Commissioner Marty Makary said that the FDA will soon start requiring only one pivotal trial, instead of two, for companies seeking approval for new drugs.
The FDA will begin asking for only one pivotal clinical trial instead of the two that has long been standard before considering new drugs for approval.
“You can achieve the same statistical power with one trial as you would with two,” FDA commissioner Marty Makary told STAT, which broke the news on Thursday.
The change is still in the works and will be finalized in the next three to six months. However, the idea is already making waves. The push to make the idea public apparently was part of the reason that Richard Pazdur, who had just taken the helm as head of the FDA’s Center for Drug Evaluation and Research, suddenly decided to retire.
Calling it the “biggest jailbreak in biotech history,” biopharma consultant David Alderman was enthusiastic about the change.
“Makary just dropped a regulatory acceleration warhead in the middle of every portfolio model in the industry,” Alderman wrote in a post on LinkedIn, noting that in lowering the bar for data needed for approval, companies could save years of planning and billions in cost reductions.
The reaction from Wall Street was more mixed, with benefits clearly arrayed for drugmakers but confusion remaining at the regulatory level.
“While the FDA has increasingly shown flexibility in recent years, particularly for rare diseases and areas of significant unmet need, indications with large, heterogeneous populations and multiple existing therapies have historically required stronger evidence,” Truist analysts wrote in a note to investors Thursday.
Nevertheless, “while we are concerned about the long-term risks that a lower approval threshold may introduce,” a litany of companies with their eyes on FDA approvals could stand to benefit from the change, the analysts noted.
Truist pointed to Biogen as one example, saying that the company could seek approval on the basis of the positive Phase III trial data already in hand for dapirolizumab pegol in systemic lupus erythematosus.
Another example provided was Cytokinetics, which is testing ulcamten in a Phase II trial in patients with heart failure with preserved ejection fraction and could hypothetically get across the FDA finish line with just a single Phase III trial—particularly notable “in a space historically requiring multiple large studies,” the analysts wrote.
However, many questions remained. The analysts pointed out, for example, that the FDA already allows companies the flexibility to submit data from one late-stage trial under some circumstances. It’s also not clear under what conditions the agency would still ask for two trials.
Furthermore, companies planning global launches will likely run multiple trials anyway in order to satisfy the requirements for ex-U.S. nations, Truist said.
