Boss of drug watchdog attacks testing method

The man in charge of drug rationing in the NHS will call for a radical new way of judging the effectiveness of treatments today. Professor Sir Michael Rawlins, chairman of the National Institute for Clinical Excellence (Nice), says current methods are flawed and the NHS needs a new approach to assessing clinical evidence. The randomised controlled trial, long seen as the gold standard of evidence, has been put on an "undeserved pedestal" and other types of evidence should be considered alongside it, he says.

Sir Michael, the chairman of Nice since its launch in 1999, has faced a barrage of criticism over decisions by the institute to restrict access to expensive drugs for cancer and other conditions. A draft recommendation to ban the use of four kidney cancer drugs on the NHS last summer drew a storm of protest from patient groups and drug companies. Criticism has centred on the mathematical model used for calculating cost effectiveness and the £30,000 threshold, which campaigners say should be raised.

In a speech to be delivered to the Royal College of Physicians tonight, Sir Michael does not mention Nice but calls the current reliance on randomised controlled trials "inappropriate".

The trials are expensive to run – up to £95m – and unnecessary when treatment effects are dramatic and impossible when the treatment is for a rare condition with few patients affected, he says. There is no consensus among statisticians over how to calculate whether a trial has been stopped too early, has created a misleadingly positive result, or whether the findings can be generalised to a wider population.

Efforts to replace judgements with more reliable and robust approaches based on "hierarchies" of evidence are wrong, Sir Michael will say. He will add: "It is scientific judgement, conditioned of course by the totality of the evidence, that is at the heart of making decisions about the benefits and harms of therapeutic interventions."

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