Report of a Workshop on Confirmatory Evidence to Support a Single Clinical Trial As a Basis for New Drug Approval
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0092-8615/2002
Copyright 0 2002 Drug Information Association Inc.
REPORT OF A WORKSHOP ON CONFIRMATORY EVIDENCE TO SUPPORT A SINGLE CLINICAL TRIAL AS A BASIS FOR NEW DRUG APPROVAL* CARLC. PECK, MD Director, Center for Drug Development Science, Georgetown University Medical Center, Washington, District of Columbia
JILLWECHSLER, MA Editor and Writer, Chevy Chase, Maryland
A novel approach for more eflcient, economical, and faster clinical evaluation of new drugs is to couple effectiveness data from phase 2 and other studies with a phase 3 single clinical trial (SCT)for regulatory approval. Sanctioned in the Food and Drug Administration Modernization Act (FDAMA), this approach challenges the traditional requirement for two controlled phase 3 trials for approving a new therapy. Academic, industry, legal, and regulatory experts participated in a workshop to define adequate confirmatory evidence of effectiveness to support regulatory approval based on data from an SCT. Participants examined qualities of confirmatory evidence and SCTS and implications of this model for clinical safety information. Acknowledging the evolutionary nature of scientific evidence of effectiveness, participants identijied risks and benefits of this approach, concerns of FDA and pharmaceutical companies, and policy changes that may further encourage widespread use of the confirmatory evidence-SCT model. For example, these policy changes include explicit FDA publication of the basis for effectiveness determinations of new drugs, use of end-of-phase 1 industry-regulator meetings for prospective planning of the confirmatory evidence-SCT program, and more use of established pharmacological knowledge to qualifv confirmatory evidence biomarkers and surrogate endpoints. Key Words: Confirmatory evidence; Single clinical trial; Evidence of effectiveness; FDAMA Section 115a; Drug approval standards
A WORKSHOP ENTITLED “Confirmatory Evidence (CE) to Support a Single Clinical Trial ( S C T ) as a Basis for Drug Approv-
al-An Exploratory Workshop” was held January 15-16,2002 at Georgetown University in Washington, District of Columbia.
Reprint address: Carl C. Peck, MD, director, Center for Drug Development Science, Georgetown University Medical Center, Med-Dent NE 405, 3900 Reservoir Road NW, Washington, Dc 20007-2195. *This article is based upon the workshop entitled “Confirmatory Evidence to Support a Single Clinical
Trial (SCT) as a Basis for Drug Approval,” January 15-16. 2002, Washington, DC. This workshop was supported by an unrestricted educational grant from the Yamanouchi USA Foundation and unrestricted supporting grants from AstraZeneca, Inhale, Lilly, Immunex, Novartis. and Wyeth-Ayerst.
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Carl C. Peck and Jill Wechsler
Sponsored by the Center for Drug Development Science ( I ) , the workshop aimed to achieve the following objectives: 1. Explain the legislative and legal implica-
tions of using confirmatory evidence and an SCT, 2. Define the nature and sourc
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