Study: Weak Evidence Used to Support NCCN Recommendations


A new study published in BMJ (online March 7, 2018; doi:10.1136/bmj.k668) suggests the strength of evidence cited by the National Comprehensive Cancer Network (NCCN) to support recommendations deviating from United States Food and Drug Administration (FDA)-approved therapy recommendations is not robust.

NCCN has provided Journal of Clinical Pathways with a response to this finding.

NCCN is one of the most influential organizations for the use of cancer therapies in the United States. When a drug for a particular indication is supported by the NCCN, reimbursement from government and private payers is often expected.

Vinay Prasad, MD, school of medicine, Oregon Health & Science University (Portland, OR), and colleagues conducted a study to determine the differences between recommendations by the NCCN guidelines and FDA approvals of anticancer drugs. Researchers also examined the evidence cited by the NCCN to justify recommendations where differences exist. The retrospective observational study assessed 47 new molecular entities approved by the FDA between 2011 and 2015 for adult hematologic or solid cancers.

The main outcome measures of the study were comparison of all FDA-approved indications with all NCCN recommendations as of March 2016. NCCN recommendations beyond the FDA’s approvals were classified and the cited evidence was noted.

Results of the study showed that the 47 drugs were authorized for 69 FDA-approved indications, whereas the NCCN recommended these drugs for 113 indications. Among these NCCN indications, 62% (n = 69) overlapped with the 69 FDA-approved indications and 39% (n = 44) were additional recommendations.

Researchers reported that the average number of recommendations beyond the FDA-approved indications was 0.92. A total of 10 (23%) additional recommendations were based on evidence from randomized controlled trials, while 7 (16%) additional recommendations were based on evidence from phase III studies.


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Additionally, researchers noted that during the 21 months of follow-up, the FDA granted approval to 6 (14%) of the additional recommendations.

After finding that the NCCN frequently recommends beyond the FDA-approved indications for newer drugs, researchers concluded, "Given that NCCN endorsement is linked to reimbursement by many commercial insurers and CMS, our results suggest that payers may be covering cancer drugs with varying and scientifically less robust justification."

In a statement responding to this study, Robert W Carlson, MD, chief executive officer, NCCN, told Journal of Clinical Pathways, "The NCCN Clinical Practice Guidelines for Oncology (NCCN Guidelines) are continuously updated based on the strongest scientific evidence available. NCCN’s 1,355 panel members come from 27 leading academic cancer centers in the United States. Their expertise allows them to evaluate complex circumstances based on all available data, in order to come to a consensus about what constitutes optimal care."

Dr Carlson continued: "The NCCN process is designed to make sure the most effective, live-saving therapies are accessible and available to the patients who need them. CMS and other major providers have designated NCCN as the leading source for arbitrating oncology drug and biologic coverage because of the NCCN Guidelines’ proven track record for helping physicians to prolong their patients’ lives and reduce their suffering" (March 8, 2018).Zachary Bessette