Cancer: early access to new molecules is not always beneficial. The point with our expert

Cancer early access to new molecules is not always beneficial

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    Ivan Pourmir (medical oncologist)

    According to the results of a new study, less than half of the anticancer drugs approved on an accelerated basis by the American authorities ultimately showed a clinical benefit for patients. Why then resort to very rapid marketing? Oncologist at the Georges-Pompidou hospital (Paris), Dr Ivan Pourmir enlightens us.

    Not all anticancer drugs, approved on an accelerated basis by the Food and Drug Administration, would be beneficial for patients. According to a new study published in the JAMAless than half of these molecules ultimately showed clinical interest.

    What is early access to an anti-cancer drug?

    As part of the treatment of cancers, but also other serious illnesses, it is possible to place a molecule on the market with early access. This is a device allowing the temporary use of certain unauthorized medications, in specific indications, for patients facing a therapeutic impasse.

    This authorization is therefore granted outside of the usual rules that new molecules must follow before being placed on the market. In France, it is the High Authority of Health (HAS) and the National Medicines Safety Agency (ANSM) which examine these requests. All must be accompanied by a collection of data, making it possible to show that the medicine is safe and effective in real conditions of use.

    41% of these molecules did not provide any improvement

    It is precisely this last aspect that seems to be lacking, according to a recent study. Scientists studied 129 accelerated drug approvals, established between 2013 and 2023. They note that 46 of these accelerated approvals were granted between 2013 and 2017. This means that pharmaceutical companies have had at least five years to conduct confirmatory trials .

    Among these 46 drugs:

    • More than 4 in 10 (41% – or 19 out of 46) did not improve overall survival or quality of life in confirmatory trials after more than 5 years of follow-up:
    • For 15% of them, or 7 out of 46, the results are not yet available;
    • For 22%, or 10 out of 46, these medications were withdrawn.

    Moreover, on this specific point, the researchers also discovered that drugs benefiting from accelerated approval were more quickly withdrawn from the market, going from 9.9 years after approval to 3.6 years between 2013 and 2023. On the other hand, it now takes more time to obtain this full approval. Delays thus increased from 1.6 years to 3.6 years during the study period.

    Improve patient monitoring after authorizing these molecules

    The study’s principal investigator, Ian TT Liu, a researcher at Brigham and Women Hospital, comments on these findings. “We believe that decisions made should be both timely but, more importantly, supported by high-quality clinical results”. According to him, it is “essential for the proper functioning of the accelerated approval procedure“.

    Asked about this study, Dr Ivan Pourmir, medical oncologist member of the Doctissimo expert committee, recalls the importance of determining the benefit of these treatments approved in early access. “The main criterion for assessing the effectiveness of an authorized cancer treatment is patient survival, but it is not always possible to use it in the context of early access requests. he explains. “Other secondary criteria may then be observed, such as regression in the size of the tumor or its absence of progression for example, but this is less reliable..

    Be careful of the toxicity of certain molecules!

    The oncologist emphasizes this aspect because sometimes, the authorized molecules can be effective and cause tumors to regress, but also be very toxic for patients. “This will ultimately be harmful, because patients end up dying more quickly.”

    For him, “safeguards” are necessary, in order to better regulate these drugs and verify their long-term benefits, in order to withdraw them from the market if necessary. “But this remains difficult at present, because the collection of data – such as patient survival, subsequent treatment protocols, etc. – is not always correctly ensured during trials.“.

    The authors of the American study conclude by putting the patient back at the center: “Patients should be clearly informed about cancer drugs that use the accelerated approval pathway and do not ultimately show benefit in patient-centered clinical outcomes“.