Using a stated preference discrete choice experiment to assess societal value from the perspective of decision-makers in Europe. Does it work for rare diseases?

OBJECTIVES: To pilot the feasibility of using a discrete choice experiment (DCE) design to investigate individual preferences from the decision-maker perspective regarding the use of public funding for orphan drugs and generate prior information for future experimental designs. METHODS: A DCE was used on a convenience sample of participants from five European countries (England, France, Germany, Italy and Spain), exploring their preferences in distinct healthcare scenarios involving orphan drugs. A preliminary review of the empirical literature on distributive preferences informed the selection of attributes and their levels in the design. An online questionnaire was used to conduct the DCE survey. RESULTS: A total of 199 questionnaires were completed. The five country model showed relative preference for some attributes over others: cost of treatment, improvement in health, value for money and availability of treatment alternatives received the greatest attention. However, disease severity, beginning of life, waiting times and side effects were also shown to be important social values that should not be ignored. CONCLUSIONS: The findings presented in this study provide insight about the preferences that can influence decisions on orphan drugs in different countries. This study also provides valuable prior information that could inform future DCE designs in this area.

Population norms for the EQ-5D-3L: a cross-country analysis of population surveys for 20 countries.

This study provides EQ-5D population norms for 20 countries (N = 163,838), which can be used to compare profiles for patients with specific conditions with data for the average person in the general population in a similar age and/or gender group. Descriptive EQ-5D data are provided for the total population, by gender and by seven age groups. Provided index values are based on European VAS for all countries, based on TTO for 11 countries and based on VAS for 10 countries. Important differences exist in EQ-5D reported health status across countries after standardizing for population structure. Self-reported health according to all five dimensions and EQ VAS generally decreased with increasing age and was lower for females. Mean self-rated EQ VAS scores varied from 70.4 to 83.3 in the total population by country. The prior living standards (GDP per capita) in the countries studied are correlated most with the EQ VAS scores (0.58), while unemployment appeared to be significantly correlated in people over the age of 45 only. A country's expenditure on health care correlated moderately with higher ratings on the EQ VAS (0.55). EQ-5D norms can be used as reference data to assess the burden of disease of patients with specific conditions. Such information, in turn, can inform policy-making and assist in setting priorities in health care.

Patient involvement in health research: a contribution to a systematic review on the effectiveness of treatments for degenerative ataxias.

This study aims to incorporate patients' perspective in the design of a systematic review of scientific literature on the effectiveness of degenerative ataxias (DA) treatments. 53 patients with DA from different regions of Spain were consulted using the Delphi method, with three rounds via e-mail. In the first round, obtained information was on treatments used and relevant self-perceived health problems related to DA. The following two rounds were used to prioritize and achieve a consensus on the answers. The participation rate was 100% for all rounds. The most relevant self-perceived health problems were limitations in activities of daily living (ADL), visual and auditory problems and diminished self-esteem. The bibliographic search for the systematic review was enriched by these patient contributions. No study offered information on treatment effectiveness for the following problems prioritized by patients: ADL, social relationships, disease acceptance and quality of life. Thus some of the self-perceived DA-related health problems identified by the patients have never been investigated and should be considered to improve future research projects which should be adapted to meet patients' needs. Effective participation of patients can extend the value of systematic reviews to ensure they respond to both clinicians' information needs and patients' expectations.