Effects of Trial Design on Participation and Costs in Clinical Trials With an Examination of Cost Analysis Methods and Data Sources

by Meredith L. Kilgore

Download eBook for Free

Full Document

FormatFile SizeNotes
PDF file 0.8 MB

Use Adobe Acrobat Reader version 10 or higher for the best experience.

Summary Only

FormatFile SizeNotes
PDF file 0.2 MB

Use Adobe Acrobat Reader version 10 or higher for the best experience.

This dissertation comprises a series of studies conducted as part of the Cost of Cancer Treatment Study (CCTS). 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 patients could be accounted for by exclusion criteria based on comorbid conditions that disproportionately afflict the elderly. The author compared data from patient interviews, medical records abstraction, provider billing records, and Medicare claims as data sources for estimating health care utilization rates and costs; the data were compared in terms of completeness and accessibility. 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.

Table of Contents

  • Chapter One

    Introduction

  • Chapter Two

    The Effect of Clinical Trial Design on Participation Rates of Elderly Cancer Patients

  • Chapter Three

    Comparing Data Sources for Health Services Research

  • Chapter Four

    Pricing Health Service Utilization Measures Using Medicare SEER Linked Data

  • Chapter Five

    The Effect of Clinical Trial Participation on Prescription Drug Costs and Out of Pocket Expenses

  • Chapter Six

    Conclusion

Research conducted by

This report is part of the RAND Corporation dissertation series. Pardee RAND dissertations are produced by graduate fellows of the Pardee RAND Graduate School, the world's leading producer of Ph.D.'s in policy analysis. The dissertations are supervised, reviewed, and approved by a Pardee RAND faculty committee overseeing each dissertation.

Permission is given to duplicate this electronic document for personal use only, as long as it is unaltered and complete. Copies may not be duplicated for commercial purposes. Unauthorized posting of RAND PDFs to a non-RAND Web site is prohibited. RAND PDFs are protected under copyright law. For information on reprint and linking permissions, please visit the RAND Permissions page.

The RAND Corporation is a nonprofit institution that helps improve policy and decisionmaking through research and analysis. RAND's publications do not necessarily reflect the opinions of its research clients and sponsors.