41 percent of cancer drugs that were accelerated to market in the US between 2013 and 2017 did not meet expectations, a study has now shown.
Ian Liu from the Department of Pharmaepidemiology and his co-authors, all from the world-renowned Harvard Medical School in Boston in the USA, asked a simple question in their scientific study: “What is the clinical benefit of cancer drugs that are approved on an accelerated basis and on what basis was the (provisional; note) approval finally converted into a regular one?”
Fast market approvals
The background: At least since the AIDS pandemic in the mid-1980s and the then hectic search for therapies effective against HIV, the drug authorities – in the USA the Food and Drug Administration/FDA, in the EU now the European Medicines Agency EMA – also on particularly quick market approvals. This often happens on the basis of small phase I or phase II clinical studies when no data from large efficacy studies are yet available.
For example, ethical arguments are brought into the meeting: one cannot withhold possible new and effective therapies from patients who otherwise do not have good treatment options. For the pharmaceutical industry, on the other hand, this means particularly quick and lucrative market access. After a preliminary market release in an accelerated procedure, the information from phase III studies should then be submitted in order to achieve regular approval.
Liu and the other scientists involved looked at this for cancer drugs that had received preliminary approval from the FDA in the US following an abbreviated process. The scientists: “In this cohort study of cancer drugs that received accelerated approval between 2013 and 2017, 41 percent (19 of 46) did not improve survival or quality of life in subsequent confirmatory studies with more than five years of observation. For 15 percent of the drugs (seven out of 46) there was no information on this. For 60 percent of the drugs that were finally approved (29 out of 48) this was based exclusively on surrogate markers.”
The latter means that it was not the decisive criteria such as extending life or improving quality of life that were responsible for this, but rather substitute markers that merely made a positive effect probable.
Less than half proved successful
In total, exactly 129 cancer drugs were provisionally approved by the US Food and Drug Administration (FDA) between 2013 and 2023 as part of an accelerated procedure. Of the 46 with more than five years of observation, almost two thirds were finally approved regularly, and one in five were taken off the market altogether. Less than half, according to the authors in the study in the JAMA Network of the US Medical Association (AMA; doi:10.1001/jama.2024.2396), proved successful in subsequent confirmatory studies.
The scientists’ conclusion: “Most cancer drugs with accelerated approval showed no benefit in overall survival (deaths from all possible causes; note) or in quality of life within five years of faster registration. Patients should be clearly informed about cancer drugs, which become available through the faster approval process and ultimately show no advantage in terms of the results that are crucial for those affected.”
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