INTRODUCTION

  1.  Kidney Cancer UK (KCUK) is a registered charity that was founded in 2000. Its objectives are to provide patients and their carers with improved access to reliable information about kidney cancer and its treatment, and to establish a network of individuals and groups capable of offering mutual support. It draws its membership from across the whole of the United Kingdom.

  2.  KCUK welcomes the opportunity to submit evidence relating to the work of the National Institute for Health and Clinical Excellence (NICE). For this is something about which KCUK is much exercised at the present time. Two of the Select Committee's terms of reference are especially germane to KCUK's concerns, namely the question of whether any particular groups are disadvantaged by NICE's evaluation process, and the issue regarding the speed at which NICE publishes its guidance. KCUK's particular concerns focus on the lack, so far, of any guidance on a new class of drugs used in targeted therapies for metastatic kidney cancer.

KIDNEY CANCER

  3.  There are approaching 6,700 new cases of kidney cancer in the UK each year. All but about 100 of these are of the renal cell carcinoma (RCC) type, the type that the new class of drugs is designed to combat. Some 50% of patients with RCC either present with, or go on to develop, metastatic disease which, without treatment, is usually fatal. In 2004 there were 3,300 kidney cancer deaths in the UK. The median survival once metastatic disease has developed is only 10 months (Motzer, Mazumdar et al, 1999).

  4.  Up to now systemic treatment options for metastatic RCC have been extremely limited. Standard care in the UK has been with single agent interferon-alpha, but the results from this have been very disappointing in the terms of lengthening survival. In the United States high-dose interleukin-2 is licensed by the Food and Drug Administration, but this drug is highly toxic and generates some serious side-effects. There is therefore a pressing need for more active agents; and it is to meet this need that two new drugs have been developed.

NEW DRUGS

  5.  The drugs in question are Sutent (the brand name for Sunitinib Malate) and Nexavar (the brand name for Sorafenib Tosylate). These drugs work by blocking signals in kidney cancel cells which lead to the tumor growing, spreading and developing a blood supply. Their effects have been studied in a number of large fully randomised clinical trials (Eisen et al, 2006; Escudier et al, 2005; Motzer, Michaelson et al, 2006; Motzer, Rini et al, 2006; Motzer, Hutson et al, 2006; Ratain, et al, 2005; and Motzer, Hutson et al, 2007). In summary these studies showed that the new drugs resulted in some tumour shrinkage in about 75% of patients and at the same time doubled, or more than doubled, a patient's progression free survival time. It is true that neither drug can eradicate the cancer, but they have a crucial role to play in stabilising the disease and keeping patients alive possibly long enough to benefit from other innovations in the more distant future (such as stem cell research or vaccines for kidney cancer). The other advantage of the new drugs is that they are more easily tolerated by patients, generating less serious side-effects.

  6.  Both drugs have received the approval of the US Food and Drug Admiistration and the European Medicines Agency for the treatment of metastatic RCC. They are both licensed, initially for second line treatment (ie when other treatments have failed) and later for second line treatment (meaning that clinicians can now prescribe them instead of interleukin or interferon). The view of clinicians expert in the treatment of metastatic RCC is that these drugs "should now be made routinely available in the management of this disease in the UK" (NCRI Renal Cancer Clinical Studies Group, 2006). Despite all this, neither drug has so far been reviewed by NICE. Nexavar has made it onto the list of drugs to be reviewed; but Sutent has not been accorded even that.

IMPORTANCE OF NHS FUNDING

  7.  In the absence of a "cost-effectiveness badge" awarded by NICE (or by the equivalent authorities in Wales and Scotland) primary care trusts are not obliged to fund the new drugs, even when these are recommended by the patient's clinician. In fact, under these circumstances, only a very few PCTs do fund the drugs, and then only on an "exception" basis. When the drugs are not approved for NHS funding, the consequences for individual patients can be very serious.

  8.  The new drugs work out rather expensive on a per patient basis, at something between £3,000 and £3,500 per patient per month. This is inevitable given the enormous research and development that had to be undertaken to produce them, much of the R & D effort being necessitated by the difficult nature of the disease itself. Most patients in the UK do not carry private medical insurance; and even if they did it is not always certain that the insurer will cover the cost of drugs not approved as cost-effective by NICE. Many patients simply do not have the financial resources to fund treatment for what, it can now fervently be hoped, is a considerably longer period of time. Some who have been refused funding by their PCTs have been considering some desperate measures, like selling (or at least remortaging) their homes or commuting annual pensions into immediate lump sums. But these courses of action can of course affect other members of the patient's family and consequently cannot be entered into lightly. In some cases a patient's children come to the rescue, but the magnitude of the sums required often militates against this. As things stand, there are many kidney cancer patients who could benefit greatly from the new drugs but who are not currently being treated with them. That compares most unfavourably with the position in many other advanced countries, in which Sutent and Nexavar now constitute a standard of care for RCC patients.

CONCLUSIONS

  9.  The UK is often one of the last of the advanced countries to agree funding for new drugs (not just in the case of kidney cancer but in the case of other forms of cancer as well). This seems to be partly a result of the well-meaning objective of NICE to attempt to enable equality of healthcare across the country and partly due to the intense financial pressures under which many PCTs currently operate. But whatever the reason, the relatively slow uptake of new drugs could well have something to do with the UK's position someway down the international "league table" of cancer survival rates.

  10.  Maybe not by intent, but most certainly in effect, a lack of NHS funding for Sutent and Nexavar bears spectacularly unfairly upon kidney cancer patients. After all these are patients who may not—for the most regrettable of reasons—constitute much of a charge on NHS funding. Hence, it seems only fair that when new treatments arrive promising to stabilise the disease, a little extra should be spent on them.

  11.  KCUK is calling upon the Government to make the newly licensed and approved drugs, Sutent and Nexavar, available to kidney cancer patients throughout the country on prescription and fully funded by the NHS. To this end KCUK has drawn up a petition on the No 10 Downing Street website. At the time of writing this petition has been signed by 2,638 people. The question of NHS funding is vital. Without it, more and more treatments for kidney cancer will become the preserve of the wealthy. Also, over the longer term, improvements in treatment could well lead to cancer changing from a killer disease to a chronic condition that is manageable. In other words cancer might join other chronic conditions such as diabetes, heart disease and asthma as illnesses with which people can live but will not inexorably lead to death. If the UK is to avoid establishing a two-tier system—under which the rich (and well informed) can search out and pay for the most effective new treatments, while the poor are forced to make do with second-rate care—then the process of NICE approval for NHS funding has to change.

REFERENCES

Eisen, T, et al (2006) Journal of Clinical Oncology 24 (18S): 4524

Esaidier, B, et al (2005) Proceedings of ECCO13 Paris, 30 October-3 November

Motzer, RJ, TE Hutson, et al (2006) Journal of Clinical Oncology, 24 (18S): 4343

Motzer, RJ, TE Hutson, et al (2007) New England Journal of Medicine 356 (2); 15-24

Motzer, RJ, M Mazumdar, et al (1999) Journal of Clinical Oncology 17 (8): 2530

Motzer, RJ, MD Michaelson, et al (2006) Journal of Clinical Oncology 24 (1): 16-24

Motzer, RJ, BI Rini, et al (2006) Jama 295 (21): 2516-24

NCRI Renal Cancer Clinical Studies group (2006) London, May, unpublished

Ratain, MJ, et al (2005) Proceedings of American Society of Clinical Oncologists 23:A6 2454

Dr Pat Hanlon

Kidney Cancer UK

March 2007