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.

Rotavirus vaccine and intussusception: how much risk will parents in the United States accept to obtain vaccine benefits?

Postlicensure surveillance of a newly licensed rotavirus vaccine suggested an increased risk of intussusception. Little was known about the amount of risk parents would tolerate to obtain the vaccine's benefits or the extent to which risk would reduce the price parents would pay for the vaccine. Parents of infants aged 12 months or younger were asked to accept or reject two hypothetical vaccines associated with varying degrees of risk. Parents chose from a list the amount they would pay for two additional hypothetical vaccines, with and without a risk of intussuception. The authors conducted face-to-face surveys in September 1999 among a convenience sample of parents in three US cities. Of 405 eligible parents, 260 (64%) participated. To achieve a 90% acceptance rate, the vaccine could be associated with no more than 1,794 (95% confidence interval: 1,551, 2,025) cases of intussusception in a fully vaccinated, national cohort of infants. The median willingness to pay for three vaccine doses, when vaccination was associated with 1,400 cases of intussusception, was $36 (95% confidence interval: $28, $46) compared with $110 (95% confidence interval: $96, $126) for the risk-free vaccine. The most important aspect of this study may be the methodology to assess how parents balance the benefits and risks of childhood vaccines.

Economic analysis of a child vaccination project among Asian Americans in Philadelphia, Pa.

OBJECTIVE: To ascertain the cost-effectiveness and the benefit-cost ratios of a community-based hepatitis B vaccination catch-up project for Asian American children conducted in Philadelphia, Pa, from October 1, 1994, to February 11, 1996. DESIGN: Program evaluation. SETTING: South and southwest districts of Philadelphia. PARTICIPANTS: A total of 4384 Asian American children. INTERVENTIONS: Staff in the community-based organizations (1) educated parents about the hepatitis B vaccination, (2) enrolled physicians in the Vaccines for Children program, and (3) visited homes of children due for a vaccine dose. Staff in the Philadelphia Department of Public Health developed a computerized database; sent reminder letters for children due for a vaccine dose; and offered vaccinations in public clinics, health fairs, and homes. MAIN OUTCOME MEASURES: The numbers of children having received 1, 2, or 3 doses of vaccine before and after the interventions; costs incurred by the Philadelphia Department of Public Health and the community-based organizations for design, education, and outreach activities; the cost of the vaccination; cost-effectiveness ratios for intermediate outcomes (ie, per child, per dose, per immunoequivalent patient, and per completed series); discounted cost per discounted year of life saved; and the benefit-cost ratio of the project. RESULTS: For the completed series of 3 doses, coverage increased by 12 percentage points at a total cost of $268 660 for design, education, outreach, and vaccination. Costs per child, per dose, and per completed series were $64, $119, and $537, respectively. The discounted cost per discounted year of life saved was $11 525, and 106 years of life were saved through this intervention. The benefit-cost ratio was 4.44:1. CONCLUSION: Although the increase in coverage was modest, the intervention proved cost-effective and cost-beneficial.

The Denver school-based adolescent hepatitis B vaccination program: a cost analysis with risk simulation.

OBJECTIVES: This study sought to compare the cost-effectiveness of a school-based hepatitis B vaccine delivery program with that of a vaccine delivery program associated with a network health maintenance organization (HMO). METHODS: The vaccination program enrolled 3359 sixth-grade students from 18 middle schools in Denver, Colo. Immunization status and direct and indirect program costs were compiled. The sensitivity of the outcomes was assessed by simulation methods. RESULTS: The per-dose cost-effectiveness ratio for the school-based delivery system was $31. This cost-effectiveness ratio remained stable when the model was simulated with costs that were underestimated or overestimated by 20%. In the network HMO, the direct cost per dose was $68 and the societal cost was $118 when the child's father worked full-time and the mother worked part-time. There is less than a 5% chance that the network HMO-based vaccination program could be more cost-effective than the school-based program. CONCLUSIONS: The cost per dose of the school-based program was significantly less than that of the network HMO-based program, because in the school program government-purchased vaccine was available at a lower cost and parents did not incur work-loss costs.

An integer programming model for vaccine procurement and delivery for childhood immunization: a pilot study.

The National Immunization Program of the Centers for Disease Control and Prevention has identified several challenges that must be faced in childhood immunization programs to deliver and procure vaccines to protect against the common preventable diseases. The biomedical challenge is how to combine and formulate products to take advantage of new vaccines without requiring additional injections. A programmatic challenge is to incorporate then into already crowded immunization schedules. The economic challenge is to make wise procurement choices from among a growing number of competing products. This paper reports the results of a pilot study using operations research methodologies to address the third of these challenges. The pilot is an integer programming model for procuring vaccines for a set of childhood diseases. The model is studied under various scenarios (minimum total cost, next lowest total cost, maximum total cost, minimum total cost with all manufacturers represented). The results of this pilot study demonstrate how a practical set of operations research tools can be developed to guide vaccine selection and procurement, which might stimulate the development of innovations in new vaccines to meet the challenges of disease control through immunization.

Addressing the challenges to immunization practice with an economic algorithm for vaccine selection.

The biotechnology revolution is producing a growing bounty of new vaccines which pose difficult choices in selecting among many products. Some major public and private purchasers of vaccine may offer individual physicians and clinics their choice in assembling vaccine inventories. Others might purchase only a limited stock of products that would satisfactorily immunize a typical child. In either case, current vaccine selection decisions are based principally on purchase price alone without systematic consideration of other factors of fiscal consequence. As a potential tool for decision making, we developed an economic algorithm for vaccine selection that would minimize the overall costs of disease control through immunization by considering: (1) purchase price, (2) number of doses needed, (3) preparation time, (4) route of administration, (5) cold storage needs, (6) shelf life, (7) earliest age of full immunity, (8) adverse events frequency, and (9) efficacy of protection. To demonstrate the algorithm, variables (1) to (4) above were incorporated into a pilot binary-integer linear programming model that satisfied the recommended immunization schedule for diphtheria, tetanus, pertussis, Haemophilus influenzae b, and hepatitis B, using eleven vaccines (DTaP, DTaP-Hib, Hib, HepB and Hib-HepB) from four manufacturers. Five (or six) opportunities to vaccinate were modeled at (1), 2, 4, 6, 12-18, and 60 months of life, assuming US$40 per clinic visit, $15 per injection, and $0.50 per minute of nurse preparation time. Vaccine costs were varied using actual March and September 1997 US Federal vaccine prices, as well as estimates for unpriced new vaccines. Over 16,000 distinct vaccine stocking lists by vaccine type and brand were possible. Including a 1-month visit, the lowest-cost 'solution' of the algorithm was $529.41 per child in the March cost-assumption case, and $490.32 in the September one (both included four doses of DTaP-Hib, three HepB, and one DTaP). Without a 1-month visit, the lowest-cost solution in the March case cost $486.67 (four DTaP, two Hib-HepB, one DTaP-Hib, and one HepB), while the September case cost $450.32 (four DTaP-Hib, three HepB, and one DTaP). Ensuring at least one product was selected from each of the four manufacturers increased costs about $13.00, and the needed injections rose from eight to nine. The most economical selection of vaccines to use cannot be intuitively predicted, as permutations are large and solutions are sensitive to minor changes in costs and constraints. A transparent, objective selection method that weighs the economic value of distinguishing features among competing vaccines might offer the 'best value' to vaccine purchasers, while also creating strong market incentives for continuing innovation and competition in the vaccine industry.