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BIO Comments on Tissue Agnostic Therapies in Oncology: Regulatory Considerations for Orphan Drug Designation

June 8, 2018

BIO submitted comments to the Food and Drug Administration’s (FDA) on Tissue Agnostic Therapies in Oncology: Regulatory Considerations for Orphan Drug Designation, following the Public Workshop on May 9, 2018.

While advances in genomics and precision medicine have significantly improved the ability to develop medicines aimed at specific molecular targets that may be present in multiple diseases and/or cancers, there is much to be learned through continued research and development. BIO appreciates the FDA taking a forward-looking approach and having early discussions about how these advances may affect other aspects of drug development and review, such as orphan drug designations.

In comments following the public workshop, BIO provided several items for the FDA’s consideration, including on the importance of the Orphan Drug Act as well as how to encourage innovation for novel therapies to treat rare diseases. BIO encourages the FDA to consider how to apply a tissue agnostic approach outside of oncology. BIO also asks the FDA to partner with stakeholders to discuss the impacts of tissue agnostic therapies on drug development and review.

Download Full Comments Below
BIO Lettter Tissue Agnostic Therapies In Oncology FDA–2018-N-0663
Read full comment letter below
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