UK NICE to expand use of life-extending drugs for the terminally ill – Update
This article was originally published in SRA
The UK’s health technology assessment body has revised its policy to provide terminally ill patients suffering from rare, incurable diseases with wider access to costly life-extending medicines under the National Health Service1,2. Experts estimate that some 10,000 cancer patients a year could benefit from the move3.
The National Institute for Health and Clinical Excellence, which relies on cost-effectiveness as its main criterion in its health technology assessments, issued new supplementary advice for its appraisal committees to consider when appraising treatments for patients with a life expectancy of less than two years.
Treatments with an incremental cost-effectiveness ratio of more than £30,000 per quality-adjusted life year usually do not pass NICE’s cost-effectiveness criterion, although there have been some exceptions.
The latest guidance states that it may be appropriate to allow treatments beyond this upper limit for terminally ill patients if there is evidence that the treatment would extend the patient’s life by at least three months in comparison with treatments presently available on the NHS.
The new policy, NICE said, will allow appraisal committees to support the development of innovative treatments for small groups of patients suffering from incurable diseases. The institute wants the Department of Health to collect data on drugs assessed under the revised criteria to ascertain the extent to which the anticipated survival gains are evident when the treatments involved are used in routine practice. “The outcome of this exercise will be evaluated when the guidance for that treatment is reviewed,” it said.
For its part, NICE plans to carry out a methodological evaluation of how the supplementary advice is applied by its appraisal committee to ensure that it achieves its intended purpose. The results, to be made public, would be used to make modifications to the guidance document in this regard, if necessary.
Background
NICE’s “impassionate approach” to the drug appraisal process drew widespread criticism from patient advocacy groups and academics in August 2008, after it issued a draft appraisal report on four cancer drugs terming them as clinically effective, but not cost-effective4. In response, the government announced new measures in November to improve patient access to medicines, allowing them to pay for expensive medicines privately without losing their NHS cover5.
Concurrently, the institute launched a consultation process with proposals for greater flexibility in appraising treatments for patients with short life expectancy and which are indicated for use in small populations. The majority of the 850 comments that NICE received from 300 respondents said that rejecting proven life-extending treatments on the grounds of cost-effectiveness was not acceptable.
Reactions
James Raftery, professor of health technology assessment at Southampton University, suggested that the new policy adopted by NICE would do little to improve availability of expensive drugs6.
Dr Raftery examined the effect of the new policy on the 11 cancer drugs turned down by NICE between 1999 and 2008 on grounds of cost-effectiveness. Few of the drugs provisionally refused would qualify under the new policy, he found, as most failed to meet the criterion that no alternative treatment with comparable benefits should exist.
Advocacy group Rarer Cancers Forum welcomed NICE’s decision to allow greater flexibility when considering life-extending treatments for patients with rare incurable diseases, such as those for advanced cancer7.
“However, there are many treatments which can never be assessed by NICE due to the extreme rarity of the cancers they can treat,” the group noted. “Patients with these cancers will still be forced into the inconsistent and demeaning ‘exceptional case processes’ unless action is taken. To address this inequality, the government needs to explain how these patients can receive the life sustaining treatments they need without relying on top ups.”
Cancer Research UK’s policy manager Hilary Jackson said flexibility in NICE’s appraisal process would mean patients with rare diseases could gain crucial extra weeks or months of life.
References
1. NICE, Appraising treatments which may extend life at the end of life: Consultation response, 2 January 2009, www.nice.org.uk/aboutnice/howwework/devnicetech/endoflifetreatments.jsp
2. NICE, Supplementary advice to the appraisal committees, 30 December 2008, www.nice.org.uk/media/88A/F2/SupplementaryAdviceTACEoL.pdf
3. Cancer Research UK, News, 6 January 2008, http://info.cancerresearchuk.org/news/archive/newsarchive/2009/january/18957283
4. The Regulatory Affairs Journal – Pharma, 2008, 19(10), 703-704
5. The Regulatory Affairs Journal – Pharma, 2008, 19(12), 853-854
6. BMJ, 2009; 338:b67, http://press.psprings.co.uk/bmj/january/NICE.doc
7. Rarer Cancers Forum press release, 2 January 2008, www.rarercancers.org.uk/news/current/improving_access_to_end_of_life_treatments