oncology guidelines off-label drug use

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

by R. Kurzrock, L.A. Gurski, R.W. Carlson, D.S. Ettinger, S.M. Horwitz, S.K. Kumar, L. Million, M. von Mehren, A.B. Benson III

Aug 2^nd 2019
8 mins
Oncology practice| Volume 30, ISSUE 10, P1647-1652, October 2019

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Summary

The analysis reviewed 113 NCCN recommendations, focusing on 44 off-label uses of drugs.
14 of these off-label recommendations were later FDA-approved or backed by RCT data.
13 recommendations were minor extrapolations from the FDA label or actually on-label.
Of the remaining 17 extrapolations:
- 8 were for mechanism-based agents in rare cancers with limited treatment options (median response rate = 43%).
- 7 were based on non-RCT data showing significant efficacy (>50% response rates).
- 2 were removed from guidelines due to newer, more effective therapies.
Off-label drug use is common in cancer treatment, and NCCN's recommendations are generally well-supported by robust evidence.

The National Comprehensive Cancer Network® (NCCN®) is a not-for-profit alliance of 28 leading cancer centers in the United States devoted to patient care, research, and education. The NCCN mission includes improving the quality, effectiveness, and efficiency of cancer care so that patients can live better lives, and, in order to do so, NCCN promotes continuous quality improvement and creates clinical practice guidelines appropriate for use by patients, clinicians, and other health care decision-makers. The NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) comprised recommendations for the prevention, diagnosis, and management of malignancies across the continuum of care.

he guidelines currently apply to over 97% of patients living with cancer in the United States and incorporate real-time updates of the rapid advances in cancer research . The NCCN Guidelines are used worldwide, with nearly half (46.6%) of registered users being from outside the United States and guideline downloads from over 180 countries.

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oncology, guidelines, off-label drug use