G G Liu1, J Hay. 1. University of North Carolina at Chapel Hill, Department of Pharmaceutical Policy and Evaluative Sciences, 27599-7360, USA. ggliu@unc.edu
Abstract
PURPOSE: The study conducted an economic cost analysis of oral ganciclovir prophylaxis in preventing cytomegalovirus (CMV) disease for AIDS patients in a randomized clinical trial setting. METHODS: Data were generated from patient interviews, medical records, and case reports from a multi-center, randomized, double-blind, and placebo-controlled pharmacoeconomic study appended to a clinical trial. The outcomes were measured in monthly cost per patient. Various cost functions were tested in the context of sample-selection model (SSM) and two-part model (TPM), and were estimated using both the ordinary least squares (OLS) and the bounded influence estimation (BIE) methods. RESULTS: The use of informal caregiver services did not differ significantly between patients in the treatment group and those in the placebo group. The OLS estimates for the ganciclovir prophylaxis arm showed a reduced, but statistically insignificant use of formal care in both outpatient and inpatient settings. The BIE results for the ganciclovir prophylaxis arm, in contrast, showed a significant reduction of 27% in hospital cost among hospital users, and 44% among the total sample of AIDS patients. The monthly total cost function also identified a decreasing but insignificant trend due to the treatment effect. CONCLUSIONS: At the methodological level, this study demonstrated the value of employing more rigorous econometric techniques in identifying subtle treatment effects on cost outcomes from clinical trial data in the economic assessment of medical technologies. At the empirical level, the study concluded that beyond its demonstrated efficacy of preventing CVM disease among AIDS patients, ganciclovir prophylaxis did not lead to additional health care costs, other than the cost of the drug therapy.
RCT Entities:
PURPOSE: The study conducted an economic cost analysis of oral ganciclovir prophylaxis in preventing cytomegalovirus (CMV) disease for AIDSpatients in a randomized clinical trial setting. METHODS: Data were generated from patient interviews, medical records, and case reports from a multi-center, randomized, double-blind, and placebo-controlled pharmacoeconomic study appended to a clinical trial. The outcomes were measured in monthly cost per patient. Various cost functions were tested in the context of sample-selection model (SSM) and two-part model (TPM), and were estimated using both the ordinary least squares (OLS) and the bounded influence estimation (BIE) methods. RESULTS: The use of informal caregiver services did not differ significantly between patients in the treatment group and those in the placebo group. The OLS estimates for the ganciclovir prophylaxis arm showed a reduced, but statistically insignificant use of formal care in both outpatient and inpatient settings. The BIE results for the ganciclovir prophylaxis arm, in contrast, showed a significant reduction of 27% in hospital cost among hospital users, and 44% among the total sample of AIDSpatients. The monthly total cost function also identified a decreasing but insignificant trend due to the treatment effect. CONCLUSIONS: At the methodological level, this study demonstrated the value of employing more rigorous econometric techniques in identifying subtle treatment effects on cost outcomes from clinical trial data in the economic assessment of medical technologies. At the empirical level, the study concluded that beyond its demonstrated efficacy of preventing CVM disease among AIDSpatients, ganciclovir prophylaxis did not lead to additional health care costs, other than the cost of the drug therapy.
Authors: S A Spector; G F McKinley; J P Lalezari; T Samo; R Andruczk; S Follansbee; P D Sparti; D V Havlir; G Simpson; W Buhles; R Wong; M Stempien Journal: N Engl J Med Date: 1996-06-06 Impact factor: 91.245