Quantitative benefit-harm assessment for setting research priorities

The example of roflumilast for patients with COPD

Milo A. Puhan, Tsung Yu, Cynthia Boyd, Gerben ter Riet

Research output: Contribution to journalArticle

Abstract

Background: When faced with uncertainties about the effects of medical interventions regulatory agencies, guideline developers, clinicians, and researchers commonly ask for more research, and in particular for more randomized trials. The conduct of additional randomized trials is, however, sometimes not the most efficient way to reduce uncertainty. Instead, approaches such as value of information analysis or other approaches should be used to prioritize research that will most likely reduce uncertainty and inform decisions. Discussion: In situations where additional research for specific interventions needs to be prioritized, we propose the use of quantitative benefit-harm assessments that illustrate how the benefit-harm balance may change as a consequence of additional research. The example of roflumilast for patients with chronic obstructive pulmonary disease shows that additional research on patient preferences (e.g., how important are exacerbations relative to psychiatric harms?) or outcome risks (e.g., what is the incidence of psychiatric outcomes in patients with chronic obstructive pulmonary disease without treatment?) is sometimes more valuable than additional randomized trials. Summary: We propose that quantitative benefit-harm assessments have the potential to explore the impact of additional research and to identify research priorities Our approach may be seen as another type of value of information analysis and as a useful approach to stimulate specific new research that has the potential to change current estimates of the benefit-harm balance and decision making.

Original languageEnglish (US)
Article number157
JournalBMC Medicine
Volume13
Issue number1
DOIs
StatePublished - Jul 2 2015

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Chronic Obstructive Pulmonary Disease
Research
Uncertainty
Psychiatry
Roflumilast
Patient Preference
Decision Making
Research Personnel
Guidelines
Incidence

Keywords

  • Benefit-harm assessment
  • Chronic obstructive pulmonary disease
  • Randomized trials
  • Research priorities

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Quantitative benefit-harm assessment for setting research priorities : The example of roflumilast for patients with COPD. / Puhan, Milo A.; Yu, Tsung; Boyd, Cynthia; ter Riet, Gerben.

In: BMC Medicine, Vol. 13, No. 1, 157, 02.07.2015.

Research output: Contribution to journalArticle

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