POWER AND SAMPLE SIZE CALCULATIONS FOR A CLINICAL TRIAL CONSIDERING COST-EFFECTIVENESS AND STATISTICAL ERRORS
Author(s)
Kamae I, Yanagisawa S, Division of Health Informatics and Sciences, Graduate School of Medicine, Kobe University, Kobe, Japan
In the last meeting of ISPOR, we presented a paper that showed the actual cost-effectiveness of treatments in practice can be reduced by the influence of statistical errors such as type II (ß) and type III (?). OBJECTIVE: Based on that research, this paper developed a further theoretical framework for such estimation which provides a new method for power analysis of a clinical trial and proper translation of its outcomes in terms of cost-effectiveness. METHODS: Consider a statistical model of a clinical trial for two regimens: a new treatment TA and TB. Under the traditional two-sample binomial analysis, we theoretically expanded it into power and sample sized calculations with the cost-effectiveness ratios ?A and ?B, respectively, for the regimens TA and TB. Furthermore, we assume that the truth is TA is more cost-effective than TB. Then, the binomial analyses were generalized under the decision-analytic model that can reflect a reality in practice with three alternatives according to no error, ß and ? errors possibly contained in the statistical conclusion of the clinical trials for TA and TB. RESULTS: Two formulae were developed mathematically to incorporate cost-effectiveness; one is an expansion of the traditional formula for sample-size calculation with the cost-effectiveness ratios ?A and ?B, and the other a more realistic one with ?A and ?B, and the modified cost-effectiveness ratio adjusted by the statistical ß and ? errors using ?A and ?B. CONCLUSIONS: The newly developed formulae can offer a more theoretically sound description to design a clinical trial beyond the traditional techniques.
Conference/Value in Health Info
2000-11, ISPOR Europe 2000, Antwerp, Belgium
Value in Health, Vol. 3, No. 5 (September/October 2000)
Code
PMDM1
Topic
Methodological & Statistical Research
Topic Subcategory
Modeling and simulation
Disease
Multiple Diseases