Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy
Autor: | Juan Manuel Ramos-Goñi, Isaac Aranda-Reneo, Armando Magrelli, Julio López-Bastida, Domenica Taruscio, Panos Kanavos |
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Jazyk: | angličtina |
Rok vydání: | 2019 |
Předmět: |
0301 basic medicine
Adult Male medicine.medical_specialty Haemophilia lcsh:Medicine Computer-assisted web interviewing 030105 genetics & heredity Social value orientations Hemophilia A Cystic fibrosis Orphan drug 03 medical and health sciences 0302 clinical medicine Rare Diseases Surveys and Questionnaires medicine Humans Orphan drugs Pharmacology (medical) Genetics (clinical) Response rate (survey) Research lcsh:R Health technology General Medicine Middle Aged medicine.disease Preference 3. Good health Italy Discrete choice experiment Family medicine Female Psychology Decision making Rare disease 030217 neurology & neurosurgery |
Zdroj: | Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-7 (2019) Orphanet Journal of Rare Diseases RUIdeRA. Repositorio Institucional de la UCLM instname RUIdeRA: Repositorio Institucional de la UCLM Universidad de Castilla-La Mancha |
ISSN: | 1750-1172 |
Popis: | Background: Decision makers have huge problems when attempting to attribute social value to the improvements achieved by new drugs, especially when considering the use of orphan drugs for rare diseases. We present the results of a pilot study aimed to investigate patient preferences regarding public funding for drugs used to treat rare diseases.Methods: An online questionnaire was used as a discrete choice experiment (DCE) survey to explore the preferences of patients with cystic fibrosis and haemophilia in Italy. The questionnaire focused on relevant issues that were defined in a review of the literature. A conditional logistic model showed preferences for specific attributes.Results: A total of 54 questionnaires (20% response rate) were completed. The issues that received the greatest attention were improvement in health, treatment cost and value for money. However, disease severity and the availability of other treatments were important social values that could not be ignored.Conclusions: The findings presented here provide evidence as to what patients with cystic fibrosis or haemophilia think are the most important considerations on which to base decisions in health technology scenarios, and regarding the priorities for funding. |
Databáze: | OpenAIRE |
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