Rarer Cancers

John Leech Excerpts

Wednesday 27th October 2010 Westminster Hall

[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.