fibrosis is the most common life-limiting inherited condition in the UK. It affects about 10,500 people in England—and more, of course, in Scotland, Northern Ireland and Wales—more than half of whom are adults. Cystic fibrosis is one of the UK’s commonest life-threatening inherited diseases. It is caused by a single defective gene. As a result, the internal organs and especially the lungs and digestive system become clogged. That results in chronic infections, inflammation in the lungs and difficulty digesting food.

The number of adults living with CF is gradually increasing over time, because of improvements in diagnosis from newborn screening and new treatments. The condition affects everyone differently—that is an important point—but for many it involves a rigorous daily treatment regime including physiotherapy, oral, nebulised and occasionally intravenous antibiotics, and taking enzyme tablets with food. For those who are very ill with cystic fibrosis and who have very poor lung function, daily life can be a struggle as basic tasks can leave them breathless. Some patients use a wheelchair to get around, and use oxygen to help them breathe.

For patients and their families, managing the condition is extremely challenging. That is made worse by the absence of an effective treatment or cure—or, as several colleagues have explained today, by the tantalising presence of a possible treatment or cure that cannot yet be administered to them or their suffering loved ones. I pay ribute to patients who grapple with the disease day in, day out, and who have done so for years, for their patience as we try to bring new solutions to the table. Current treatments generally target the complications rather than the cause of the condition. Treatments can be broadly classified as nutritional support, relief of airway obstruction, treatment of airway infection and, ultimately, lung transplantation.

What are the Government doing? I want first to talk about what the NHS is doing in England and in Scotland and the other devolved areas, and then to say something about what we are doing more strategically to tackle the new landscape.

Since April 2013 NHS England has been responsible for securing high-quality outcomes for patients with cystic fibrosis as part of its remit to deliver specialised services. Its service specifications for cystic fibrosis—one for adults and one for children—set out what providers must have in place to offer high quality care and support equity of access to services for patients with cystic fibrosis, wherever they live. The NHS England cystic fibrosis clinical reference group has developed a number of clinical policies for the treatment of patients with cystic fibrosis and it reviews outcomes with the Cystic Fibrosis Trust and with patients and charities.

As we have heard, Scotland, leading within the United Kingdom—and it is not the first time—has launched a dedicated fund worth £40 million this year to give patients greater access to new medicines, as the Scottish Health Secretary, Alex Neil, has announced today. The £40 million new medicines fund expands and replaces the rare conditions medicines fund established in March 2013, giving health boards access to greater resources. In 2013-14 the rare conditions medicines fund supported the cost of 45 different medicines, benefiting more than 200 patients, including ivacaftor for cystic fibrosis as well as other treatments for related rare diseases.

NHS England is investing significant resources into the provision of new medications that work directly on the genes causing cystic fibrosis. Since 2013, it has routinely commissioned ivacaftor or Kalydeco for the treatment of cystic fibrosis in those with a certain gene mutation affecting only 5% of the CF population. Earlier in 2015, that indication was extended to an additional eight mutations for patients aged six years and above. NHS England is considering a policy proposition for extending the use of ivacaftor for the same gene mutations to children aged two to five years. That will consider the evidence base and be included with other therapies requiring investment as part of NHS England’s prioritisation process for specialised services for 2016-17.

Several colleagues raised the matter of Orkambi. Some drugs for cystic fibrosis will be considered by NICE through its technology appraisal process, including Orkambi, which, as many will know, is lumacaftor in combination with ivacaftor. NICE is currently developing technology appraisal guidance on the use of Orkambi for the treatment of patients with cystic fibrosis. It currently expects to issue final guidance in July 2016. NHS England will commission drugs where there is a positive NICE technology appraisal, and I will say something about the changes that we envisage in the landscape in that respect.

NHS England operates a horizon-scanning process to identify new treatments and the cystic fibrosis clinical reference group advises on the development of services for patients and keeps relevant published literature under review. Where NICE is not considering a therapy, NHS England can consider the evidence base and may propose commissioning treatments through its policy development process. I shall say something shortly about changes that we are considering in the way NHS specialist commissioning might embrace the new freedoms in the accelerated access review to accelerate the commissioning of rare disease treatments.

In fact, ivacaftor is something of a mild success story. NHS England commissioned it earlier than might otherwise have been expected, having agreed, in discussion with the company that makes it, a flexible pricing model. We want to see more of that sort of innovation.

I am grateful to the hon. Member for Denton and Reddish (Andrew Gwynne) for giving me some time to answer the various questions asked, which I will try to do in some detail. First, I want to set the scene in terms of why this debate is happening and why this landscape is under such pressure. The truth is that breakthroughs in genomics and informatics—our ability to understand patients’ genetic predisposition to different diseases and to respond to different drugs, as well as the availability of large-scale data sets, including individualised patient treatment histories and anonymised cohort studies—are transforming the traditional pathway for drug R and D, which normally takes years. It now takes roughly 15 years and $2 billion to bring the average drug to patients.

Genomics and informatics, particularly for some of the rare genetic diseases, allow us to take time, cost and risk out of the development pathway in a profound way. That is driving opportunity and challenge in our system; the Prime Minister created this post and put me in it to ensure we respond to that challenge with ambition.

Julian Sturdy: My hon. Friend the Minister is absolutely right to say that the medical landscape is changing hugely at the moment, but does he feel that the wider implications of new medicines are being fully explored by NHS England and NICE? We have heard about the huge consequences of cystic fibrosis for not only the sufferer but their wider family and the NHS. Does he feel that those wider consequences are being fully explored?

George Freeman: My hon. Friend raises an important point. Over the past few decades, the NHS across the UK has played an inspiring role in leading a lot of the breakthroughs in new treatments, but we have become latterly a slower adopter of the very treatments we often helped to discover. That is partly because the pressure of an ageing society and the rising cost for the health system today of just treating existing conditions are extremely challenging. In some areas, that has made innovations appear a cost to the system, when in fact good innovations may come with a cost spike on day one but generally lead to downstream savings in years 2, 3 and 4.

My hon. Friend puts his finger on a profound challenge at the heart of this landscape: in order really to assess the impact of innovative treatments, we need a much better handle on the existing costs, many of which are hidden, that come with a diagnosis. For that reason, I am spearheading work in the Department of Health to drive through a system of per-patient costing, so that we can begin to get a much clearer handle on what a CF diagnosis means on day one for both the patient and the health economy. That will allow NICE and NHS England to develop much more intelligent systems for assessing whether an innovation really represents good value.

Genomics and informatics are changing the landscape; for that reason the Prime Minister has created my post and we have launched a series of initiatives. On genomics, we have launched a groundbreaking £300 million initiative to sequence the genomes from 100,000 NHS patients of cancer and rare diseases. We have also launched 11 genomic medicine centres across the NHS, so that genomics is fundamentally embedded in our health system. On informatics, we have released huge amounts of cohort data to drive research, and we just announced in the comprehensive spending review a major £3.5 billion programme to invest in NHS digital infrastructure to support that.

We have launched precision medicine and cell therapy catapult centres with the Medical Research Council and industry partners to lead in both understanding causal mechanisms of rare diseases and developing and accelerating new treatments. We continue to fund the excellent National Institute for Health Research, for which it is my privilege to be responsible, to the tune of £1 billion a year, and we committed this year in the CSR to fund it throughout this Parliament, at a cost of £5 billion. We have funded the £700 million Francis Crick Institute, and roughly £2 billion of the drugs budget is allocated to new medicines and new treatments in this Parliament.

There is a major commitment, in terms of science and funding, to trying to tackle this issue, but crucially we need policy reforms to ensure that breakthroughs in science can be harnessed for much quicker benefits for patients. That is what the accelerated access review and a number of other initiatives, such as the test bed programme and the vanguards I am running with NHS England, are about—trying to ensure we can change the pathways for getting innovation into our health system for much quicker patient benefit.

I want to say something about the accelerated access review and the specialist commissioning reforms that NHS England is putting in place. I know all Members here take an interest in this subject, so I hope they will be aware that I have launched the independent AAR to ask and answer one big question: what can we better do to harness the extraordinary infrastructure here in the UK in terms of our deep science research base, our NHS-NIHR research base and our NHS daily treatment platform?

The NHS is the fifth biggest organisation in the world, making millions of diagnoses and carrying out millions of treatments every day. Its original founding mission was to be a research organisation, but unless we better capture the data on those interventions, we are still practising, in many cases, blind medicine; we are not harnessing that intelligence enough to inform treatment.

I have asked that the AAR tackles three big questions. First, what can we do to allow the innovators—the developers of new drugs and innovations—quicker access to patients, to reach the all-important moment of proving an innovation works in patients? Secondly, what can we do to harness our leadership in genomics and informatics in order to create a more intelligent system for NICE and NHS England, with more flexibilities, so that they can assess, adopt, approve and reimburse innovations using real-time data about real patients? That will allow us to develop a more flexible set of pathways and adaptive tools with which to embrace this revolution.

When a drug comes to us with a genomic biomarker and we know that it will work for a certain sub-cohort of patients, that profoundly changes the risk dynamic of a traditional pharmaceutical clinical trials programme and should allow us to accelerate adoption for particular patient groups.

Rachael Maskell: Within those considerations, will the Minister also look at international evidence, so that we are looking at not only our own clinical trials but those on a global scale? Clearly, developments are global rather than just national.

George Freeman: The hon. Lady makes an important point. I have been to Washington three times and to Berlin, Paris and Brussels to highlight that while the UK is leading in this field, we need a transatlantic—European and American—agreement on how we move things forward. That is why I am convening and chairing a summit this afternoon with the Washington-based FasterCures campaign, which is a cross-party group on the Hill pushing for innovations in this space. I have been talking to the Commission about the European framework. I want the UK to be the best entry point into the European market, but I also want the European regulatory framework to be consistent and coherent; that is an important point.

The second question I have asked the AAR to look at is: what freedoms, flexibilities and new pathways can we envisage giving NICE and NHS England, particularly in the field of specialist commissioning? For CF, the decision to purchase ivacaftor is a national one, made by an NHS England specialist commissioning unit. I would like that unit to work much more closely with the Department of Health pricing team, so that where we can offer a company faster access to a key patient cohort, data and genomic information, we are able to do a much better deal with the company.

At the moment, we are operating the Translarna and Vimizim programme in the existing landscape. I share colleagues’ frustration, but it is important we go through due process. I do not think anyone wants a world in which Ministers decide what drugs come through on the basis of political pressure, tempting though it may be. I have done everything I can this year to expedite the existing process.

Following the positive news on Vimizim, I am hopeful about Translarna—a similar drug. NICE has been consulting on the process, and I believe the company has been engaging with NICE on pricing. I am hopeful that there will be a decision in the next few months to parallel the one on Vimizim, but that decision is not in my gift: it is up to NICE, which is rightly working on the basis of the very best clinical evidence.

Mrs Gillan rose

George Freeman: I had better crack on; I will come to the questions that my right hon. Friend the Member for Chesham and Amersham (Mrs Gillan) asked later, if I may.

Hon. Members have raised a number of questions and I want to deal with them all. The hon. Member for Dudley North asked about timings for the accelerated access review. We have had an interim report. I have asked for final recommendations in the spring—in March or April—and also that the review considers whether the process should go on. I want recommendations that we can implement quickly, but equally these discussions are complex and we may well need to go on to look at other bits of the landscape. I would be delighted to meet the Cystic Fibrosis Trust—in fact, that is already arranged; we are meeting this afternoon at the summit that I have organised.

The hon. Gentleman also asked about safeguards, which is a very important point. Although all of us share a recognition of the need to accelerate access, nothing in what is happening must in any way undermine patient trust and confidence in safety and protections. That is an important balance to strike. Nothing in what we are doing in any way looks at changing the legal basis in terms of negligence, consent or the clinical trials framework. The issue is about ensuring that our systems have the flexibilities to embrace the very latest science, and particularly, in this case, genomic biomarkers.

The hon. Gentleman asked about amending the NICE appraisal process to weight wider societal costs. At this point, the review is not specifically looking at the internal mechanics of NICE’s current high-technology appraisal process, but we are looking at giving it, with its new flexibilities and freedoms, a suite of different types of innovation that might come through. We are particularly looking at where that can be ring-fenced and targeted at particular patient groups.

The hon. Gentleman asked about a ring-fenced fund. As I said, we are looking at the allocation that we have had from the Treasury, which is about £4 billion extra on drugs in this Parliament, £2 billion of which is more or less the existing demand driven by demographic change. There is about a £2 billion allocation in there for new medicines. The difficulty is that the drugs that we might consider now to be most worthy of ring-fencing and accelerating may not be the ones that in five years’ time, on the basis of the clinical evidence, we look back on and say, “Why did we not accelerate that?”

We want to make sure, through the AAR, that we are putting in place a system that gives us the flexibilities to pull through those drugs that have the most transformational effect. But let me be clear: we are looking at wanting to build in, over the next few years, a wider understanding of the real costs to our health economies—local and national—of different forms of disease. That is why the Secretary of State and I are leading on per-patient costing so that, in due course, we can develop a more intelligent system to reflect that.

The hon. Gentleman asked about the NIHR and specialist muscle centres. This debate covers a number of different disease areas, and it is a tribute to the NIHR’s research network that more and more charities are now wanting to build centres of excellence. In the forthcoming NIHR five-year funding cycle, looking at the biomedical research centres and the biomedical research units, I am keen to make sure we consider where we can bring funding in from charities to complement that core research network.

Finally, the hon. Gentleman asked about the accelerated access review and the powers that we are looking at for NICE and NHS England. I do not want to pre-empt the findings of that independent review, but I have asked that the review looks precisely at how we can make it easier for NICE and NHS England to work more closely together. Specialist commissioning would be an obvious place to start to share those data and look at how we can get a better deal for everybody—for patients, the system and the economy.

My right hon. Friend the Member for Chesham and Amersham asked about Translarna and Vimizim and how quickly we may be able to get good news for Archie. I pay tribute to my right hon. Friend; she has been a very doughty campaigner on this matter during the last year. I share her frustration that in the existing system, due process has to be gone through and that, although we have expedited this as much as we can, it has taken a long time. I pay tribute, as she has, to Archie. He, like so many of these patients, is an inspiring example of the very best of this sector and of this country. They are people who have the most reason to complain, but tend to be the least likely to and the most inspiring, given their generosity about the system and their demand that we take their suffering and use it to make sure that others do not have to suffer.

I have touched on the timetable. I am very hopeful that we should get a decision from NICE on the basis of the secondary consultation early in the new year.

Mrs Gillan: I thank the Minister for the way in which he is still pursuing this matter on behalf of my constituent and the other boys. However, does he share my frustration? I know we have to go through due process, but why does due process have to take so long? Every day matters to these children and to their quality of life. I cannot impress enough on the Minister, NICE and anybody else watching this debate, that due process must be executed in a more timely fashion. This is nothing short of torture for these boys and these families. I know that the Minister has tried very hard, but I just hope that the people at NICE will be listening to this. I appeal to them directly through him to make a positive decision on this before Christmas; it would be the best Christmas present that these boys and their families could have.

George Freeman: My right hon. Friend makes her point as powerfully as ever. I shall not add to it; it has been put on the record very clearly.

My right hon. Friend asked about contact with the company. It is not for Ministers to get actively involved—much as, at times, I would like to—in negotiating these deals, but I have made contact with the company, both on Vimizim and Translarna, to urge it to be as flexible as it can in discussions. I can only say that I am hopeful that it will have been able to reach a point where NICE feels able to make a recommendation.

Part of the reason why due process is important is that when NICE makes a recommendation, NHS England is bound in law to provide the drug in perpetuity, so it is a major cost undertaking. In some cases, these drugs cost £200,000 or £300,000 a year, so it is a commitment of several hundred million pounds from NHS England. Other patients would say, “We must make sure that when you make a decision like that, it is done properly.” However, I share her frustration that a lot of these breakthroughs scientifically mean that we ought to be able to speed things up.

My right hon. Friend asked whether the Prime Minister is holding my feet to the fire. She need not worry; I am as passionate about this as ever and very impatient to make sure that the AAR is landed with some good recommendations.

My right hon. Friend made an excellent point about NIHR staffing. I am working with the chief medical officer and the NIHR on that at the moment. A number of our clinical research facilities could, with a few more staff, turn over more and do more trials work. There is an opportunity for us to get more people internationally to enrol in NIHR training—in clinical trials and translational research training—which would give us more capacity and allow us to move things along faster.

The hon. Member for York Central (Rachael Maskell) raised an important point about cost. I have touched on the work that we are doing on per-patient costing to try and make sure that we develop a system that more intelligently captures the real cost of disease.

I am grateful to the hon. Member for Denton and Reddish, the Opposition spokesman, for his comments. I congratulate him on the by-election victory. He asked about NICE reform, which I have touched on, through the AAR. We do not want to interfere with or undermine NICE’s independence and their “gold standard” reputation, but we want to create a place in which the accelerated access review gives them the freedoms that they are, indeed, helping to shape.

In conclusion, this debate has highlighted not only the challenges from the rising costs of new drug discovery—£200,000 to £400,000 a year for patients in the rare disease space—and the pressure on the one-size-fits-all model of assessment, but the opportunities for us to unleash our leadership in genomics and informatics to create a new landscape. That is why this week, the Association of Medical Research Charities conference and my summit this afternoon, and the accelerated access review work is creating momentum for a new landscape for accelerated pathways for patient-led innovation.

I think we will look back in two or three years at this as a crucial turning point at which the system that was set up to assess a very one-size-fits-all, 20th-century model was rapidly adapted, creating new opportunities for patient-led innovations and charities such as the CF Trust to bring through innovations that benefit their patients more quickly.

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