Elicitation of utility scores in Canada for immune thrombocytopenia treated with romiplostim or watch and rescue.

OBJECTIVES: The objective of this study was to obtain utilities, or preference-based quality-of-life values, from the Canadian general public, for potential health states experienced by immune thrombocytopenia (ITP) patients receiving either romiplostim (a new thrombopoietin mimetic agent) or 'watch and rescue' therapy. Utilities are needed to conduct a cost-utility analysis of romiplostim for formulary and reimbursement decisions. METHODS: An electronic Time Trade-off (TTO) survey was developed and administered to a sample of the general public in Canada, with 12 distinct health states derived from two randomized clinical trials of romiplostim vs watch and rescue treatment. Two pilot tests assessed interpretability and respondent burden. In the final survey, each subject was administered the TTO for four randomly-selected health states. Descriptive statistics were computed for utility scores, and differences between health states were evaluated with an analysis of variance model. RESULTS: Eight hundred and twenty-one adults completed the TTO survey. Mean age was 36.4 (SD = 15) years; 63% were female. Mean (SD) utility scores ranged from 0.476 (0.271) for the most severe health state (significant bleeding) to 0.633 (0.282) for the least severe health state depicting successful treatment with romiplostim. Statistical significance was found on the mean difference between the most severe health state and five other health states (p < 0.05). After adjusting utilities for matching Canadian demographic parameters, no substantial difference was found between original utility scores and adjusted scores. CONCLUSIONS: This study provides evidence of the Canadian general public's preference for 12 ITP health states pertaining to romiplostim treatment or watch and rescue. This study had a number of limitations, the main ones being the lack of perfect match in demographics between this sample and the Canadian population, as well as the fact that the scenario descriptions were based on both published literature and expert opinion. Despite those limitations, the obtained utility scores may be used in cost-utility models of romiplostim as a treatment for ITP patients in Canada.

The impact of delayed chemotherapy-induced nausea and vomiting on patients, health resource utilization and costs in German cancer centers.

BACKGROUND: Delayed chemotherapy-induced nausea and vomiting (CINV) remains a major adverse effect of cancer chemotherapy. We assessed, under current practice patterns, the occurrence and impact on healthcare resource utilization of CINV in patients receiving emetogenic chemotherapy. An additional aim of this study was to estimate costs imputable to CINV in the German healthcare environment. MATERIALS AND METHODS: This prospective, multi-center, cross-sectional cost-of-illness study was conducted in three hospitals and in three office-based facilities in Germany. Consecutive patients undergoing emetogenic chemotherapy (levels 4 or 5 according to Hesketh classification of emetogenicity) were enrolled. Data were obtained from preplanned chart reviews and from self-administered patient questionnaires. Analysis of direct costs was performed from the perspectives of third party payer (statutory sick fund), provider (hospital) and patients. Indirect costs were assessed on the basis of paid workdays lost. RESULTS: During the 5-day observation period, 134 of 208 chemotherapy cycles observed (64.4%) were associated with at least one episode of nausea or vomiting. More patients experienced delayed than acute CINV (60.7% versus 32.8%), and more patients reported nausea than vomiting (62.5% versus 26.0%). A total of 68 patients (32.6%) utilized healthcare resources due to CINV. The most frequently used resources were rescue medications and outpatient hospital and office physician visits. Only one patient required hospitalization and only three patients lost workdays due to CINV. Average costs imputable to CINV per patient (with or without CINV) per treatment cycle incurred by third party payers and hospital providers were Euro 49 and Euro 48, respectively. Patient or treatment characteristics that were associated with high costs imputable to CINV were as follows: cisplatin-containing regimen; experience of emesis; and presence of delayed CINV. CONCLUSIONS: A substantial proportion of patients continue to experience CINV. This entails not only clinical but also economic consequences, and highlights a continuing need for improved utilization of existing antiemetic agents and for new, more efficacious treatments. The greatest improvements in patient care and potential for cost offset may be realized by preventing delayed CINV.