Multi-criteria decision analysis (MCDA): testing a proposed MCDA framework for orphan drugs.

BACKGROUND: Since the introduction of the orphan drugs legislation in Europe, it has been suggested that the general method of assessing drugs for reimbursement is not necessarily suitable for orphan drugs. The National Institute for Health and Clinical Excellence indicated that several criteria other than cost and efficacy could be considered in reimbursement decisions for orphan drugs. This study sought to explore the multi-criteria decision analysis (MCDA) framework proposed by (Orphanet J Rare Dis 7:74, 2012) to a range of orphan drugs, with a view to comparing the aggregate scores to the average annual cost per patient for each product, and thus establishing the merit of MCDA as a tool for assessing the value of orphan drugs in relation to their pricings. METHODS: An MCDA framework was developed using the nine criteria proposed by (Orphanet J Rare Dis 7:74, 2012) for the evaluation of orphan drugs, using the suggested numerical scoring system on a scale of 1 to 3 for each criterion. Correlations between the average annual cost of the drugs and aggregate MCDA scores were tested and plotted graphically. Different weightings for each of the attributes were also tested. A further analysis was conducted to test the impact of including the drug cost as an attribute in the aggregate index scores. RESULTS: In the drugs studied, the R 2, that statistically measures how close the data are to the fitted regression line was 0.79 suggesting a strong correlation between the drug scores and the average annual cost per patient. CONCLUSION: Despite several limitations of the proposed model, this quantitative study provided insight into using MCDA and its relationship to the average annual costs of the products.

[Cost-effectiveness in Dutch mental health care: future because of ROM?]. / Doelmatigheid in de ggz: toekomst dankzij ROM?

BACKGROUND: The document reporting Dutch mental health care negotiations for 2014-2017 calls for a cost decrease based on cost-effectiveness. Thanks to ROM, the Dutch mental health care seems well prepared for cost-effectiveness research. AIM: Evaluate how valid cost-effectiveness research should be established in mental health care and the role of rom therein. METHOD: Evaluation of requirements of cost-effectiveness research, trends, and a translation to Dutch mental health care. RESULTS: Valid cost-effectiveness research in mental health care requires the application of a societal perspective, a long time-horizon and an adequate evaluation of quality of life of patients. Healthcare consumption, outcome of care and characterisation of the patient population should be measured systematically and continuously. Currently, rom-data are not suitable to serve as a basis for cost-effectiveness research, although a proper basis is present. Further development of rom could lead to a situation in which mental health care is purchased on the basis of cost-effectiveness. However, cost-effectiveness will only really be improved if quality of care is rewarded, rather than rewarding activities that are not always related to outcome of care. CONCLUSION: Cost-effectiveness research in mental health care should focus on societal costs and benefits, quality of life and a long time-horizon. If developed further, rom has the potential to be a basis for cost-effectiveness research in the future.

Factors affecting the uptake of vaccination by the elderly in Western society.

OBJECTIVE: To present the results of a literature review on factors related to vaccine uptake by elderly persons. METHODS: A systematic literature search was performed using Medline, Embase, and SciSearch to collect all publications available on factors related to vaccine uptake from 1966 until October 2012 for West European and North American societies. In total, 1001 articles were identified and 60 were included in the review. RESULTS: We identified six main themes that influence the willingness to be vaccinated: 1) attitudes and beliefs regarding vaccination in general including positive and negative attitudes and beliefs; 2) perceived risk and severity including knowledge, perceived susceptibility and severity and personal experience; 3) vaccine characteristics including side-effects, effectiveness, content of the vaccine and knowledge; 4) advice and information including influence of the healthcare worker and relatives and the information source and format; 5) general health-related behavior including previous vaccinations, visiting GP or senior center and other preventive behaviors; and 6) accessibility and affordability including logistics, combinations of vaccines and costs. CONCLUSION: The most important factors related to vaccine uptake are people's attitudes and beliefs regarding vaccination (especially their negative attitudes), recommendations of healthcare workers, side effects and effectiveness of the vaccine, and perceived susceptibility.

The Dutch tariff: results and arguments for an effective design for national EQ-5D valuation studies.

The objective of this study was to estimate a Dutch EQ-5D tariff and to determine in a simulation study using the dataset of the original UK valuation study, the number of health states and respondents needed to estimate a reliable tariff. In all, 300 Dutch respondents directly valued 17 states compared to 3000 respondents and 42 states in the original MVH protocol. The results reaffirmed differences in health-related preferences between countries, justifying the estimation of national tariffs. The mean absolute error was 0.030. The design of this study is recommended for national EQ-5D valuation studies.

[Measuring the quality of life in economic evaluations: the Dutch EQ-5D tariff]. / Kwaliteit van leven meten in economische evaluaties: het Nederlands EQ-5D-tarief.

OBJECTIVE: To value EQ-5D health states by a general Dutch public. EQ-5D is a standardised questionnaire that is used to calculate quality-adjusted life-years for cost-utility analysis. DESIGN: Descriptive. METHOD: A sample of 309 Dutch adults from Rotterdam and surroundings was asked to value 17 EQ-5D health states using the time trade-off method. Regression analysis was applied to the valuations of these 17 health states. By means of the estimated regression coefficients, which together constitute the so-called Dutch tariff, valuations can be determined for all possible EQ-5D health states. These values reflect the relative desirability of health states on a scale where 1 refers to full health and 0 refers to death. Societal valuations are necessary in order to correct life-years for the quality of life. RESULTS: Complete data were obtained from 298 persons. Theywere representative for the Dutch population as far as age, gender and subjective health were concerned, but had a somewhat higher educational level. The estimated Dutch EQ-5D tariff revealed that the respondents assigned the most weight to (preventing) pain and anxiety or depression, followed by mobility, self-care and the activities of daily living. The Dutch tariff differed from the UK ('Measurement and Valuation of Health') tariff, which is currently used in Dutch cost-utility analyses. Compared to UK respondents, Dutch respondents assigned more weight to anxiety and depression and less weight to the other dimensions. Conclusion. The valuation of health states by this representative Dutch study group differed from the valuation that is currently used in Dutch cost-utility analyses.