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Schaeffer experts submitted comments to the Food and Drug Administration in response to revised draft guidance, “Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products.” The guidance advances a framework that gives sponsors clearer direction on how a single adequate and well-controlled clinical investigation, together with confirmatory evidence, could satisfy the statutory substantial evidence standard.
The experts’ comments, which build on their Schaeffer Center white paper on FDA reform, recommend five refinements to the draft guidance:
1. Clarify its terminology on “regulatory flexibility” to avoid creating misconceptions regarding how it applies the statutory standard of substantial evidence of effectiveness;
2. Provide more affirmative guidance on the appropriate use of external controls — including when and how externally controlled trials can support a finding of substantial evidence — rather than framing external controls primarily in terms of their limitations;
3. Explicitly encourage novel trial designs — including master protocols and adaptive designs — as tools for use in pivotal trials and confirmatory evidence;
4. Incorporate patient preference information (“PPI”) as a tool for trial design — including endpoint selection and the calibration of success criteria; and
5. Address rare disease-specific considerations in its discussion of external controls.
Read the comment letter here.
