Drugmakers Request Clarification of FDA Flexibility in Rare Disease Drug Development

Wednesday, October 28, 2015


Drugmakers want the FDA to provide more details and examples of how it would show regulatory flexibility to developers of drugs for rare diseases, saying recent draft guidance is unclear about what may or may not be appropriate.

The Aug. 13 guidance says the FDA may provide flexibility in applying regulatory standards, since certain aspects of drug development that are feasible for common diseases may not be for rare diseases. This flexibility would extend from the early phases of development to design of adequate and well-controlled clinical studies required to demonstrate safety and effectiveness.

In comments to the agency, PhRMA praises the flexible approach but asks for more specific recommendations on issues such as acceptability of single-arm and open-label studies.

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