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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.
PERSPECTIVE End points to establish the efficacy of new agents in the treatment of acute leukemia1 Fred Hutchinson Cancer Research Center, Seattle, WA; 2 National Institutes of Health, Bethesda, MD; 3 Sidney Kimmel Comprehensive Cancer Center at John Hopkins, Baltimore, MD; 4 Mount Sinai and New York University Schools of Medicine, New York, NY; 5 Duke University School of Medicine, Durham, NC; 6 City of Hope National Medical Center, Duarte, CA; 7 University of Chicago Medical Center, IL; 8 Dana-Farber Cancer Institute, Boston, MA; 9 Children's Cancer Research Institute, University of Texas Health Sciences Center at San Antonio; 10 M. D. Anderson Cancer Center, Houston, TX; 11 US Food and Drug Administration, Rockville, MD; 12 Cincinnati Children's Hospital Medical Center, OH; 13 Northwestern University Medical School, Chicago, IL Federal regulations provide 2 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 end point 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 demonstration of clinical benefit after approval 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 and 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 end points for drug approval in acute leukemia, the Food and Drug Administration invited the American Society of Hematology 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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| Copyright © 2007 by American Society of Hematology Online ISSN: 1528-0020 | |||||||||
