J López-Bastida1, J M Ramos-Goñi2, I Aranda-Reneo3, M Trapero-Bertran4, P Kanavos5, B Rodriguez Martin6. 1. University of Castilla-La Mancha, Faculty of Occupational Therapy, Speech Therapy and Nursing, Talavera de la Reina, Toledo, Spain. Electronic address: julio.lopezbastida@uclm.es. 2. Axentiva Solutions, Tacoronte, Spain. 3. University of Castilla-La Mancha, Faculty of Social Science, Talavera de la Reina, Toledo, Spain. 4. Universitat Internacional de Catalunya (UIC), Faculty of Economics and Social Sciences, Barcelona, Spain. 5. London School of Economics and Political Science, London, UK. 6. University of Castilla-La Mancha, Faculty of Occupational Therapy, Speech Therapy and Nursing, Talavera de la Reina, Toledo, Spain.
Abstract
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.
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.