Drugs on test
Well, at least one thing is clear: when the National Institute for Health and Clinical Excellence (NICE) approves a new drug for use in the Health Service we can be sure it's been through a rigorous and extensive appraisal involving a series of randomised controlled trials...or at least we could.
In an astonishing turn of events the chairman of NICE, Sir Michael Rawlins has turned this established wisdom on its head. Arguing that RCT's have been elevated to an "undeserved pedestal" and holding out the prospect that, in future, some drugs could be approved without meeting this exacting gold standard.
Talking at the Royal College of Physicians Sir Michael said the current methods for testing the effectiveness of new drugs were flawed, and the NHS needs a new, more flexible, system for assessing clinical evidence that takes more account of observational studies.
Randomised trials are certainly expensive: according to one manufacturer the average cost per patient rose from £6,300 in 2005 to £9,900 in 2007. Where the benefits of a drug are obvious or dramatic RCT's are often unnecessary, and where the treatment is designed to target a rare condition the results can be inconclusive.
NICE has come under sustained attack in recent years over the length of time it takes to assess new drugs. In August the National Kidney Foundation branded the regulator's decision not to licence four new kidney cancer drugs as "barbaric, damaging and unacceptable". At the time Sir Michael responded by launching his own withering attack on the pharmaceutical industry and the overpricing of medicines to boost profits. "We are told we are being mean but what nobody mentions is why the drugs are so expensive".
It may be that in appealing for more flexibility Sir Michael is hoping to develop a little more wiggle-room to approve drugs that, although safe and effective, don't perform well in randomised trails.