A cost-benefit analysis of a cardiovascular disease prevention trial, using folate supplementation as an example.
OBJECTIVE
This study illustrates a cost-benefit analysis of clinical trial design, using as an example a trial of folate supplementation to prevent cardiovascular disease.


METHODS
Bayesian statistical and decision-analytic techniques were used to estimate the cost-benefit and sample size of a placebo-controlled trial of folate targeted to US citizens, aged 35 to 84 years, with elevated serum homocysteine levels. The main end point is event-free survival (i.e., survival without new ischemic heart disease or stroke) at 5 years.


RESULTS
Because the screening cost and annual cost and inconvenience of taking folate is small compared with the consequences of stroke, ischemic heart disease, or death, the increase in 5-year event-free survival with folate that should compel the use of folate is just 1.1%. The sample size per group needed to establish this level of folate's medical effectiveness is estimated to be 17310. Such a trial would provide an expected societal cost-benefit savings exceeding $11 billion within 15 years.


CONCLUSIONS
This study illustrates how Bayesian methods may help in assessing the societal cost-benefit consequences of proposed disease prevention trials, deciding which trials are worth sponsoring, and designing cost-effective trials.
Hornberger
J
J
Department of Health Research and Policy, Department of Medicine, Stanford University School of Medicine, CA, USA.
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D001499:Bayes Theorem; D002318:Cardiovascular Diseases / Q000191:economics / Q000517:prevention & control*; D002986:Clinical Trials as Topic; D003362:Cost-Benefit Analysis; D003661:Decision Support Techniques; D018572:Disease-Free Survival; D005492:Folic Acid / Q000191:economics* / Q000627:therapeutic use*; D006801:Humans; D008875:Middle Aged
D005492:Folic Acid