LAST month, plans were announced to change the way new medicines are assessed by the National Institute for Health and Care Excellence (NICE). This body decides which medicines can be used in the National Health Services in England and Wales. While some proposals are clearly welcome, like getting firms to put submissions to NICE in plain English, others require caution, like accepting less rigorously conducted trials as supporting evidence.
NICE says the changes are designed to speed up the introduction of new medicines and support innovation by pharmaceutical companies. It also wants to encourage drug companies to launch their products in the UK first, now the country has left the European Union. Faster access to medicines sounds like an unalloyed good, but the experience of the US Food and Drug Administration (FDA) when trying to achieve the same thing is a cautionary tale.
The FDA has a different job to NICE: to assess whether new medicines are safe, effective and are manufactured properly. The UK has an equivalent body that does that: the Medicines and Healthcare Products Regulatory Agency. NICE assesses medicines on a fourth criterion: whether the benefits brought by a treatment are worth the price. Nevertheless, both the FDA and NICE act as brakes that stop pharmaceutical firms from being able to market whatever medicines they like.
The FDA has come in for criticism for letting an increasing number of products be reviewed under fast-track schemes that require less stringent proof. Companies that get fast-track approval are supposed to carry out randomised trials after a product’s launch to show it really is effective, and to stop selling their medicine if the trial finds it doesn’t work.
But the system isn’t always working as intended. In many cases, the companies’ post-approval trials are delayed for years. Even if a trial is negative, a drug might not be withdrawn; a study of fast-tracked cancer medicines in the US found that one-third were still in use years after being found not to work.
The FDA’s most recent controversy was over its approval, in June, of Aduhelm to treat Alzheimer’s disease, after its scientific advisory panel advised against the move, triggering resignations and an inquiry.
The fact that the FDA has these problems doesn’t mean that NICE will go the same way. But NICE’s consultation document, which invites the public to give feedback on its proposals, gives the impression that there is little need for caution. Its website says the moves are to allow “earlier patient access to valuable innovative treatments”. Few people are likely to respond saying they want slower access to medicines, suggesting the consultation is something of a PR exercise.
Another of the proposed changes is to accept less stringent forms of supporting evidence, namely non-randomised trials and “real-world evidence”. In practice, the latter means hearing reports from people who have the relevant medical condition about their experience with the drug or how much they want to receive it.
Such testimonies are often organised by patient support charities that take funding from the manufacturers and so may be biased. Seven out of 10 such charities contributing to NICE appraisals had recently received money from the manufacturer of the medicine under review, according to a 2019 study.
NICE already does a difficult job, balancing the needs of patients against National Health Service budgets, without upsetting big pharma too much. Now it has political masters who want it to fulfil another role: showing that Brexit has been a success. Let us hope that this important medical body manages to successfully walk the tightrope.
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