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BIO Comments on FDA Draft Guidance on Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease

February 15, 2018

BIO submitted comments on the Food and Drug Administration’s (FDA) Draft Guidance, Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease.

BIO welcomes the FDA’s efforts to provide clear, concise guidance on the regulatory and scientific framework for product developers of targeted treatments. This will help sponsors with clinical trial development, including the types/levels of evidence to support a grouping strategy for clinical trial eligibility and benefit-risk evaluation approaches within a disease exhibiting differences in molecular subset.

BIO encourages the FDA to continue to gather stakeholder input on the development of targeted therapies to ensure the guidance keeps pace with scientific advances. BIO’s comments include additional suggestions to clarify the guidance, the scope, and FDA’s receptivity to innovative approaches for the development of targeted therapies.

Download Full Comments Below
BIO Letter Low-Frequency Molecular Subsets FINAL 2-15-18
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