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Blood, 1 March 2007, Vol. 109, No. 5, pp. 1810-1816.
Prepublished online as a Blood First Edition Paper on November 9, 2006; DOI 10.1182/blood-2006-08-041152.
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Submitted August 24, 2006
Accepted September 27, 2006
Endpoints to establish the efficacy of new agents in the treatment of acute leukemia
Frederick R. Appelbaum*, Daniel Rosenblum, Robert J. Arceci, William L. Carroll, Philip P. Breitfeld, Stephen J. Forman, Richard A. Larson, Stephanie J. Lee, Sharon B. Murphy, Susan O'Brien, Jerald Radich, Nancy S. Scher, Franklin O. Smith, Richard M. Stone, and Martin S. Tallman
Fred Hutchinson Cancer Research Center, Seattle, WA
National Institutes of Health, Bethesda, MD
The Sidney Kimmel Comprehensive Cancer Center, John Hopkins, Baltimore, MD
Mount Sinai and New York University Schools of Medicine, New York, NY
Duke University School of Medicine, Durham, NC
City of Hope National Medical Center, Duarte, CA
University of Chicago Medical Center, Chicago, IL
Dana-Farber Cancer Institute, Boston, MA
Children's Cancer Research Institute, University of Texas Health Sciences Center at San Antonio, San Antonio, TX
MD Anderson Cancer Center, Houston, TX
Fred Hutchinson Cancer Research, Seattle, WA
U.S. Food and Drug Administration, Rockville, MD
Cincinnati Children's Hospital Medical Center, Cincinnati, OH
Northwestern University Medical School, Chicago, IL
* Corresponding author; email: fappelba{at}fhcrc.org.
Federal regulations provide two pathways for approval of new agents for the treatment of acute leukemia, regular and accelerated approval. Regular approval requires evidence of clinical benefit, which is generally defined as either prolongation of life or improved quality of life, or an effect on an endpoint established as a surrogate for clinical benefit. Accelerated approval can be obtained based on demonstration of an effect on a surrogate measure "reasonably likely" to predict clinical benefit, but requires post-approval demonstration of clinical benefit as well. The acute leukemias are a heterogeneous and relatively uncommon group of diseases. The design and execution of prospective randomized clinical trials demonstrating prolongation of life or improved quality of life for patients with these disorders can be difficult, costly and require lengthy follow-up. Thus, the development of novel trial design and inclusion of validated surrogate markers for clinical benefit are needed. To explore some of the issues pertinent to the choice of endpoints for drug approval in acute leukemia, the FDA invited the American Society of Hematology (ASH) to participate in the organization and conduct of a joint workshop. In this report, we present the results of that effort.
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