Rarer Cancers

Wednesday 27th October 2010 Westminster Hall

15:58

[Mr John Leech (]

Manchester, Withington) (LD): It is a pleasure to see you in the Chair this afternoon, Mr Robertson. I start by thanking my hon. Friend the Minister for contacting me in advance of the debate for an indication of the matters that I want to raise on a wide-ranging subject. The title is particularly wide. I also thank the numerous organisations that contacted me with information for the debate and apologise to those whose issues will not be addressed this afternoon.

In this short debate, I want to make some time available to my hon. Friend the Member for Gillingham and Rainham (Rehman Chishti), who wants to say a few words, and to take the opportunity to raise two specific issues, the first of which is lack of awareness among MPs of the issues surrounding funding for the treatment of rarer cancers in general and the lack of understanding of the complex processes and institutions that make decisions about the availability of drugs, especially to sufferers of rarer cancers.

The second, related, topic is the recent health technology appraisal by the National Institute for Health and Clinical Excellence of the use of azacitidine for the treatment of myelodysplastic syndrome. That is a bit of a tongue twister, so I shall refer to it as MDS. NICE’s appraisal and decision-making process for azacitidine highlights the problems and inequalities facing sufferers of rarer cancers in getting access to what are often life-saving treatments. NICE’s refusal to recognise orphan drug status has created a great disparity in the treatment options for the sufferers of rarer cancers. With the move to value-based pricing not due to come into effect until 2014 and with the lack of details about how it will work, it is essential that that inequality is addressed.

The initial £50 million of funding from the cancer drugs fund has been distributed to the strategic health authorities, and today sees the opening of the consultation on the main cancer drugs fund. I suggest that today is the perfect day to restate the case for far greater funding for rarer cancers.

I start by mentioning the need for MPs to have a greater awareness of rarer cancers, and of the processes and institutions that evaluate the drugs needed to treat them. A number of patients and their family members attended the MDS UK lobby of Parliament yesterday. As part of that lobby, MDS UK launched the results of a survey, which showed that two thirds of MPs had no understanding of the term “health technology assessment”, and one third had no understanding of the term “quality-adjusted life year.” I confess that I was one of those people until I was contacted by MDS UK.

MPs are expected to have a broad knowledge of many subjects, but we cannot claim, or expect, to have a detailed knowledge of all subjects—some of our constituents may argue that we do not have much knowledge of any subjects. With large-scale reforms of the NHS due to be discussed and voted on, it is vital that MPs gain a greater knowledge of such subjects, and understand how those who suffer from rarer forms of cancer are currently discriminated against.

There is a need for greater awareness about how the details of value-based pricing and decisions on how to allocate the cancer drugs fund may, or may not, remove barriers to treatment for those with rarer cancers. I do not intend to spend any more time on that aspect of the debate, other than to highlight the rarer cancers toolkit collated by MDS UK, which I hope the Minister is aware of and has seen. Although today I am concentrating on MDS, there are other rare cancers such as pancreatic cancer, ovarian cancer or mesothelioma that also struggle with either a lack of access to drugs, or with the need to raise awareness among the general public.

I want to mention the fiasco surrounding the NICE appraisal of azacitidine. The process has already taken 18 months and, as a solution is not necessarily assured until March 2011, it may end up being a two-year process in total. In the meantime, up to 1,500 patients will have missed out on the opportunity to access azacitidine during that decision-making process.

The mishandling of the evidence by the evaluation team, as highlighted by the appeals committee, has not helped. The process is long-winded and ponderous even without an appeal. More worryingly, setting aside the length of time involved, the decision-making process itself is flawed, especially NICE’s refusal to recognise orphan drug status. The MDS Forum, the Royal College of Pathology and the British Society for Haematology, which collectively represent the UK’s top haematologists, have all written to NICE to highlight the unsuitability of NICE’s health technology assessments for considering orphan medicines. Haematologists from centres of excellence across Europe have signed a letter echoing that view. Of the 50 orphan-status drugs that are licensed by the European Medicines Agency, only three have been recommended by NICE.

There is an exceptionally strong case for further reform to the NICE process to ensure that it captures the full value of new and innovative medicines for patients with rarer cancers. It is widely accepted that there are problems with the NICE process and that it is unfair to those who suffer with rarer cancers. Extremely high costs are associated with the research and development of drugs, and the relative lack of expertise means that drugs for rarer conditions cost even more. The fact that some drugs are for small patient groups means that sales are relatively low, and the unit cost of such drugs is incredibly high. Nevertheless, the innovative value added by those drugs is great. They are not “me too” drugs, with a relatively low incremental clinical improvement to the patient compared with the drug that is replaced. Such drugs are unique and often provide the only treatment available to the patient. Azacitidine for MDS is a perfect example. The only alternative to it is usually “best supportive care”, which treats only the symptoms and not the disease.

I do not know why the process cannot be adjusted to take orphan status into account, and I would be grateful if the Minister could address that question in his response. It seems that the UK is almost unique in that decision. The problems with the NICE process are not unknown, and I know that the Minister and his Department are fully aware of them. The decision to move to value-based pricing, and the introduction of end-of-life criteria and patient access schemes are recognition of such problems. However, end-of-life criteria and patient access schemes have not succeeded in making appraisals more flexible to innovative treatments for rarer cancers, especially cancers where the small number of patients eligible to take part in trials means that there is often considerable uncertainty surrounding the data.

An interesting example of the current problems with NICE’s methodology is the fact that even if azacitidine were to be given to the patient for free, its quality-adjusted life year cost would still be £20,000. I do not understand how that can be the case; perhaps the Minister will explain.

Unfortunately, value-based pricing is more than three years away. That will be too late for some patients. Furthermore, the devil will be in the detail as to how the scheme will work and how effective it will be at addressing the needs of small patient groups. In the meantime, short of further reform of the NICE process, the only option for MDS patients and similarly disadvantaged sufferers of other rare cancers is the cancer drugs fund.

Before I move on to the cancer drugs fund, I urge the Minister to ensure that value-based pricing is constructed in such a way that drugs such as azacitidine become available to UK citizens, just as they are available across the rest of Europe. Azacitidine provides a vast improvement to patient outcomes compared with current treatments, and it is therefore the very definition of “high value”, which value-based pricing must be designed around.

I urge the Department of Health to ensure that the cancer drugs fund is used to help sufferers of cancers such as MDS. MDS is a life-threatening illness and it is terribly served by the NICE process. The only available treatment is currently being denied to patients, and although I hope that NICE reverses its decision, I fear that ministerial intervention may be needed to bring access to azacitidine to MDS sufferers. It is exactly the kind of treatment that the cancer drugs fund was designed to support and must provide for. Of course, MDS sufferers will have to wait even longer while NICE makes its decision, before they are even allowed to apply to the fund.

In conclusion, I urge the Minister to ensure that NICE methodology is reformed to make it more suitable for assessing medicines for rarer cancers, or that cancers such as MDS are taken out of the NICE health technology assessment process. I request value-based pricing to be constructed in a way that ensures that innovative drugs with a high benefit to patient outcome, such as azacitidine, are correctly appraised. In the meantime, I ask for the cancer drugs fund to be used to correct the inequality faced by sufferers of rarer cancers due to the disparity in the appraisal process of drugs for those rarer cancers.

16:09

[Rehman Chishti (Gillingham and Rainham) (Con)]

It is a pleasure to speak under your chairmanship, Mr Robertson. I thank and congratulate the hon. Member for Manchester, Withington (Mr Leech) on securing this debate on such an important and sensitive issue. Applying the basic principle that brevity is a virtue, not a vice, I shall keep my submission short. I place on the record my thanks to organisations such as Macmillan Cancer Support, which have been very helpful in providing information for me to consider and base my submission on.

My first point is that representations have been made to me that people who suffer from rarer forms of cancer should be given priority in the Government’s proposals for the new drugs fund, on the basis that they are the ones who are the most disadvantaged by the current NICE drug appraisal system.

Secondly, the services available through the clinical nurse specialist system are very unlikely to be given to people with rarer forms of cancer. I urge the Minister to consider extending that system and ensuring that people with rarer forms of cancer have access to it. That is especially important because new research shows that clinical nurse specialists save the NHS money by co-ordinating services more effectively, along with significantly improving the patient experience and outcomes.

Thirdly, I seek an assurance from the Minister that the full maximum of the money allocated in the coalition agreement to the cancer drugs fund will be available. It is vital that we ensure that people who need those drugs get them at the earliest opportunity.

16:11

[The Minister of State, Department of Health (Paul Burstow)]

I am grateful for the opportunity to respond to the debate. I congratulate my hon. Friend the Member for Manchester, Withington (Mr Leech) on securing it and I particularly thank him for giving me notice of the focus of the debate, because, as he rightly says, the range of issues that could be covered in a debate on rare cancers is very wide. I hope that my remarks will therefore address his particular concerns.

The argument that my hon. Friend makes about the iniquities facing people with rare cancers is a clear riposte to the challenge posed by those who say that the NHS needs no reform because it works perfectly. That is often said to me, but my hon. Friend has set out a compelling case why there are areas in which scrutiny, change and reform are undoubtedly necessary. In fact, there are significant failings in the current system of drug pricing and access. The Government are determined to put them right so that we can help more people to get fair access to drugs and treatments that will help them.

In that regard, my hon. Friend rightly draws attention to today’s launch of the cancer drugs fund consultation. It is an important moment for the 27,000 people diagnosed with rarer cancers each year. The £200 million a year investment that we shall be making from 1 April 2011, on top of the £50 million that started to go into the system from 1 October this year, means that the NHS will be in a position to provide more support than it has been able to in the past. We will ensure that more patients get drugs and treatments not otherwise available on the NHS.

The Rarer Cancers Foundation has said that our interim funding for the cancer drugs fund will benefit more than 2,000 cancer patients this year alone. Clearly, the £200 million investment that we are making from next April will benefit thousands more. My hon. Friend—

[Emily Thornberry (Islington South and Finsbury) (Lab)]

Will the hon. Gentleman give way?

[Paul Burstow]

I am afraid not, because in these debates one has to have obtained the permission of the hon. Member who secured the debate and then the Minister. For that reason, I cannot give way.

[Emily Thornberry]

Please excuse my inexperience. I have just one question—

[Paul Burstow]

I would be more than happy to discuss it with the hon. Lady after the debate.

My hon. Friend is right to stress that we need to ensure that the fund and the eventual value-based pricing approach are accessible and intelligible. He is right to challenge all of us to test our own understanding of QALYs and technology appraisals. My officials did supply me with a plain English version of what QALYs are, but I will not detain hon. Members by reading it out today. My hon. Friend is right to say that MPs need to understand the detail of how the new cancer drugs fund will work in practice, so that they can advise constituents effectively and their constituents can make the maximum use of it.

The rarer cancers toolkit, which my hon. Friend described, contains a lot of useful information to help MPs to understand cases brought to them by their constituents. I place on the record my gratitude to MDS UK for taking the time to develop it. I hope that it is a successful toolkit that is widely taken up and used by colleagues in the House.

In addition, today’s consultation launch is an ideal opportunity for hon. Members to interrogate the proposals, offer constructive challenges and add their own thoughts on how we can take the issue forward. I certainly encourage my hon. Friend and the hon. Member for Gillingham and Rainham (Rehman Chishti) to consider it in more detail and be prepared to comment in more detail. I will ensure, however, that the comments made about it today are fed into the consultation.

I was asked a specific question about the role of clinical nurse support. Every cancer patient should have access to appropriate care, support and information. We are considering the evidence to see whether one-to-one nurse specialist support can improve care for cancer patients, as well as ensuring that there is efficient use of NHS resources. We shall say more about that as we publish our NHS cancer reform strategy review later this year.

My hon. Friend rightly highlighted value-based pricing as one of the ways in which we can address a number of the issues. The cancer drugs fund provides us with a bridge to that more sustainable system, which we hope to have in place from 2014, when the current pharmaceutical price regulation scheme ends. Our intention is to develop a system that makes a much closer link between the price that the NHS pays and the value that a new medicine delivers. That is the key to securing better access to medicines for patients and a better deal for the taxpayer.

Developing a new medicines pricing system is clearly not a simple task. It is important to ensure that when the new system comes in, we get it right. There is international experience on which we can draw, but it will be groundbreaking work and we shall want to engage with stakeholders to ensure that we get it right. I agree with my hon. Friend that the new pricing system must be carefully designed so that it provides fair outcomes for all patients, including those with rarer forms of cancer.

There is, though, a balance to be struck, as I am sure my hon. Friend would acknowledge. The pricing model needs to support innovation in the pharmaceutical sector; it needs properly to reward research and development in respect of new treatments. We need to ensure that the incentives are there to encourage firms to invest in the future treatments that will fulfil unmet needs and save and improve people’s lives. However, we also need to deliver better value for the taxpayer. In the current financial climate, that is even more the case.

My hon. Friend makes valid points about the unit costs of drugs for rarer conditions and how we tally up the development costs with the relatively low sales of those drugs. I will ensure that his comments about those matters are fed into the future discussions about the design of and consultation on value-based pricing of drugs. Later this year, we shall publish a consultation document, setting out our plans in detail. I hope that he and people from the many charities representing those with rarer cancers take part in that consultation and help us to shape the system in a way that makes a difference for those whom they represent.

My hon. Friend made comments about the treatments available for MDS; indeed, he said that none was available. I am informed by officials that some people with low or intermediate-risk MDS and no symptoms may not need treatment, but they will be monitored. Most MDS patients will have treatment to control or improve the symptoms. That is called supportive treatment. Chemotherapy may be helpful for some people, depending on the type of MDS that they have. A small number of patients may benefit from stem cell transplants, and the aim in those circumstances is to cure the disease. There are things that are done, but I accept my hon. Friend’s general point about the need to have access to appropriate drugs as well.

Let me answer the points that my hon. Friend raised about NICE. NICE performs a very difficult but essential job. Its independence is a key strength in the system. It should not be subject to micro-management and meddling by Ministers on a case-by-case basis. For that reason, I will not be able to give my hon. Friend the comfort that he wants—a ministerial intervention to direct NICE to behave differently with regard to the particular treatment under discussion. However, I note my hon. Friend’s criticism of the way in which the appraisal has been handled. That is the very reason why we have to have within it the appeal mechanisms that exist. That appeal mechanism has allowed some of those issues to be flagged up and reconsideration to take place.

I appreciate what my hon. Friend has said and his frustration, which I suspect is shared by many hon. Members and, more importantly, by those hoping for access to the drugs, about the length of time that it can take to get an appraisal through. NICE has attempted to streamline its appraisal process in recent years and, in many cases, is now able to start work before a new drug is licensed. For the majority of new cancer drugs, NICE aims to publish draft or final guidance within six months of licensing.

The process tends to be more protracted when there is an appeal against the decision, as there was in that case, or when there are particularly complex issues surrounding the drug in question. While we would welcome greater speed, none of us would advocate dispensing with the vital features of NICE’s work, such as public consultation and an independent appeal stage.

It is worth pointing out, however, that people can access the new funding for cancer drugs before NICE makes a final decision. Let me be absolutely clear: in other words, people do not need to wait for NICE to say no before they can apply to the cancer drugs fund. We have also made it clear to primary care trusts that they should not refuse to fund a drug locally simply because no NICE guidance is yet available.

I am also aware of the problems about providing drugs for which there might be limited evidence of effectiveness. Again, today’s consultation is asking about and seeking comments on that, to see how the fund is developed and what advice we give to the members of the panels that make the decisions. We are open to ideas and, through this debate, I put to my hon. Friend and others the idea of participating in the consultation.

Turning to orphan status drugs and drugs appraisal, my hon. Friend rightly drew attention to the fact that NICE has recommended only three of the 50 orphan drugs licensed by the European Medicines Agency. I understand his point, but we need to put his point into context. NICE has issued final guidance on, I believe, only seven drugs that have a current orphan designation, recommending only three of them, including one drug for more than one indication. NICE also issued positive guidance on the drug Sutent, which was an orphan drug when designated, but has since had that designation withdrawn.

My hon. Friend asked why NICE approval processes could not be adjusted to take into account orphan status. I point out, as he rightly mentioned, that the end-of-life criteria give NICE flexibility in appraising life-extending drugs for patients with terminal cancer. One of the criteria that must be satisfied for NICE to apply those extra flexibilities is that the treatment must be licensed, or otherwise indicated, for small patient populations. He suggested that such flexibility has not been helpful with orphan drugs, but it has helped make a number of drugs available to patients, including orphan drugs such as Yondelis for soft-tissue sarcoma and Revlimid for multiple myeloma.

I know that NICE is always open to ideas and I encourage my hon. Friend to engage directly with it, along with MDS UK, about their concerns. I will certainly ensure that a transcript of the debate is passed to NICE, so that it can see for itself the concerns of my hon. Friend.

To sum up, such issues are incredibly difficult and emotive. I know that from my own constituency mailbag and from constituents who come to see me, battling to get the medication that they think is appropriate for them on the advice of clinicians. The cancer drugs fund begins to transform and unlock new opportunities in that regard. Decisions concerning treatments often affect patients’ chances of beating their cancer. That and the possibility of patients having more time with their families are the very motivation behind the drugs fund.

The Government are clear. We need a system that treats people with rarer cancers in a fairer way, as my hon. Friend said today. He offered us a helpful and insightful spur to ensure that we have a clear focus as we progress in developing policy in the area. The fact is that many thousands of people have lost out over the years, owing to how cancer drugs are priced and funded. Starting with the £50 million already made available this year, and continuing with the £200 million cancer drugs fund from next April, we want to end that unfairness. More people will be able to get the drugs that they want to extend or improve their lives.

I hope that my hon. Friend has found the debate useful. We must ensure that the system works around the needs of patients and is evidence-based and properly accountable. I look forward to continuing the discussion. I hope that he and others will respond properly to the cancer drugs consultation and our work around designing the value-based pricing system to come.

16:24

Sitting suspended.