Format

Send to

Choose Destination
BMJ. 2018 Mar 7;360:k668. doi: 10.1136/bmj.k668.

Frequency and level of evidence used in recommendations by the National Comprehensive Cancer Network guidelines beyond approvals of the US Food and Drug Administration: retrospective observational study.

Author information

1
School of Medicine, Oregon Health & Science University, Portland, OR 97239, USA.
2
Divison of Hematology Oncology, Knight Cancer Institute, Oregon Health & Science University, Portland, OR, USA.
3
Division of Myeloma, Memorial Sloan Kettering Cancer Center, New York, NY, USA.
4
Divison of Hematology Oncology, Knight Cancer Institute, Oregon Health & Science University, Portland, OR, USA prasad@ohsu.edu.
5
Department of Public Health and Preventive Medicine, Oregon Health & Science University, Portland, OR, USA.
6
Center for Health Care Ethics, Oregon Health & Science University, Portland, OR, USA.

Abstract

OBJECTIVE:

To determine the differences between recommendations by the National Comprehensive Cancer Network (NCNN) guidelines and Food and Drug Administration approvals of anticancer drugs, and the evidence cited by the NCCN to justify recommendations where differences exist.

DESIGN:

Retrospective observational study.

SETTING:

National Comprehensive Cancer Network and FDA.

PARTICIPANTS:

47 new molecular entities approved by the FDA between 2011 and 2015.

MAIN OUTCOME MEASURES:

Comparison of all FDA approved indications (new and supplemental) with all NCCN recommendations as of 25 March 2016. When the NCCN made recommendations beyond the FDA's approvals, the recommendation was classified and the cited evidence noted.

RESULTS:

47 drugs initially approved by the FDA between 2011 and 2015 for adult hematologic or solid cancers were examined. These 47 drugs were authorized for 69 FDA approved indications, whereas the NCCN recommended these drugs for 113 indications, of which 69 (62%) overlapped with the 69 FDA approved indications and 44 (39%) were additional recommendations. The average number of recommendations beyond the FDA approved indications was 0.92. 23% (n=10) of the additional recommendations were based on evidence from randomized controlled trials, and 16% (n=7) were based on evidence from phase III studies. During 21 months of follow-up, the FDA granted approval to 14% (n=6) of the additional recommendations.

CONCLUSION:

The NCCN frequently recommends beyond the FDA approved indications even for newer, branded drugs. The strength of the evidence cited by the NCCN supporting such recommendations is weak. Our findings raise concern that the NCCN justifies the coverage of costly, toxic cancer drugs based on weak evidence.

PMID:
29514787
PMCID:
PMC5838851
DOI:
10.1136/bmj.k668
[Indexed for MEDLINE]
Free PMC Article

Conflict of interest statement

Competing interests: All authors have completed the ICMJE uniform disclosure form at www.icmje.org/coi_disclosure.pdf and declare: no support from any organization for the submitted work; no financial relationships with any organizations that might have an interest in the submitted work in the previous three years; no other relationships or activities that could appear to have influenced the submitted work.

Supplemental Content

Full text links

Icon for HighWire Icon for PubMed Central
Loading ...
Support Center