Project Confirm: Accelerated Drug Approvals for Chronic Myeloid Leukemia

Kendra L Sweet; Jorge E Cortes; Jane F Apperley; Mel Mann; Michael J Mauro; Vivian G Oehler; Cristina Ruiz; Charles A Schiffer; Lori A Ehrlich; Gulsum E Pamuk; Joseph Wynne; Gautam U Mehta; R Angelo de Claro; Marc R Theoret; B Douglas Smith; Kelly J Norsworthy

doi:10.1158/1078-0432.CCR-22-2628

Project Confirm: Accelerated Drug Approvals for Chronic Myeloid Leukemia

Clin Cancer Res. 2023 Jun 13;29(12):2179-2183. doi: 10.1158/1078-0432.CCR-22-2628.

Authors

Kendra L Sweet¹, Jorge E Cortes², Jane F Apperley³, Mel Mann⁴, Michael J Mauro⁵, Vivian G Oehler⁶, Cristina Ruiz⁷, Charles A Schiffer⁸, Lori A Ehrlich⁹, Gulsum E Pamuk⁹, Joseph Wynne⁹, Gautam U Mehta⁹, R Angelo de Claro⁹, Marc R Theoret¹⁰, B Douglas Smith¹¹, Kelly J Norsworthy⁹

Affiliations

¹ Moffitt Cancer Center, Tampa, Florida.
² Georgia Cancer Center, Augusta University, Augusta, Georgia.
³ Centre for Haematology, Imperial College, London, United Kingdom.
⁴ Patient Advocate, Jonesboro, Georgia.
⁵ Myeloproliferative Neoplasms Program, Memorial Sloan Kettering Cancer Center, New York City, New York.
⁶ Fred Hutchinson Cancer Research Center, Seattle Cancer Care Alliance, Seattle, Washington.
⁷ Patient Advocate, Los Angeles, California.
⁸ Department of Hematology-Oncology, Karmanos Cancer Center at Wayne State University, Detroit, Michigan.
⁹ Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Washington, DC.
¹⁰ Oncology Center of Excellence, U.S. Food and Drug Administration, Washington, DC.
¹¹ The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland.

Abstract

The FDA has an accelerated approval program for drugs that have been identified as promising treatments for serious conditions when the available data suggest that the benefits outweigh the foreseeable risks. All of the currently available treatment options for chronic myeloid leukemia (CML) initially went through the accelerated approval program. Here, a group of academic CML experts, patient panelists, and members from the FDA convened to discuss the utility of the accelerated approval program as it pertains to CML, and the utility of this program in future drug development in this disease. The results of that discussion are summarized here.

Publication types

Research Support, N.I.H., Intramural

MeSH terms

Drug Approval*
Humans
Leukemia, Myelogenous, Chronic, BCR-ABL Positive* / drug therapy
United States
United States Food and Drug Administration

Grants and funding

FD999999/ImFDA/Intramural FDA HHS/United States