A MULTINATIONAL ECONOMIC-EVALUATION OF RHDNASE IN THE TREATMENT OF CYSTIC-FIBROSIS

Economic evaluations of pharmaceuticals are increasingly being conduct ed in conjunction with randomized phase III clinical trials to meet th e demand for pharmacoeconomic data when new products are launched. Whi le the need for such data is often global, the trials in which relevan t information may be collected are often conducted in only one or a li mited number of countries. A critical issue is how data from pivotal c linical trials in one setting can serve as the basis for pharmacoecono mic evaluations in others. We address this issue and report on four ec onomic evaluations that we undertook in conjunction with a recent U.S. phase III clinical trial of recombinant human deoxyribonuclease (rhDN ase), which is used to improve pulmonary function in patients with cys tic fibrosis (CF). The objective of these evaluations was to estimate the potential impact of rhDNase therapy in France, Germany, Italy, and the United Kingdom on the direct costs of medical care for the treatm ent of respiratory tract infections (RTIs) in patients with CF. Analys es of economic impact were undertaken both with and without adjustment for differences in practice patterns between the United States and th e countries of interest. Our findings suggest that rhDNase therapy may reduce the cost of RTI-related care by between US$600 and US$1,100 ov er a 24-week period; the cost of rhDNase is not included in these figu res, as a price was unavailable when our analyses were undertaken. Des pite methodologic challenges, economic evaluations that meet the infor mation needs of decision makers in diverse countries can nonetheless b e undertaken in conjunction with phase III clinical trials.