New Indications for Oncology Drugs

Background

Substantial uncertainty exists about the benefits and harms of many off-label uses of oncology drugs, creating an important challenge for clinical and health policy decision makers. Current approaches to research on new indications for cancer drugs that frequently do not incorporate the type and/or quality of evidence requested by post-regulatory decision-makers.  As a result, there are large evidence gaps that impair decision making at the individual and population levels, resulting in inconsistency in how prescribing for new indications occurs across prescribers and patient groups.  Simultaneously, coverage for new indications is similarly inconsistent.

The Government Accountability Office (GAO) reported that one-third of anticancer medication prescribing represented off-label use in 1991.[i] By 2005, off-label prescribing in oncology had increased to one-half to three-quarters, according to a 2005 survey by the National Comprehensive Cancer Network.[ii]  The widespread use of oncology drugs outside the indications approved by the FDA underscores one reason why oncology appears as an outlier and warrants a specific focus for research.  In 2009, off-label use accounted for about 20% of all prescriptions and over 50% of expensive chemotherapy drugs.[iii]

[iii] Gillick MR. Controlling Off-Label Medication Use. Ann Intern Med 2009;150:344-347.

Purpose and Methods

The Center for Medical Technology Policy (CMTP) conducted a six-month initiative with the overarching goal of developing recommendations on methods and strategies to improve the validity, relevance and consistency of clinical research designed to assess the comparative effectiveness and value of oncology drugs used for off-label indications. As part of this initiative, CMTP convened a one-day multi-stakeholder New Indications for Oncology Drugs Think Tank to improve the systematic generation of evidence about the effectiveness of oncology drugs for new indications, providing a neutral ground to establish the steps necessary for improved evidence for drug therapy in cancer. A series of semi-structured interviews was also held with approximately twenty-five leading experts and stakeholders.

Ultimately, this will address the unmet medical needs of oncology patients by establishing the value of oncology drugs in new indications.  To accomplish the goal, there is a need to reach consensus among experts and stakeholders on better ways to more frequently and consistently design, fund, and implement prospective clinical studies of new indications for oncology drugs that will be more informative to:

• Patients making decisions to enter, continue, or switch treatment
• Physicians making treatment recommendations
• Investigators designing clinical trials to improve treatment 
• Professional societies developing clinical guidelines
• Producers of compendia drug information
• Expert panels making decisions about compendia listing
• Payers making coverage and reimbursement decisions

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