Statement on data accuracy issues with recently approved gene therapy | FDA

Statement on data accuracy issues with recently approved gene therapy | FDA

Statement by Peter Marks, M.D., Ph.D., director of FDA’s Center for Biologics Evaluation and Research, on data accuracy issues with recently approved gene therapy

As a public health agency, we believe that it is critical to facilitate the development of innovative safe and effective medical products, like the cellular and gene therapy products that have shown enormous potential to treat previously untreatable diseases. As part of the approval process for biological products, the agency reviews extensive information submitted by manufacturers, including details of how the product is made in a reproducible manner, how it has been tested in animals, and how it has been investigated in human clinical trials to demonstrate its safety and efficacy.   It is the manufacturer’s responsibility to submit complete and accurate information in marketing applications for evaluation by the FDA. If we become aware of a concern with data submitted to ...

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