Watchdog deems some treatments for rare variants are 'too expensive'

Thousands of cancer patients are being denied access to costly drugs by the National Institute for Clinical Excellence (Nice), the NHS medicines watchdog, a year after ministers ordered the institute to relax its spending criteria for patients close to the end of their lives, campaigners claim.

Nice is failing to follow the "spirit" of new guidance which was supposed to raise the threshhold for costly drugs that could extend life, the Rarer Cancers Forum (RCF) said. There are hundreds of rare cancers, each affecting a small number of patients, but which together account for between a third and a half of all cancer cases. Because of their low numbers, it is more difficult for drug companies to research and produce drugs for them cost-effectively.

Last year, the Government ordered Nice to increase its cost limits for new drugs for end-of-life conditions, from an estimated £30,000 per quality-adjusted life year (Qaly) to £40,000-£45,000 per Qaly, following the "top-ups" review of funding for cancer drugs by Professor Mike Richards, published in 2008.

But 16,000 cancer patients are still being denied the drugs they need, an RCF report shows. It says Nice has also failed to speed its assessment process: appraisals of new cancer drugs still take an average of 21 months rather than the six months ministers pledged would be achieved "by 2010".

Of the 16,000 patients denied access to drugs, about half have been judged not eligible for consideration under the new end-of-life criteria. The remaining 8,000 patients, of the total of 16,000, have been considered under the higher cost threshhold but the drugs have still been rejected because they are considered too expensive for the benefits they bring. The RCF says Nice should make greater efforts to agree "patient access" schemes with pharmaceutical companies.

Last year, Nice was given the right to negotiate patient access arrangements to cut the cost of drugs to the NHS. Under these schemes, pharmaceutical companies agree to share the cost of treatment with the drug with the NHS.

In December, in one of several deals since agreed by Nice, the institute first rejected thwen approved the drug trabectedin, the first new treatment in 20 years for soft tissue sarcomawhich affects 2,000 people a year. The Spanish manufacturer, PharmaMar, had agreed to cover the costs of the drug for any patient who needed it beyond five treatment cycles. Trabectedin costs £3,500 to £5,000 per infusion, every three weeks to a month, and patients survive on average for 12 months. The deal halved the cost to the NHS.

Andrew Wilson, chief executive of the Rarer Cancers Forum, said: "Many thousands of patients are still missing out on the treatment they need. The changes introduced by Nice should be benefiting more patients." Sir Andrew Dillon, chief executive of Nice, said: "Our end of life treatments protocol, introduced at the beginning of 2009, has already made it possible for very expensive cancer treatments, which extend life for short periods, to be recommended."