Collection of health-economic data alongside clinical trials: Is there a future for piggyback evaluations?

TY - JOUR

T1 - Collection of health-economic data alongside clinical trials

T2 - Is there a future for piggyback evaluations?

AU - O'Sullivan, A K

AU - Thompson, D

AU - Drummond, M F

PY - 2005

Y1 - 2005

N2 - Objective: The objective of this article is to discuss issues surrounding the conduct of "piggyback evaluations," in which health-economic data are collected within an otherwise typical clinical trial.Methods: We review the methodologic literature on piggyback economic evaluations, as well as selected empiric studies. We summarize the challenges encountered in the conduct of these studies, alternative ways of addressing these challenges, and their future role in pharmacoeconomic research.Results: Piggyback evaluations have certain advantages over other types of pharmacoeconomic studies. An economic evaluation can benefit from the experimental design that maximizes the trial's internal validity, and it is often more practical to collect economic data alongside a trial rather than to fund a stand-alone economic study. However, piggyback evaluations are subject to problems deriving from the competing nature of clinical versus economic study objectives, which can give rise to tension in such fundamental aspects of study design as the selection of study subjects and sites; the extent of protocol-mandated health-care services; and the determination of sample size, length of follow-up, and the study comparator( s). Many solutions have been put forth in the literature to address these challenges.Conclusions: Piggyback evaluations can be an appropriate means to measure the economic impact of medical interventions, provided that the methodologic challenges are acknowledged and addressed within the context of each individual study. As long as a desire for patient-level data from clinical trials exists, there will be a need for piggyback economic evaluations in the future.

AB - Objective: The objective of this article is to discuss issues surrounding the conduct of "piggyback evaluations," in which health-economic data are collected within an otherwise typical clinical trial.Methods: We review the methodologic literature on piggyback economic evaluations, as well as selected empiric studies. We summarize the challenges encountered in the conduct of these studies, alternative ways of addressing these challenges, and their future role in pharmacoeconomic research.Results: Piggyback evaluations have certain advantages over other types of pharmacoeconomic studies. An economic evaluation can benefit from the experimental design that maximizes the trial's internal validity, and it is often more practical to collect economic data alongside a trial rather than to fund a stand-alone economic study. However, piggyback evaluations are subject to problems deriving from the competing nature of clinical versus economic study objectives, which can give rise to tension in such fundamental aspects of study design as the selection of study subjects and sites; the extent of protocol-mandated health-care services; and the determination of sample size, length of follow-up, and the study comparator( s). Many solutions have been put forth in the literature to address these challenges.Conclusions: Piggyback evaluations can be an appropriate means to measure the economic impact of medical interventions, provided that the methodologic challenges are acknowledged and addressed within the context of each individual study. As long as a desire for patient-level data from clinical trials exists, there will be a need for piggyback economic evaluations in the future.

KW - cost-effectiveness analysis

KW - economic evaluation

KW - methodology

KW - piggyback trial

KW - COST-EFFECTIVENESS

KW - TREATMENT PARADIGM

KW - RANDOMIZED-TRIAL

KW - MEDICAL RECORDS

KW - HYLAN G-F-20

KW - DESIGN

KW - CARE

KW - OUTCOMES

KW - ISSUES

KW - THERAPY

M3 - Article

SN - 1524-4733

VL - 8

SP - 67

EP - 79

JO - Value in Health

JF - Value in Health

IS - 1

ER -