Drugs

Praise for FDA Bayesian Methodology Draft Guidance

David McFarland

— 1 min read
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Pharmaceutical Research and Manufacturers of America (PhRMA) says it commends FDA for issuing a draft guidance, Use of Bayesian Methodology in Clinical Trials of Drug and Biological Products, to facilitate the advancement and use of complex adaptive, Bayesian, and other novel clinical trial designs. In a comment letter, PhRMA says it supports the expanded use of innovative clinical trial approaches, including Bayesian approaches, “as they offer the potential for more patient-focused, efficient, timely, and targeted medical product development to support regulatory decision-making.” The industry group calls on FDA to:

  • balance expectations related to specifications, justification, and documentation with the need for flexibility and reducing operational burden in clinical trials using Bayesian methodology;
  • revise the draft guidance to discuss the broader applicability of Bayesian methods; and
  • add visual aids or additional documents to improve the accessibility for readers who are not familiar with Bayesian methods.

PhRMA also submits specific comments by section and subsection of the draft.

In its comment, the Biotechnology Innovation Organization (BIO) says the draft guidance is “clear, comprehensive, and reflects meaningful progress toward enabling innovative clinical trial methodologies that can help patients gain more timely access to safe and effective therapies.” BIO says several areas of the guidance would benefit from additional clarification and emphasizes these principles:

  • Bayesian methods should be recognized as a general evidentiary framework, not only a niche tool;
  • proportionality of evidentiary and documentation expectations is essential;
  • clearer expectations are needed about operating characteristics and calibration;
  • flexibility in incorporating relevant evidence should be preserved;
  • rare diseases and small population settings require tailored considerations;
  • practical implementation and communication should be addressed explicitly; and
  • predictive distributions for decision-making should be discussed.

The Critical Path Institute says the draft represents a significant and timely advancement in regulatory science and reflects the agency’s continued leadership in modernizing evidentiary frameworks to support innovation in drug development. It says the guidance strikes an appropriate balance between flexibility and rigor. “By avoiding overly prescriptive requirements while clearly communicating expectations for scientific justification and documentation, FDA enables innovation while maintaining high evidentiary standards,” the institute says.

The Duke-Margolis Institute for Health Policy says it particularly values the inclusion of examples of when Bayesian approaches have been used, success criteria, and details on reporting. “We recommend that FDA include examples of where they envision Bayesian approaches being used in the future,” it says.

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