The U.S. Food and Drug Administration on Wednesday proposed a new process to streamline the approval of drugs targeting rare diseases with very small patient populations, including cases supported by data from single-arm clinical trials.
The regulator said eligibility will be based on whether the disease affects a small population – fewer than 1,000 individuals in the United States – and is intended to treat a genetic defect.
It will also depend on whether there are no adequate alternative therapies that alter the course of the disease.
The agency added that it expects substantial evidence of effectiveness may generally be established based on one adequate study, which may be a single-arm trial.
This will be supported by data that provides confirmatory evidence of the drug’s treatment effect, which could be data on its action at the target site and pre-clinical data.
FDA’s proposal comes after Health Secretary Robert F. Kennedy Jr. said in June that the U.S. drugs regulator would look for ways to fast-track approval for rare disease treatments and remove obstacles to their path to market.
The appointment of Vinay Prasad, the FDA’s top vaccine and biologics official, as the head of the FDA’s Center for Biologics Evaluation and Research had stoked fears among industry observers.
Concerns arose that he could raise the bar for companies to secure approval for new drugs, including accelerated approvals for potential treatments of serious conditions.
The smaller the population in the rare disease being studied, the more difficult it is to generate evidence to meet requirements for demonstrating efficacy through traditional trial designs and the quantity of clinical studies, the FDA said.
