FDA last week launched what it hopes will be a lively public discussion on whether extremely sensitive outcomes measures or very large trials can produce efficacy results that are statistically significant but are too clinically trivial to support approval.
At bottom is the question of whether FDA should make approvals contingent on achieving minimum levels of efficacy. Senior staff say they want the issue aired publicly, but they lean against such a requirement.
Traditionally, FDA has said that in the absence of countervailing safety risks, any significant benefit on a clinical endpoint can serve as the basis for approval. Shifting from that position would have profound implications for drug development.
Robert Temple, director of FDA's Office of Medical Policy, gave a presentation entitled "Effect Size - Can the Effect Be Too Small?" during last week's meeting of the Cardiovascular and Renal Drugs Advisory Committee to review Cellegesic nitroglycerin ointment to treat anal fissure pain, from Cellegy Pharmaceuticals Inc. (CLGY, South San Francisco, Calif.) (see "Cellegesic Splits Its Panel," A9).
Norman Stockbridge, the primary reviewer of