BIO Comments on Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments
September 23, 2022
BIO and its members appreciate the opportunity to work with FDA to develop and align on approaches that are robust, practical, and expedite patient focused drug development. To further enhance the Guidance, we believe that a few areas would benefit from more explanation, examples, and references. We have identified through our comments areas where these additions would be beneficial. BIO commends this shift to a fit-for-purpose evidentiary framework and believes this will support thoughtful and pragmatic COA development. BIO also supports the draft guidance’s recognition that some health aspects are complex, more than one type of COA might be used to assess different aspects of a concept of interest, and COAs might measure concepts of interest that are indirect reflections of a meaningful health concept.
However, BIO notes that with a shift in thinking, the draft guidance introduces new terminology and concepts that have not been previously discussed in prior PFDD guidances or discussion documents. We request that the Agency consider providing an updated glossary and/or detailing new or shifting terminology between the final guidance and previous PFDD guidances. To further enhance the guidance, BIO recommends the inclusion of references and case examples (real/hypothetical). Furthermore, BIO emphasizes the importance of clearly defined opportunities for FDA-sponsor interactions early and throughout drug development. Given the new concepts and methods introduced in this draft guidance, communication between sponsors and FDA will be necessary to ensure that the guidance recommendations are implemented correctly.
Lastly, BIO encourages FDA to describe whether and how digital health technology tools (i.e., digital endpoints) could be utilized to support regulatory decision-making in this guidance.
Download Full Comments Below
BIO Comment Letter_PFDD Guidance 3_FDA-2022-D-1385
On Tuesday, January 3rd, BIO submitted comments in response to the Food and Drug Administration’s (FDA) recent draft guidance containing recommendations for sponsors on monitoring growth and evaluating pubertal development in clinical trials which…
On Wednesday, June 22nd, BIO submitted comments on the FDA’s Quality Metrics (QM) Reporting Program. In the comments submitted, BIO stated appreciation for FDA’s intent to support a general shift toward a risk-based approach to regulation by…
On Wednesday, September 28th, BIO submitted comments to the Food & Drug Administration on the ICH Q2(R2) Guideline: Validation of Analytical Procedures and the ICH Q14 Guideline: Analytical Procedure Development. In addition to a number of…
.png){kind=logo}
BIO and its members appreciate the opportunity to work with FDA to develop and align on approaches that are robust, practical, and expedite patient focused drug development. To further enhance the Guidance, we believe that a few areas would benefit from more explanation, examples, and references. We have identified through our comments areas where these additions would be beneficial. BIO commends this shift to a fit-for-purpose evidentiary framework and believes this will support thoughtful and pragmatic COA development. BIO also supports the draft guidance’s recognition that some health aspects are complex, more than one type of COA might be used to assess different aspects of a concept of interest, and COAs might measure concepts of interest that are indirect reflections of a meaningful health concept.
However, BIO notes that with a shift in thinking, the draft guidance introduces new terminology and concepts that have not been previously discussed in prior PFDD guidances or discussion documents. We request that the Agency consider providing an updated glossary and/or detailing new or shifting terminology between the final guidance and previous PFDD guidances. To further enhance the guidance, BIO recommends the inclusion of references and case examples (real/hypothetical). Furthermore, BIO emphasizes the importance of clearly defined opportunities for FDA-sponsor interactions early and throughout drug development. Given the new concepts and methods introduced in this draft guidance, communication between sponsors and FDA will be necessary to ensure that the guidance recommendations are implemented correctly.
Lastly, BIO encourages FDA to describe whether and how digital health technology tools (i.e., digital endpoints) could be utilized to support regulatory decision-making in this guidance.