(Reuters) -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 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.
(Reporting by Puyaan Singh in Bengaluru; Editing by Mohammed Safi Shamsi)
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