evidence-based compendium focused specifically on off-label uses of drugs for rare diseases could inform clinicians, health plans, and potentially patients and families. Such a compendium is not likely to be feasible for commercial publishers but could be undertaken by a public agency such as AHRQ that has experience in similar analyses. Based on experience with the U.S. Pharmacopeia, which developed one of the early compendia but discontinued it because sales did not cover expenses, the estimated potential cost of a pilot project to develop and update a pilot compendium is within the range of current AHRQ grants (Dr. Roger Williams, CEO, U.S. Pharmacopeia, June 2010, personal communication to Carolyn Asbury [Committee member].)

RECOMMENDATION 6-2: The Agency for Healthcare Research and Quality or a similar appropriate agency should undertake a pilot project to develop an evidence-based compendium to inform health plan decisions on both orphan and nonorphan drugs that may have indications for rare conditions that have not been evaluated or approved by FDA.

Some of the issues that such a pilot effort would confront include determining a focus (e.g., rare cancers, rare metabolic disorders), establishing criteria for evaluating research that involves small numbers of participants and nontraditional research designs, and exploring the use of Medicare or Part D claims data for analyses to supplement the review of published studies. Depending on its experience, AHRQ or another agency could propose a strategy for updating or expanding the compendium.

In the process of developing the compendium, analysts may also identify directions for future research on specific drugs to demonstrate efficacy, side effects, or optimum dosage. If not undertaken by industry, such studies might be supported through National Institutes of Health awards, FDA orphan products grants, AHRQ grants to its Centers for Education and Research on Therapeutics, or other grant programs.

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