Designing Comparative Effectiveness Research on Prescription Drugs

Lessons from the Clinical Trial Literature

The $1.1 billion authorized by Congress for comparative effectiveness research in early 2009 was welcomed with optimism. The funding was viewed as the stimulus that would lead to a stream of studies to directly compare the benefits and harms of competing medical treatments.

This infusion of resources has been well timed. Amid concern by physicians and patients about the unanticipated side effects of widely prescribed drugs such as rofecoxib (Vioxx) and rosiglitazone (Avandia), interest has grown in the potential for effectiveness research to reduce the risk and improve the evidence base that supports prescription drug decision-making. In addition, to payers concerned about the affordability of similar treatments with widely varying costs, more federally funded comparative effectiveness research offered the promise of helping control spending without sacrificing quality of care.

As comparative effectiveness research becomes a more prominent feature of clinical medicine, investigators and policy-makers would do well to seek lessons from prior examples of this type of research.

This analysis of previous examples revealed lessons in three key areas:

  1. Choice of comparison treatments;
  2. Time frame of study; and
  3. Widespread applicability of study results.

Based on their observations, the authors offer suggestions for increasing the clinical applicability of comparative effectiveness research, such as employing surrogate endpoints that meet a specific threshold of validity. Future trials that address these areas of concern hold the greatest promise for improving patients’ outcomes.

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