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Clinical Technology Appraisals (NICE)

Volume 585: debated on Monday 1 September 2014

Motion made, and Question proposed, That this House do now adjourn.—(Dr Thérèse Coffey.)

I welcome the Minister to his place. I hope that he does not feel that he has drawn the short straw by having the 10 o’clock slot. I am pleased to be here to have the opportunity to raise an issue of growing concern to NHS patients throughout the country, particularly those suffering from cancer. It will be a debate about how new drugs and treatments are approved, or not, by the National Institute for Health and Care Excellence. That organisation’s acronym is NICE, but, sadly, for many patients, the decisions it makes often seem anything but that.

Tonight’s debate is particularly timely given the announcement last week about the expansion of the cancer drugs fund. That is of course welcome, but it does serve to highlight the ongoing problem. That announcement comes on the back of a number of recent decisions by NICE not to fund important new cancer drugs. Both Kadcyla for metastatic breast cancer and Abiraterone for routine use in prostate cancer are high- profile examples, so the whole issue of the affordability of the drugs bill is back in the news, and especially how NICE carries out its cost-benefit analysis and comes to its decisions.

My original reason for raising this issue was the current review of NICE’s technical appraisals, the consultation on which closed earlier in the summer. I will make some specific points on that, but first I want to set out the problem as I see it and give some wider context. Put simply, I do not believe that the appraisal system is fit for purpose, at least not for cancer drugs. In 2011-12, NICE rejected 60% of the cancer medicines that it assessed. What makes it worse is that the rate at which they have been turned down has actually increased since 2010. That is largely because the methods used to work out the cost-benefits of each drug are too restrictive. The use of what are called “quality of adjusted life years” does not take into account many potential benefits for patients and their families. The appraisal process is also far too long—it can take a year to conclude that a new drug priced by a company at X is not cost-effective. Subsequent resubmissions by a company, based on a new price or new clinical data, can then take just as long.

Most of these drugs are already licensed for use in the UK but can be obtained only through private health care. It was that repeated failure to give patients on the NHS the same access to cancer drugs that led to the creation of the cancer drugs fund. That was a hugely welcome move, and I commend the Government for it. Because of the fund, 55,000 patients have since 2010 been able to access drugs that they would not otherwise have had. But we have to admit that the CDF is, in a sense, a sticking plaster. It was only ever envisaged as an interim measure until NICE got its act together and became more user-friendly. Even with the welcome new boost to its total funds, the CDF is scheduled to expire in 2016. That expiry date is fast approaching and many, particularly among the charities, are worried about what will happen after that date.

There is another level of uncertainty. Drugs on the CDF can be de-listed at any time. The failure of NICE to approve treatments means that the fund has been working at or above its planned financial capacity. Again, I am grateful that the Government have recognised that with the announcement last week, but the chair of the fund seemed to hint last week that time may be called on some of the drugs on the list very soon. That would be a disaster for possibly thousands of patients and their families around the country. I understand that these things need to be considered in detail, but I would be very grateful if the Minister could address this specific issue in his response. In particular, will he clarify the process and likely time scale for any de-listing of drugs?

What does this mean in practice? The Minister knows that I have a particular concern for improved treatments for pancreatic cancer patients. He may remember an Adjournment debate back in March when we discussed the new drug Abraxane. When used in combination with standard chemotherapy, it has been shown in trials to extend eligible patients’ lives by an average of just over two months, although in some cases it is significantly more.

At the time of the debate in March, I expressed my support for Pancreatic Cancer UK’s “Two More Months” campaign, which gave a number of examples of what two more months in life would have achieved for various people, and asked Ministers for the drug to be added to the CDF. Imagine my delight when that happened. As I understand it, between the end of March and the end of June this year, 118 patients have accessed Abraxane on the NHS. That would not be happening if the CDF did not exist.

Abraxane is currently under consideration by NICE for routine use for eligible metastatic pancreatic cancer patients on the NHS—a move that would make it easier for patients to access the drug. I believe that a decision on whether to approve Abraxane for use will be made in the next few weeks. However, if we use the quality-adjusted life-year system, the price of an additional two months of life looks set to be deemed too costly. Most drugs are capped at £30,000 a year, and Abraxane is estimated at slightly above £50,000.

Although NICE allows a higher cost threshold of about £50,000, that is only for drugs that meet its current end-of-life criteria, which demand that a new drug provide at least three months’ extra survival on average. The evidence so far does not show that Abraxane does that. However, if we consider that the average survival time for a pancreatic cancer patient from diagnosis is just two to six months, we can imagine that an extra two months’ survival is massively significant. Instead of having arbitrary targets in terms of months, why do we not look at percentages of the average survival rate as a means of dealing with these more difficult cancers and diagnoses?

If Abraxane is rejected by NICE, as I fear it might be, we will be back to relying on the cancer drugs fund to provide it. It will end up like other treatments that are available for pancreatic cancers, only one of which has been approved by NICE. Non-drug alternatives such as NanoKnife and CyberKnife, which have been discussed in this Chamber, are in use in private practice but have not been approved by NICE for clinical use on the NHS. Patients are forced to spend tens of thousands of pounds of their savings to access those treatments privately.

We are in a position where new treatments might start to make a small difference in survival rates for pancreatic cancer—there have been no improvements in those rates over the past 40 years—but such improvements will have been despite, not because of, NICE. That must change.

Indeed, change is now in prospect. NICE is currently looking at introducing a new way of assessing drugs, called value-based assessment. The consultation on it closed in June and I understand that some next steps are due to be announced very soon.

I have looked at the proposals, but I must say to the Minister that there are worries that the new system will not improve the current situation. Briefly, my concerns are these. First, the proposals maintain a quality-adjusted life-years system as the basis of an appraisal. That will still mean that many benefits to patients and their loved ones highlighted by patient groups might not be taken into account. Secondly, it is proposed that specific end-of-life criteria be removed and incorporated in a wider “burden of illness” measure. There is no guarantee that this measure will capture the unique requirements of end-of-life drugs. Thirdly, the way the proposals are framed could well mean that the age of patients with a particular condition could count against the drugs, which is a particular concern given, as everybody knows, that the likelihood of getting cancer increases with age. Perhaps more importantly, I see nothing in the proposals that suggests that the new appraisal process will be any quicker than the current one, and time is one thing that many cancer patients do not have.

I have serious reservations about the proposals. The crucial question is this: will the new system make more cancer treatments available or fewer? If there were confidence that the new set of criteria would solve the problem, we might not be facing the need for extra funds, but as things stand, the extra CDF money remains the lifeline. If the new system does not solve the problem and the CDF ends in 2016, we will be back to square one, with thousands of patients not getting the drugs they need and deserve—drugs which over the past four years have been proved to make an immense difference to patients’ and their families’ and friends’ lives.

These fears are shared by many cancer charities and patients groups. I hope that their responses to the consultation will be given the consideration they deserve. If they are, it should be possible to devise a system that genuinely works. That means a system that works faster. The NICE process is extensive, but it takes too long. Why on earth when a drug is rejected at one price does the whole elongated process that I have mentioned have to start again when it is resubmitted at a new price? There is an argument that that simply encourages companies to pitch a high price to begin with, and therefore creates further delays as the argument goes on to get the price down. Why can we not have a system where there are sensible negotiations between NICE and the drug companies immediately after a drug is licensed, along the lines of systems used in many continental countries?

The system should also give more weight to patients’ and carers’ needs and experiences. At the moment, the process is almost exclusively focused around clinical effectiveness—that is understandable—and value for money. Patient engagement is ostensibly taken into account, but it needs to be given a higher priority. The Scottish Medicines Consortium has just changed its system along those lines, and that might be a good model to consider.

Lastly, a specific end-of-life weighting really must be maintained in the new system. It cannot, however, be so prescriptive that it excludes new treatments for cancers with extremely poor prognoses, such as pancreatic cancer. NICE’s current three-month rule makes that a specific problem that the new system must tackle.

To sum up, of course the NHS does not have an unlimited budget—we all understand that—and it is right to seek value for money for taxpayers. Drug companies must also be realistic when pricing their drugs, but as I have said, the current system provides incentives for them to pitch high. This review of appraisals gives us an opportunity to make them work better. We urgently need reform because for too many cancer sufferers NICE simply is not working.

Until that improved system is in place, the cancer drugs fund will remain vital, and the extra money will be well spent. I hope that it will be extended beyond 2016 to make sure there is at least one fast and effective route for cancer drug approvals. However, that can only ever be a temporary fix. There must be a more reliable long-term system to get cancer patients the treatments that they so desperately need. Such decisions can literally be life-and-death ones, and too often we are not getting them right. Cancer need not be a death sentence, but the rejection of a drug based on a flawed assessment might be one.

For many patients, extra days and months are not just numbers in a cost-benefit equation, but precious moments with loved ones. NICE must recognise that the timeline is arbitrary, and it must start to build on the success of the cancer drugs fund.

May I say what a pleasure it is to start my role on the Front Bench under your careful guidance tonight, Mr Speaker?

I pay tribute to my hon. Friend the Member for Lancaster and Fleetwood (Eric Ollerenshaw) for his tireless campaigning on this subject, his leadership in the House and his work on the all-party group on pancreatic cancer. He has been a tireless campaigner on behalf of patients affected by pancreatic cancer, which is a terrible disease. I know that the subject is very close to his heart, having been affected by the tragic loss of his partner from pancreatic cancer. I applaud him and pay tribute to him for the work he has done. I pay tribute to all the patients and charities who are so active in this area, and I assure him and all those affected that we want to see the best possible outcomes for all NHS patients and loved ones, including those with pancreatic cancer. As is so often the case, the impassioned advocacy of patients and their loved ones forces the system to address the priorities of the patients whom we are all here to serve.

It is vital for people, especially those affected by long-term and life-threatening conditions, to have access to new and promising treatments. That is one of my key priorities in my new role. I well understand how disappointing it is for patients and carers when NICE does not recommend a particular drug or treatment for which they have held out such hope. A central part of my mission as the UK’s first Minister for life sciences—an opportunity that reflects the Government’s commitment and the Prime Minister’s personal commitment to this agenda—is to accelerate the discovery, design and adoption of new drugs in the NHS, making the UK the best place in the world to discover and design 21st-century health care technologies.

My hon. Friend raised a number of important points that I want to address. In particular, he asked whether the appraisal process of the National Institute for Health and Care Excellence is fit for purpose and spoke about the time that is taken over the approval of new drugs and the importance of the cancer drugs fund. He made a number of specific references to treatments, not least to Abraxane. I will reflect on those references and write to him so that I have time to address his substantive points.

I will start by taking a step back to reflect on the context in which NICE works, including the pressures and opportunities that the extraordinary pace of biomedical discovery is unlocking. In an age when so many new drugs and treatments are being discovered because of the extraordinary success of our scientists and our life sciences industry in bringing innovation to the market, and in a world in which more people are living longer and in which our resources are under ever more sustained pressure, it is no surprise that we hear with increasing frequency stories of new drugs and of NICE’s decisions to restrict their use to smaller groups of patients.

As my hon. Friend is all too aware, we, like other Governments across the globe, face a great challenge to ensure that every pound of health service money is well spent on effective treatments. The NHS does not have an infinite budget. As we make significant advances in these areas, which in turn will bring more treatment options, the demands on those resources will increase. The discovery of new treatment options is great news, but it brings new challenges. That is particularly relevant to cancer, for which a number of new high-cost drugs have arrived in recent years. Some of those, despite their cost, offer only marginal improvements in quality and length of life. I recognise, as my hon. Friend pointed out, that for patients who are dying, a few extra months provide precious extra time for them and their loved ones. The challenges is to find an equitable way of dealing with the difficult ethical, medical and economic judgments that we have to make. That is where NICE fits in.

Over the years, this country has, through NICE, led the way in developing an evidence-based assessment system to determine how drugs and treatments are best used in the NHS. Initially set up in 1999, NICE has played an invaluable role in providing internationally respected guidance to the NHS on the clinical and cost effectiveness of drugs and treatments. Those are difficult decisions to make. NICE reaches its final decision on the use of a particular technology only after careful and thorough consideration of all the available evidence and following extensive consultation with stakeholders.

The Government firmly believe that cost-effective, clinically appropriate drugs and devices should be routinely available to NHS patients. That is why NHS commissioners are legally required to fund treatments that are recommended by NICE’s appraisals, why the right of patients to NICE-recommended drugs and treatments is enshrined in the new NHS constitution, and why we are acting to promote the rapid uptake of NICE-recommended drugs in the NHS. Many thousands of people in England have, as a result, benefited from the treatments that are recommended by NICE, including for cancer. I am pleased to note that so far this year, NICE has recommended five new cancer drugs on the basis that they are clinically effective and cost effective.

However, the rapid development of breakthroughs in genomics, informatics and new diagnostics means that NICE’s processes will have to adapt. Since it was established in 1999, NICE’s internationally renowned technology appraisal process has evolved constantly to meet new challenges. I am confident that it will continue to do so—it will need to if it is to keep its place at the vanguard of international health technology assessment.

The UK and NICE led the world in the late 20th century in pioneering the health economics of drug discovery. However, the breakthroughs in genomics and clinical informatics are transforming the way in which drugs are discovered and are allowing us increasingly to design drugs around patients and their tissues, data and genomic and phenotypic history. That is unlocking a new era of targeted and, ultimately, personalised medicine. Drugs may be designed for patients in whom we know they will work. That will allow us to change the way in which we procure and provide reimbursement for drugs—from payment on performance to outcome-based payments.

We are at the dawn of an exciting new age of more accurate, effective, specific and targeted medicines. We no longer expect to give every drug to every patient. We do not expect the industry to bring drugs to us that can be proven to work in every single patient. We will be able to unlock huge efficiencies in the NHS’s £12 billion drugs budget through a much more targeted model of medicine, and it will mean a radically different mission for NICE in the coming years. Accelerating this country’s leadership in that area is central to my appointment, and I am delighted to be able to take the opportunity to spell that out.

I note my hon. Friend’s concerns about the length of time that it can take for new drugs to be made available. I very much recognise that as both a core barrier to making the UK the best place in the world to develop new medicines and a great opportunity. I should like to speak a little about a particular case that illustrates the importance of early access.

I recently met, and got to know, a leukaemia and lymphoma patient who was diagnosed 10 years ago with chronic myeloid leukaemia. He was in his early 40s and had a young family, and he was given about three years to live—10 years ago. He is alive today because he was one of just three patients placed on a clinical trial by his oncologist at the university of Birmingham hospital back in 2001. The trial, of Glivec, now one of the most commonly used CML drugs, was his lucky break. He was one of the first patients to receive a groundbreaking new treatment, giving him an extra decade with his young family and now a clean bill of health and a life that he is using as a pioneering campaigner for the revolution of targeted medicine.

Of course, it is important that any new medicine is shown to be effective and safe before it is used routinely within the NHS, but I believe that we need to do much more to help the patients in the direst need to access pioneering drugs more quickly so that luck does not come in to it. That is why the Prime Minister and I have committed to the early access to medicine scheme, which we launched earlier this year. That UK-only scheme addresses unmet need on an unlicensed or off-label basis for patients with life-threatening or seriously debilitating conditions who do not have adequate alternative treatments, by supporting access to very promising new treatments long before we would normally expect to receive them through the usual approval process. We have much more work to do, but I am pleased to see that the UK medicines regulator, the Medicines and Healthcare products Regulatory Agency, is now starting to receive applications for the scheme. I am also pleased to say that pioneering life science companies have pledged their support and made applications to the European Medicines Agency’s adaptive licensing pilot. NICE has also been involved in discussions on both those initiatives, along with NHS England, to ensure that licensing decisions translate into patient access. The central aim of the early access to medicines scheme is to create a new fast-track runway to get a new generation of innovative medicines to the patients for whom there is no alternative treatment, to give them a chance and some hope that their suffering might help to prevent suffering for future generations.

My hon. Friend also raised the important issue of the length of the NICE appraisal process. I appreciate how important it is, when new drugs are licensed, that the NHS has good guidance on their use as quickly as possible so that clinicians can make the best possible use of them. In drug discovery, time is money, and accelerating assessment and approval is the single most important reform that we have to make. I am absolutely committed to it. In fact, I met NICE’s chief executive and chair today to highlight that point. For me, tonight’s debate marks the beginning of a conversation about the new model for NICE and our work with it on that model. NICE is an independent organisation, and it is not for me to second-guess what its final proposals will look like, but we have made it clear in the terms of reference that the end of life will continue to be given important consideration. There will certainly be no unlawful discrimination.

I mentioned genomics. Genomic technologies are allowing us to make groundbreaking discoveries about how disease works, how different patients become susceptible to different diseases and how we can diagnose disease earlier, treat it and ultimately prevent it. That is why the Prime Minister and I recently announced a £300 million investment to make the UK the world leader in genomic medicine. For the first time in the world, 100,000 full human genomes from volunteers will be sequenced by 2017. It is the most ambitious project in biomedicine in the world, and it is a world first. The development of genomically informed medicine will lead to faster diagnosis, with knowledge based on genomic technologies being used to better target treatments and get the right patients the right treatment faster than is possible today. I do not think it is an exaggeration to say that it has the potential to transform how we evaluate medicines, the benefits they deliver, who they deliver them to and how effectively. It will help bring to an end the averaging of outcomes, which sits at the heart of the current NICE model, allowing us to work out how to give the right drugs to the right patients much quicker than is currently possible.

My hon. Friend mentioned the cancer drugs fund and the benefits that it has brought to many patients and their loved ones. More than 55,000 patients have benefited from the fund since October 2010, and I am delighted to be able to confirm that the Prime Minister, the Secretary of State for Health and I are all committed to the renewal of the fund. Indeed, we announced last week that an additional £160 million would be made available to the fund through to March 2016, which will allow patients to continue to receive the extra drugs that are right for them.

We also announced plans for NHS England, NICE, charities and industry to work together to improve the way cancer drugs are commissioned. We must make better use of the cancer drugs fund to generate better data on new drugs, so that we can address the uncertainties that so often affect early clinical trial evidence, and help NHS patients and clinicians to understand the real world value of those drugs—indeed, today I met Harpal Kumar from Cancer Research UK to discuss that point. I note my hon. Friend’s concerns about future access to cancer drugs, and will carefully consider with NHS England what arrangements should be put in place for the fund in the longer term.

Central to this quiet revolution is patient empowerment, and 21st-century health care will move from being something in the 20th century that was done to patients by the Government when they deemed it appropriate, to something that 21st-century citizens will be entitled to and empowered to take more responsibility for themselves. In access to research and drugs, access to outcomes data, and people’s ability to shape their health choices, we are committed to accelerating that quiet revolution.

Ensuring patients have more say in how their care is delivered and embedding choice are key themes in the Health and Social Care Act 2012, and I intend to accelerate that in the field of health care technology. This quiet revolution is what the Government’s life science strategy sets out to frame and accelerate. That is why we have created a new Office for Life Sciences, bringing together officials from the Department of Health, the Treasury, and the Department for Business, Innovation and Skills. There is a new directorate in the Department of Health to accelerate innovation, and a new ministerial post, which it is my honour to hold. A package of other measures will help to make the UK in the 21st century once again what we were in the early 20th century—the world leader in medicines discovery.

Twenty-first century health care is being utterly transformed by breakthroughs in genetics, informatics, diagnostics and the new field of targeted medicine. It will transform the way we design and procure new medicines, and the way that NICE assesses their costs and benefits. I intend to ensure that this country seizes the opportunity for the benefits of our children, so that Britain is once again the best place in the world to discover new medicines and to be treated by them.

I congratulate my hon. Friend again on his advocacy and the important issues he has raised tonight, and I will write to address his specific points in detail. I look forward to working with him and the many other groups in Parliament and outside who are increasingly active in that space, to ensure that his partner, and many hundreds of thousands of others like him, have not died in vain.

Question put and agreed to.

House adjourned.