Hostname: page-component-cd9895bd7-fscjk Total loading time: 0 Render date: 2024-12-19T06:53:26.314Z Has data issue: false hasContentIssue false

The Role of Pilot Studies in the Economic Evaluation of Health Technologies

Published online by Cambridge University Press:  10 March 2009

Michael Drummond
Affiliation:
University of York
Douglas Coyle
Affiliation:
Ottawa Civic Hospital

Abstract

An increasing number of economic evaluations are being conducted alongside clinical trials. While this practice offers the prospect of collecting comprehensive and accurate cost data, it requires considerable time and effort. In the case of clinical data, key analytic decisions such as which data to collect and sample size are often made with reference to smaller (pilot) trials. However, this approach is not normally followed in the case of economic evaluation.

This study was based on a recently completed health technology assessment comparing conventional radiotherapy with continuous hyperfractionated accelerated radiotherapy (CHART) for patients with head and neck cancer or carcinoma of the bronchus. In the full health technology assessment, cost data were available for 526 head and neck patients (314 CHART and 212 conventional therapy) and 286 bronchus patients (175 CHART and 109 conventional therapy). In order to simulate a pilot study, data were extracted for the patients recruited to both trials in the first 3 months. These were then compared with the full data set in order to assess whether such a pilot study would have given useful guidance on: a) the usefulness of undertaking a full study; b) the sample size required; and c) the important resource items for which comprehensive data collection would be required.

Pilot studies can be helpful in determining the likely advantages of undertaking full economic evaluations and in identifying important resource items. Therefore, it is important that clinical researchers and research funding bodies create the necessary time window to enable such studies to take place. However, formal sample size calculations are more difficult to perform on limited data, since they also require knowledge of the unit cost (or prices) to be attached to the resource items and the correlation between costs and clinical effects.

Type
General Essays
Copyright
Copyright © Cambridge University Press 1998

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

REFERENCES

1.Adams, M. E., McCall, N. T., Gray, D. T., et al. Economic analysis in randomized control trials. Medical Care, 1992, 30, 231–43.CrossRefGoogle ScholarPubMed
2.Burnard, B., Kernan, W. N., & Feinstein, A. R.Indexes and boundaries for ‘quantitative significance’ in statistical decisions. Journal of Clinical Epidemiology, 1990, 43, 1273–84.CrossRefGoogle Scholar
3.Chaudhary, M., & Stearns, S.Estimating confidence intervals for cost-effectiveness ratios: An example from a randomized trial. Statistics in Medicine, 1996, 15, 1447–58.3.0.CO;2-V>CrossRefGoogle ScholarPubMed
4.Drummond, M. F., & Davies, L. M.Economic analysis alongside clinical trials: Revisiting the methodological principles. International Journal of Technology Assessment in Health Care, 1991, 7, 561–73.CrossRefGoogle Scholar
5.Drummond, M. F., & O’Brien, B. J.Clinical importance, statistical significance and the assessment of economic and quality-of-life outcomes. Health Economics, 1993, 2, 205–12.CrossRefGoogle ScholarPubMed
6.Gafni, A., & Birch, S.Guidelines for the adoption of new technologies: A prescription for uncontrolled growth in expenditures and how to avoid the problem. Canadian Medical Association Journal, 1993, 148, 913–17.Google ScholarPubMed
7.Goddard, M., & Hutton, J.The costs of radiotherapy in cancer treatment. (CHE/YHEC Discussion Paper 48). York: Centre for Health Economics, 1988.Google Scholar
8.Hebert, P. C., Wells, G. A., Marshall, J. C., et al. Transfusion requirements in critical care: A pilot study. JAMA, 1995, 273, 1439–44.CrossRefGoogle ScholarPubMed
9.Laupacis, A., Feeny, D., & Detsky, A. S., et al. How attractive does a new technology have to be to warrant adoption and utilization? Tentative guidelines for using clinical and economic evaluations. Canadian Medical Association Journal, 1992, 146, 473–81.Google ScholarPubMed
10.Morris, J., Goddard, M., Coyle, D., & Drummond, M. F.Measuring cost and quality of life in radiotherapy treatments. CHE/YHEC Discussion Paper 112. York: Centre for Health Economics, University of York, 1993.Google Scholar
11.Naylor, C. D., Williams, I., Basinski, A., & Goel, V.Technology assessment and cost-effectiveness: Misguided guidelines? Canadian Medical Association Journal, 1993, 148, 921–24.Google ScholarPubMed
12.Netten, A., & Smart, S.Unit cost of community care 1992/93. Canterbury; Personal Social Services Research Unit, University of Kent, 1993.Google Scholar
13.O'Brien, B., Drummond, M. F., Labelle, R. J., & Willan, A.In search of power and significance: Issues in the design and analysis of stochastic cost-effectiveness studies in health care. Medical Care, 1994, 32, 150–63.CrossRefGoogle ScholarPubMed
14.Saunders, M. I., Dishe, S., Barrett, A. et al. Randomised multicentre trials of CHART versus conventional radiotherapy in head and neck. and non-small-cell lung cancer: An interim report. British Journal of Cancer, 1996, 73, 1455–62.CrossRefGoogle Scholar
15.Spiegelhalter, D. J., Gore, S. M., Jones, D. R. et al. Being economical with the costs. Leicester: University of Leicester, 1996. Department of Epidemiology and Public Health Technical Report (Statistics) 96–06.Google Scholar
16.Torgerson, D. J., Ryan, M., & Ratcliffe, J.Economics in sample size determination for clinical trials. Quarterly Journal of Medicine, 1995, 88, 517–21.Google ScholarPubMed
17.van Hout, B. A., Al, M. J., Gordon, G. S., & Rutten, F. F. H.Costs, effects and C/E ratios alongside a clinical trial. Health Economics, 1994, 3, 309–19.CrossRefGoogle Scholar