A multinational economic evaluation of rhDNase in the treatment of cystic fibrosis
Creators: name: Menzin, Joseph
name: Oster, Gerry
name: Davies, Linda
name: Drummond, Michael F.
name: Greiner, Wolfgang
name: Lucioni, Carlo
name: Merot, Jean-Luis
name: Rossi, Franco
name: Schulenburg, Johann-Matthias Graf Von Der
name: Souêtre, Eric
Publisher: name: Cambridge : Cambridge University Press
Year: 1996
Subjects: subject: antibiotic agent
subject: dornase alfa
subject: placebo
subject: adult
subject: aerosol
subject: antibiotic therapy
subject: child
subject: clinical trial
subject: controlled clinical trial
subject: controlled study
subject: cost control
subject: cystic fibrosis
subject: female
subject: forced expiratory volume
subject: health care cost
subject: health care utilization
subject: health economics
subject: hospital admission
subject: human
subject: incidence
subject: intravenous drug administration
subject: lung function
subject: major clinical study
subject: male
subject: nebulizer
subject: oral drug administration
subject: phase 3 clinical trial
subject: randomized controlled trial
subject: respiratory tract infection
subject: review
subject: Clinical Trials, Phase III
subject: Cystic Fibrosis
subject: Deoxyribonuclease I
subject: Drug Costs
subject: Europe
subject: Expectorants
subject: Health Care Costs
subject: Humans
subject: International Cooperation
subject: Physician's Practice Patterns
subject: Recombinant Proteins
subject: Statistics, Nonparametric
subject: United States
subject: Dewey Decimal Classification::600 | Technik::610 | Medizin, Gesundheit
subject: Dewey Decimal Classification::300 | Sozialwissenschaften, Soziologie, Anthropologie::330 | Wirtschaft

description: Economic evaluations of pharmaceuticals are increasingly being conducted in conjunction with randomized phase III clinical trials to meet the demand for pharmacoeconomic data when new products are launched. While the need for such data is often global, the trials in which relevant information may be collected are often conducted in only one or a limited number of countries. A critical issue is how data from pivotal clinical trials in one setting can serve as the basis for pharmacoeconomic evaluations in others. We address this issue and report on four economic evaluations that we undertook in conjunction with a recent U.S. phase III clinical trial of recombinant human deoxyribonuclease (rhDNase), which is used to improve pulmonary function in patients with cystic 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 treatment of respiratory tract infections (RTIs) in patients with C. Analyses of economic impact were undertaken both with and without adjustment for differences in practice patterns between the United States and the 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 over a 24-week period; the cost of rhDNase is not included in these figures, as a price was unavailable when our analyses were undertaken. Despite methodologic challenges, economic evaluations that meet the information needs of decision makers in diverse countries can nonetheless be undertaken in conjunction with phase III clinical trials.