title: Statistical determination of cost-effectiveness frontier based on net health benefits.
abstract: Statistical methods are given for producing a cost-effectiveness frontier for an arbitrary number of programs. In the deterministic case, the net health benefit (NHB) decision rule is optimal; the rule funds the program with the largest positive NHB at each lambda, the amount a decision-maker is willing to pay for an additional unit of effectiveness. For bivariate normally distributed cost and effectiveness variables and a specified lambda, a statistical procedure is presented, based on the method of constrained multiple comparisons with the best (CMCB), for determining the program with the largest NHB. A one-tailed t test is used to determine if the NHB is positive. To obtain a statistical frontier in the lambda-NHB plane, we develop a method to produce the region in which each program has the largest NHB, by pivoting a CMCB confidence interval. A one-sided version of Fieller's theorem is used to determine the region where the NHB of each program is positive. At each lambda, the pointwise error rate is bounded by a prespecified alpha. Upper bounds on the familywise error rate, the probability of an error at any value of lambda, are given. The methods are applied to a hypothetical clinical trial of antipsychotic agents.
authors.affiliation: Statistical Sciences & Epidemiology Division, The Nathan S. Kline Institute for Psychiatric Research, Orangeburg, NY 10962, USA. laska@nki.rfmh.org
authors.forename: Eugene M
authors.initials: EM
authors.lastname: Laska
authors.forename: Morris
authors.initials: M
authors.lastname: Meisner
authors.forename: Carole
authors.initials: C
authors.lastname: Siegel
authors.forename: Joseph
authors.initials: J
authors.lastname: Wanderling
mesh_terms: D000465:Algorithms; D014150:Antipsychotic Agents / Q000191:economics / Q000627:therapeutic use; D016001:Confidence Intervals; D003362:Cost-Benefit Analysis / Q000706:statistics & numerical data*; D003657:Decision Making; D017048:Health Care Costs / Q000706:statistics & numerical data*; D006801:Humans; D015233:Models, Statistical; D015397:Program Evaluation / Q000191:economics* / Q000379:methods; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data*; D012559:Schizophrenia / Q000188:drug therapy / Q000191:economics; D013269:Stochastic Processes; D016896:Treatment Outcome
chemical_list: D014150:Antipsychotic Agents