Program on Prevention Outcomes and Practices

Developing Evidence-Based Research Priorities for
Off-Label Drug Use

Subaward Principal Investigator: Randall S. Stafford, MD, PHD
Overall Principal Investigator: Surrey Walton, PHD
Funding Agency: Agency for Healthcare Research and Quality
Duration: 09/15/2006 - 01/29/2007

The frequency of off-label use lacking scientific support indicates an overwhelming need for better information on safety, outcomes, and costs.  However, given finite resources there is a need to prioritize research on off-label drug use so that future efforts can target those drugs where research is likely to be most valuable.  This project will provide an evidence-based analysis to develop a prioritized list of drugs used in adult outpatient care where future research on off-label uses is expected to generate the greatest return on research efforts.  Key initial aspects of the project include a review of current sources of information on off-label use and the recruitment of a set of specialized individuals to form a Technical Expert Panel (TEP) to best inform the prioritization process and provide final consensus on a specific prioritized list of drugs.  We will then analyze the National Disease and Therapeutic Index (NDTI) and DRUGDEX data to examine the frequency and associated scientific support of off-label drug use, create a set of conceptual criteria that can be used to rank the potential importance of drugs in terms of their off-label use, and identify criteria that are measurable with available data.  Finally, we will construct a cumulative priority score of current drugs via quantitative analysis of measurable criteria, create a final prioritized drug list based on each of the final criteria and consensus from the Technical Expert Panel, and use a secondary data source (from the federal Ambulatory Care Surveys) to provide additional insights into the use of top priority drugs.  This secondary data will provide a means to assess limitations of the primary data source, use an alternative method for defining off-label use, and cover outpatient settings under-represented in NDTI.  The project will serve as an important first step in prioritizing and focusing future research on the safety, outcomes, and costs of off-label prescribing.

Specific Aims


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