Feature Medical Research

Creating credibility and value in comparative effectiveness research

BMJ 2012; 345 doi: https://doi.org/10.1136/bmj.e7302 (Published 01 November 2012) Cite this as: BMJ 2012;345:e7302
  1. Bob Roehr, freelance journalist
  1. 1Washington, DC, USA
  1. BobRoehr{at}aol.com

Bob Roehr examines the difficulties of conducting research to help doctors and patients make better treatment decisions

The unsustainable rise in healthcare costs and the transition from fee for service reimbursement to payments that are capitated, bundled, or based on outcomes is generating renewed interest in comparative effectiveness research across the spectrum of healthcare stakeholders in the United States.

However, such research, which compares existing healthcare interventions to determine which work best for which patients and which pose the greatest benefits and harms, will be accepted and integrated into practice only if there is consensus on what a study’s results truly mean, according to a cross section of experts.

Failure to reach consensus on what a study has the potential to show beforehand can fuel ongoing controversy, such as those over the timing and frequency of mammography in screening for breast cancer and interventions for prostate cancer and back pain, they said.

This discussion grew out of a recent exercise and series of papers organized by the journal Health Affairs.1 2 The exercise shows the difficulty of achieving agreement after the fact, and points to how the processes of comparative effectiveness research must change if it is to be embraced.

The exercise centered on a fictional study: a retrospective analysis of electronic health records comparing those who did not receive treatment with those who received two drugs for migraine that had different trade-offs in efficacy, ease of use, adherence, side effects, and cost.

The hypothetical study fell short on being patient centered in three important ways, said Rachael Fleurence, a scientist with the newly established Patient-Centered Outcomes Research Institute (PCORI). The quasi-governmental agency was created as part of the US Affordable Care Act to advance comparative effectiveness research.

Fleurence said that the mock study did not generate information …

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