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Cost-effectiveness analysis: discount the placebo at your peril

Cost-effectiveness analysis: discount the placebo at your peril
Cost-effectiveness analysis: discount the placebo at your peril

INTRODUCTION: The authors consider alternative mechanisms that might explain placebo responses and their implications for cost-effectiveness modeling. Three alternative placebo mechanisms are examined: a ''regression to the mean'' effect arising from natural variation and the preferential selection of patients with acutely severe disease into clinical trials, a patient expectancy effect specific to the clinical trial setting (Hawthorne effect), and a patient expectancy effect generalizable to routine clinical practice (true placebo effect).

METHODS: To estimate cost-effectiveness, the authors needed to generalize from trial data to estimate responses to treatment that they would see in routine clinical practice. They use an example analysis of the cost-effectiveness of adjunct epilepsy treatments to illustrate the potential effects of these different placebo mechanisms on this generalization and subsequent cost-effectiveness estimates and adoption decisions.

RESULTS: If an acceptable willingness-to-pay threshold of 30,000 per quality-adjusted life year (QALY) is assumed, then each of the placebo effect scenarios identifies a different treatment alternative as being optimum.

DISCUSSION: Estimated cost-effectiveness ratios and associated policy decisions may be sensitive to assumptions regarding the mechanism underlying placebo responses. These assumptions should, if possible, be investigated through analysis of trial or observational data and, in the absence of other evidence, sensitivity analysis.

Clinical Trials as Topic, Cost-Benefit Analysis, Epilepsy/drug therapy, Humans, Models, Economic, Outcome Assessment, Health Care, Patient Care/economics, Placebo Effect, Quality-Adjusted Life Years
0272-989X
536-543
Hawkins, Neil
1aa8112d-606d-4176-b306-9c22158c556d
Scott, David A
19b5fd34-9974-4ae4-8be0-27a693639e20
Hawkins, Neil
1aa8112d-606d-4176-b306-9c22158c556d
Scott, David A
19b5fd34-9974-4ae4-8be0-27a693639e20

Hawkins, Neil and Scott, David A (2010) Cost-effectiveness analysis: discount the placebo at your peril. Medical Decision Making, 30 (5), 536-543. (doi:10.1177/0272989X10362106).

Record type: Article

Abstract

INTRODUCTION: The authors consider alternative mechanisms that might explain placebo responses and their implications for cost-effectiveness modeling. Three alternative placebo mechanisms are examined: a ''regression to the mean'' effect arising from natural variation and the preferential selection of patients with acutely severe disease into clinical trials, a patient expectancy effect specific to the clinical trial setting (Hawthorne effect), and a patient expectancy effect generalizable to routine clinical practice (true placebo effect).

METHODS: To estimate cost-effectiveness, the authors needed to generalize from trial data to estimate responses to treatment that they would see in routine clinical practice. They use an example analysis of the cost-effectiveness of adjunct epilepsy treatments to illustrate the potential effects of these different placebo mechanisms on this generalization and subsequent cost-effectiveness estimates and adoption decisions.

RESULTS: If an acceptable willingness-to-pay threshold of 30,000 per quality-adjusted life year (QALY) is assumed, then each of the placebo effect scenarios identifies a different treatment alternative as being optimum.

DISCUSSION: Estimated cost-effectiveness ratios and associated policy decisions may be sensitive to assumptions regarding the mechanism underlying placebo responses. These assumptions should, if possible, be investigated through analysis of trial or observational data and, in the absence of other evidence, sensitivity analysis.

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More information

Published date: 12 March 2010
Keywords: Clinical Trials as Topic, Cost-Benefit Analysis, Epilepsy/drug therapy, Humans, Models, Economic, Outcome Assessment, Health Care, Patient Care/economics, Placebo Effect, Quality-Adjusted Life Years

Identifiers

Local EPrints ID: 441396
URI: http://eprints.soton.ac.uk/id/eprint/441396
ISSN: 0272-989X
PURE UUID: 1c5c4249-14bd-428a-a09f-9880f7c5f19b
ORCID for David A Scott: ORCID iD orcid.org/0000-0001-6475-8046

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Date deposited: 11 Jun 2020 16:30
Last modified: 17 Mar 2024 04:02

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Contributors

Author: Neil Hawkins
Author: David A Scott ORCID iD

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