CHILD POLICY
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Effects of Trial Design on
Participation and Costs in
Clinical Trials, with an
Examination of Cost
Analysis Methods and
Data Sources
MEREDITH L. KILGORE
This document was submitted as a dissertation in March 2004 in partial
fulfillment of the requirements of the doctoral degree in public policy analysis at
the Pardee RAND Graduate School. The faculty committee that supervised and
approved the dissertation consisted of Jacob Klerman (Chair), Dana Goldman,
and Emmett Keeler.
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ABSTRACT
This dissertation comprises a series of studies conducted as part of the Cost of Cancer
Treatment Study (CCTS). The specific aims include exploring theoretical issues concerning the
problem of representativeness in trial design with an explicit investigation of the causes of the
under-representation of older adults in clinical cancer trials; comparing sources of data and
modeling approaches for estimating treatment costs in health services research; and estimating
the impact of clinical trial participation on prescription drug costs.
An exploration of the sample size requirements for power and significance levels
in clinical trials suggests that proportional representation of subpopulations in trials will often
not allow valid inferences to be drawn about differential treatment effects. Where differential
treatment effects in subpopulations are suspected, targeted trials should be undertaken. Underrepresentation of older cancer could be accounted for by exclusion criteria based on comorbid
conditions that disproportionately afflict the elderly.
Data from patient interviews, medical records abstraction, provider billing
records, and Medicare claims were compared as data sources for estimating health care
utilization rates and costs; the data were compared in terms of completeness and accessibility.
Medicare claims contain data on all covered services, including charges, and reimbursements.
The costs of Medicare data compare favorably with other sources of comparable quality, but
claims data are missing for individuals in managed care and do not include information on
prescription drugs. Provider billing records, however, constituted a poor data source, primarily
because providers were unwilling or unable to provide these records. Medical records provide
accessible, detailed data on service utilization, but not costs. Self-reported health services
utilization generally agreed with other sources on inpatient care but not with respect to outpatient
services. Cost estimates for utilization measures were derived from administrative data using
hedonic regression models.
Prescription drug costs and out-of-pocket drug expenditures were compared for
patients enrolled in cancer trials and for similar cancer patients with who did not participate in
trials. Trial participation was associated with higher prescription drug costs, but that did not
result in any significant difference in out-of-pocket expenditures for participants. These results
were robust to a variety of modeling approaches.
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