Experts disagree over NICE's approach for assessing drugs

Abstract

This month, the UK's High Court ruled in favour of the National Institute for Health and Clinical Excellence decision to limit access to drugs for Alzheimer's disease. But the legal win is unlikely to stem the recent spate of controversies surrounding the institute. Richard Hoey reports. It was a legal ruling greeted with relief at the offices of the National Institute for Health and Clinical Excellence (NICE) but bitter disappointment among campaigners and the pharmaceutical industry. After 10 months of legal wrangles, a London court upheld the institute's decision to restrict access to drugs for Alzheimer's disease. But although the ruling will fortify those who believe medicines should be rigorously assessed for cost-effectiveness, no one is pretending it will quell the lasting controversy over how such assessments should be done. NICE is seen as a world leader in allocating health-care resources by evidence-based assessment, and the legal proceedings were followed by interested groups far beyond England and Wales—the area of its remit. But the institute has also proved a magnet for controversy, with the Alzheimer's furore only the most intense of a series in recent months, also involving drugs for macular degeneration, rheumatoid arthritis, and bowel cancer. David Wilkinson, a consultant in old-age psychiatry at the Hampshire Partnership Trust, is among those who remain angry at the NICE decision to limit access to the Alzheimer's drugs donepezil, rivastigmine, and galantamine, believing the institute to be insensitive to the benefits of drugs for degenerative illness. “NICE has taken the view a response is only measurable if there is an improvement over baseline, but with a degenerative disease that is clearly difficult. It is facile and simplistic”, he says. The row vividly illustrates the difficulty of assessment of treatments in a way everyone agrees is fair, and that reconciles the quality of trial evidence with patients' and doctors' personal beliefs about a treatment's value. For some, NICE is fundamentally discriminatory, through its use of the quality-adjusted life-year (QALY) to assess cost-effectiveness. The QALY is a statistical measure that assesses treatments by their effect on both length and quality of life, and cost per QALY is intended as a common currency for comparing value for money across age-groups or populations. But John Harris, professor of bioethics at the University of Manchester and editor-in-chief of the Journal of Medical Ethics, believes use of the QALY discriminates against the elderly, who have fewer years of life left to preserve. “I am rather disheartened by NICE's approach that what matters is life-years, not lives. It is perfectly reasonable to take this position, but it is not incontrovertibly true. I believe a tragedy is big or small in proportion to the lives lost, not the life-years lost”, he says. Use of the QALY is far from universal among equivalent bodies internationally. The Pharmaceutical Management Agency of New Zealand (PHARMAC), established in 1993—6 years before NICE—assesses cost-effectiveness as only part of its assessment, and unlike NICE sets no QALY threshold. Canada's Common Drug Review (CDR) does not use the QALY at all. The CDR prefers to concentrate on assessing clinical effectiveness using a more flexible “commonsense” approach to take into account cost. James Wright, managing director of the University of British Colombia's Therapeutics Initiative, which assesses evidence for the CDR, questions the evidence for use of QALYs. “The CDR does not get into QALYs and I think it is smart not to. I am not convinced that trying to calculate QALYs is useful, they are based on assumptions that are hard for most people to understand and the science is often questionable. It is much better to know the benefits and harms in the [specific] population a drug has been tested in.” But Sir Michael Rawlins, chairman of NICE, believes that the High Court ruling was a vindication of the institute's approach. He concedes that there are some procedural lessons to be learnt, to ensure that all groups are catered for, but vigorously rejects the charge that use of the QALY is discriminatory. “It is a woeful misunderstanding of the QALY. Almost invariably it works to the advantage of the elderly, because often treatments are more cost effective in them.” “We have to work out if treatments are good value for money compared with what we already have, and do this across conditions and populations. The QALY seems the most appropriate, if not the only appropriate, measure we have got”, he said. Although some countries may be reluctant to adopt the QALY, many—including Germany, France, and Sweden—plan to replicate the role of NICE more generally. The USA's Institute of Medicine also wants to introduce a resource centre for health-care providers. Just how successful NICE has been is open to question. Harris is among those who criticise it as a crude rationing body. Others, conversely, point to its failure to control escalating drug costs. Between 2000 and 2005, the UK's pharmaceutical expenditure as a proportion of gross domestic product (GDP) rose by an average of 7·3% a year. This figure compares with an annual rise of 7·7% in the USA and 10·8% in Canada between 1999 and 2004, but with rises of 6·0% and 6·7%, respectively, in Germany and France. Rawlins accepts that NICE has to take into account the limited pot of money available for health care, but insists it does not exist to save money. “I do not think anyone makes any bones that resources are limited and are allocated in the most appropriate fashion. But I have always objected to rationing as a term. I experienced rationing as a child. You got your sweeties for the week and that was it. We do not behave that way in the health service.” He estimates NICE has actually added £1·2 billion each year to the UK drugs bill. “I never expected to save money. If we are there to improve health services, that will cost more.” But New Zealand's PHARMAC has been successful at controlling drug expenditure, estimating it has kept annual increases down to 2% or 3%. Matthew Brougham, PHARMAC's acting chief executive, explains: “Where New Zealand and the UK diverge is that PHARMAC has a budget management role, so value for money and budgetary impact are inextricably linked. NICE makes recommendations independently of the impact on NHS [National Health Service] trust budgets. We have seen comment that some NICE recommendations have caused tension at trusts.” Such tensions will be one of the issues that the House of Commons Health Select Committee addresses when it reports on its current inquiry into NICE. Alan Maynard—a specialist adviser to the committee and professor of health economics at the University of York—says trusts are left to take some difficult rationing decisions because of differences in the status of NICE guidance, with a legal obligation to fund technology appraisals of new drugs, but not clinical or public-health guidelines. “London's primary care trusts [PCTs] are trying to get an agreement on rationing criteria and my view is we should do that across the country. For obesity surgery, some PCTs use a BMI [body mass index] threshold of 30, others 40. We should be looking at the evidence and coming up with a decision for everyone. PCTs are too small to do that”, he says. The Association of the British Pharmaceutical Industry (ABPI) is lobbying the inquiry for changes to the way NICE assesses new treatments, particularly drugs for terminal illnesses such as cancer. It wants NICE to shift its cost per QALY threshold for cancer drugs upwards from the current limit of £30 000. “QALYs are relative and if your comparator drug is a cheap generic, it is difficult to hit the cost-effectiveness threshold. You are saying to companies do not bother to research drugs in these areas”, an ABPI spokesperson told The Lancet. But Peter Smith, a colleague of Maynard's at York, has come to the opposite conclusion. He recently did a study showing that PCTs were using far stricter QALY thresholds for their rationing decisions than was NICE. He suggests NICE has been “if anything too liberal in accepting some high-cost treatments”, and advocates reducing the threshold to £20 000 per QALY. Smith admits a tougher threshold for new drugs would be controversial, but argues public-health interventions, or more efficient ways of delivering existing treatments, would be better value for money for the NHS than would be many new drugs. Wright agrees. He warns that “NICE has lost some of its credibility because in a number of cases it has come under political pressure and its decisions have been reversed. Any group that uses evidence is going to be making the judgment that most of the drugs coming onto the market are not very valuable”. But NICE seems unlikely to allow itself to be pushed towards a stricter QALY threshold, with Rawlins keenly aware that such a move would only intensify the controversy swirling round the organisation. What he does want is some help from politicians, calling for them to “come clean” about the difficult decisions that need to be taken over health-care funding.