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BIO Comments on FDA Draft Guidance on Human Gene Therapy for Rare Diseases

November 14, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Human Gene Therapy for Rare Diseases.

BIO says the guidance will assist stakeholders developing human gene therapy (GT) products to treat rare diseases. BIO reminds FDA that complex, innovative clinical trial designs might be fit-for-purpose to execute clinical trials of GT products for rare diseases. Consequently, BIO urges FDA to provide regulatory flexibility to allow for innovative clinical trial designs – and to not otherwise use limiting language, such as recommending a specific number of treatment arms, utilizing different doses but the same product administration procedures, or the inclusion of a sham control group. The use of innovative clinical trial designs can decrease unnecessary and unethical patient exposure to clinical procedures and experimental products while still advancing clinical development.

BIO also requests additional guidance on what types of patient experience data would be most helpful to inform regulatory decision-making.


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FINAL BIO Letter Human Gene Therapy For Rare Diseases 12-10-18
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