The use of drugs for indications beyond those stipulated in the FDA label has been a physician prerogative that the FDA has approved of for more than 30 years. A high percentage of the use of drugs and biologics in cancer care is off-label. The introduction and utilization of many innovative drugs and biologics in oncology practice, combined with their significant expense, has focused the attention of payors and other involved constituencies on processes and programs that facilitate the appropriate, effective, and efficient use of such agents. Since the late 1980s, drug compendia have been one mechanism to arbitrate such appropriate use in terms of both payors and providers.
On December 17, 2010, NCCN convened the NCCN Oncology Policy Summit: Off-Label Drug Use and Compendia in Washington, DC. The NCCN Oncology Policy Summit examined the use of compendia by public and private payors, looking at issues such as categories of evidence, compendia processes, and availability of data, among others. Further, the Summit explored the existing guidance, and surrounding interpretation, on the legality of dissemination of various clinical and scientific documents that contain off-label uses. The Summit concluded in a roundtable with the Centers for Medicare and Medicaid Services (CMS) recognized compendia where the future of compendia, their use, and their response to current and future events were discussed.