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Evidence selection for a prescription drug's benefit-harm assessment: challenges and recommendations


Fain, Kevin M; Yu, Tsung; Li, Tianjing; Boyd, Cynthia M; Singh, Sonal; Puhan, Milo A (2016). Evidence selection for a prescription drug's benefit-harm assessment: challenges and recommendations. Journal of Clinical Epidemiology, 74:151-157.

Abstract

OBJECTIVES: To describe challenges and make recommendations for researchers in how they select evidence to quantitatively assess a prescription drug's benefits and harms.
STUDY DESIGN AND SETTING: These challenges and recommendations are based on our recent experience conducting a benefit-harm assessment for the prescription drug roflumilast. We considered the selection of evidence to quantify (1) the drug's treatment effects in patients, (2) the patient population's baseline risks for beneficial and harmful outcomes without treatment, and (3) the patient population's preferences for these beneficial effects and harms. These are fundamental steps for most benefit-harm assessment methods.
RESULTS: We identify critical issues in selecting evidence for each of these steps. We justify in particular the need to incorporate (1) clinical trials for the drug's specific treatment effect; (2) observational studies with the most valid, precise, and applicable effect estimates for the baseline risk; and (3) flexible weighting approaches for balancing the drug benefits and harms.
CONCLUSION: We identify challenges and make recommendations for selecting evidence at the critical steps in a prescription drug's benefit-harm assessment. Our findings should assist other researchers conducting these assessments for prescription drugs, which could help regulators, medical professionals, and patients make better decisions about prescription drug use.

Abstract

OBJECTIVES: To describe challenges and make recommendations for researchers in how they select evidence to quantitatively assess a prescription drug's benefits and harms.
STUDY DESIGN AND SETTING: These challenges and recommendations are based on our recent experience conducting a benefit-harm assessment for the prescription drug roflumilast. We considered the selection of evidence to quantify (1) the drug's treatment effects in patients, (2) the patient population's baseline risks for beneficial and harmful outcomes without treatment, and (3) the patient population's preferences for these beneficial effects and harms. These are fundamental steps for most benefit-harm assessment methods.
RESULTS: We identify critical issues in selecting evidence for each of these steps. We justify in particular the need to incorporate (1) clinical trials for the drug's specific treatment effect; (2) observational studies with the most valid, precise, and applicable effect estimates for the baseline risk; and (3) flexible weighting approaches for balancing the drug benefits and harms.
CONCLUSION: We identify challenges and make recommendations for selecting evidence at the critical steps in a prescription drug's benefit-harm assessment. Our findings should assist other researchers conducting these assessments for prescription drugs, which could help regulators, medical professionals, and patients make better decisions about prescription drug use.

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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > Epidemiology, Biostatistics and Prevention Institute (EBPI)
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:June 2016
Deposited On:13 Mar 2017 10:18
Last Modified:22 Nov 2017 17:29
Publisher:Elsevier
ISSN:0895-4356
Publisher DOI:https://doi.org/10.1016/j.jclinepi.2016.02.018
PubMed ID:26939932

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