Gottlieb looking to approve cancer treatments on earlier evidence
Scott Gottlieb, FDA commissioner, was speaking in front of the House Committee on Energy and Commerce, regarding the 21st Century Cures Act, when he dropped a hint at a new way of approving oncology treatments that will have caught the attention of the pharma industry.
During the course of the hearing, Gottlieb mentioned that the agency is looking at ways of approving cancer treatments based on early data, where the potential benefits to overall survival are “outsized”. The treatment would still need to return with a post-marketing confirmatory study that proved its clinical benefit.
Gottlieb compared the process to the existing accelerated approval process, whereby treatments are approved for serious unmet medical need based on early-stage or smaller-than-usual data sets.
In addition to this, he also mentioned further potential steps: “To fully leverage these opportunities, and in keeping with the spirit of Cures, we’re working on a similar proposal for cancer drugs already approved for one indication – approval for a supplemental application, where the approval concerns a second indication, can sometimes appropriately rely on a more targeted data set, like a single-arm study. We intend to issue guidance further clarifying the circumstances in which this is appropriate.”
The announcement will be welcomed by the pharma industry, which stands to gain by having treatments sped through the regulatory process.
Though it will raise questions regarding what will happen should a treatment be given accelerated approval and then fail to show benefit. Objectively, it would have marketing authorisation pulled if it could not confirm the data but there have already been cases where this has not been the case for drugs going through accelerated approval.
Tecentriq is the foremost example, it gained accelerated approval based on Phase 2 data for second-line urothelial bladder cancer only for the drug to flop in Phase 3 trials, by failing to meet its primary endpoint of extending overall survival. Roche and the FDA held discussions, with a decision regarding the treatment’s potential in the setting to be based on further evidence from on-going trials.
To complicate matters further, Tecentriq had already scored approval in first-line setting for the same indication based on the same Phase 2 data.
The saga brought into question the accelerated approval process and means that any such revisions brought into the area will be particularly scrutinised.
Though, as Gottlieb referenced, the changes being brought through are part of a concerted push to speed up the drug approval process that has come from above: “It was a direction from Congress that you wanted us to think differently when it came to the potential for breakthroughs that could transform human health. We pledged to remain steadfast to our gold standard for safety and efficacy. But at the same time, you asked us to look for ways that we can make our approach to the development of breakthrough products more scientifically modern and efficient, to meet the urgent needs of patients.”
The US Food and Drug Administration (FDA) has granted de novo marketing authorisation to the …
Biogen has announced that the US Food and Drug Administration (FDA) has approved Tofidence (tocilizumab-bavi) …