Level of evidence used in recommendations by the National Comprehensive Cancer Network (NCCN) guidelines beyond Food and Drug Administration approvals.

Autor:	Kurzrock R; Center for Personalized Cancer Therapy, University of California San Diego, Moores Cancer Center, San Diego, USA. Electronic address: rkurzrock@ucsd.edu., Gurski LA; National Comprehensive Cancer Network, Plymouth Meeting, Pennsylvania, USA., Carlson RW; National Comprehensive Cancer Network, Plymouth Meeting, Pennsylvania, USA; Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA; Stanford Cancer Institute, Stanford, California, USA., Ettinger DS; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA., Horwitz SM; Memorial Sloan Kettering Cancer Center, New York, New York, USA., Kumar SK; Mayo Clinic Cancer Center, Rochester, Minnesota, USA., Million L; Stanford Cancer Institute, Stanford, California, USA., von Mehren M; Fox Chase Cancer Center, Philadelphia, Pennsylvania, USA., Benson AB 3rd; Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, Illinois, USA.
Jazyk:	angličtina
Zdroj:	Annals of oncology : official journal of the European Society for Medical Oncology [Ann Oncol] 2019 Oct 01; Vol. 30 (10), pp. 1647-1652.
DOI:	10.1093/annonc/mdz232
Abstrakt:	Background: A previous analysis of 113 National Comprehensive Cancer Network® (NCCN®) recommendations reported that NCCN frequently recommends beyond Food and Drug Administration (FDA)-approved indications (44 off-label recommendations) and claimed that the evidence for these recommendations was weak. Methods: In order to determine the strength of the evidence, we carried out an in-depth re-analysis of the 44 off-label recommendations listed in the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®). Results: Of the 44 off-label recommendations, 14 were later approved by the FDA and/or are supported by randomized controlled trial (RCT) data. In addition, 13 recommendations were either very minor extrapolations from the FDA label (n = 8) or were actually on-label (n = 5). Of the 17 remaining extrapolations, 8 were for mechanism-based agents applied in rare cancers or subsets with few available treatment options (median response rate = 43%), 7 were based on non-RCT data showing significant efficacy (>50% response rates), and 2 were later removed from the NCCN Guidelines because newer therapies with better activity and/or safety became available. Conclusion: Off-label drug use is a frequent component of care for patients with cancer in the United States. Our findings indicate that when the NCCN recommends beyond the FDA-approved indications, the strength of the evidence supporting such recommendations is robust, with a significant subset of these drugs later becoming FDA approved or supported by RCT. Recommendations without RCT data are often for mechanism-based drugs with high response rates in rare cancers or subsets without effective therapies. (© The Author(s) 2019. Published by Oxford University Press on behalf of the European Society for Medical Oncology.)
Databáze:	MEDLINE
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