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NCCN Oncology Policy Summit: Off-Label Drug Use and Compendia

By Edward Li, PharmD, BCOP, Drugs and Biologics Editor

The issue of utilizing oncology drugs for indications beyond the FDA-approved labeling (i.e., "off-label drug use") has recently garnered heightened awareness, especially in today's climate of controlling health care costs. While some scrutinize the off-label use of oncology drugs, this matter is complex. First, one must distinguish between appropriate off-label drug use and inappropriate off-label drug use. In the case of appropriate off-label use, the efficacy of a drug has been evaluated through clinical studies and/or expert judgment, resulting in an affirmative recommendation for use. On the other hand, inappropriate off-label use means that a drug has not demonstrated (through clinical studies or expert judgment) any benefit for that particular use. Therefore, patients for whom these uses are prescribed may be unnecessarily exposed to the toxicity of the agent. The discussion about minimizing off-label drug use should revolve around these inappropriate indications.

In the oncology setting, the FDA-approved indications for anti-cancer drugs tend to be narrowly focused in more recent years, typically reflecting the inclusion criteria and trial design of the pivotal trial(s) that were submitted to obtain FDA approval. However, the FDA label does not always reflect the most current evidence regarding the appropriate use of certain medications. For example, the FDA-approved indications for carboplatin are limited to the setting of ovarian cancer. However, many clinical trials have demonstrated carboplatin's usefulness in various other cancer types (such as breast and lung cancers to name a few). Complicating matters even further is the fact that on-label indications for other drugs may contradict this particular example's label. Specifically, bevacizumab is FDA-approved for the treatment of non-small cell lung cancer (NSCLC) in combination with carboplatin and paclitaxel. In this case, the use of bevacizumab in NSCLC is considered "on-label" only if it is given with another drug that is given "off-label." Conflicts such as these are the result of limitations in the current regulatory process of reviewing the most current evidence and modifying the product label.

To bridge this evidence-assessment gap, compendia can provide guidance to the appropriateness of off-label uses of cancer medications. Compendia contain an index of drugs and their corresponding body of knowledge and typically include drug information related to pharmacology and pharmacokinetic properties (dosing, adverse effects, and drug interactions) and indications (both FDA-approved and off-label). However, with the advent of molecular markers, calls for greater public transparency, availability of compendia information, use and categorization of existing evidence, and the use of comparative effectiveness – future utilization of compendia may have expanded applications.

To discuss these issues and those related to categories of evidence, compendia processes, and availability of data, NCCN is convening key policy organizations and thought leaders at an upcoming NCCN Oncology Policy Summit: Off-Label Drug Use and Compendia. This invitation-only Policy Summit will be held on December 17, 2010 at the National Press Club in Washington, DC.

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