- 作者：Renata Linertov¨¢ ; Pedro Serrano-Aguilar ; Manuel Posada-de-la-Paz ; Manuel Hens-P¨¦rez ; Panos Kanavos ; Domenica Taruscio ; Arrigo Schieppati ; Rumen Stefanov ; M¨¢rta P¨¦ntek ; Claudia Delgado ; Johann-Matthias Graf von der Schulenburg ; Ulf Persson ; Karine Chevreul ; Giovanni Fattore ; Melany Worbes-Cerezo ; Mark Sefton ; Julio L¨®pez-Bastida ; on behalf of BURQOL-RD Research Group
- 关键词：Rare diseases ; Delphi approach ; Health-related quality of life ; Burden of illness
- 刊名：Health Policy
- 出版年：2012
- 出版时间：November, 2012
- 年：2012
- 卷：108
- 期：1
- 页码：19-26
- 全文大小：245 K
Objectives
The BURQOL-RD project is intended to develop a disease based model capable of quantifying the socio-economic burden and health-related quality of life for patients with rare diseases (RDs) and their caregivers in Europe. We described the methodology used to select a set of 10 RDs to be approached in a pilot study.Methods
BURQOL-RD project includes 23 partners from 8 European countries: Spain, UK, France, Germany, Sweden, Italy, Hungary and Bulgaria. A two-round Delphi panels in combination with Carroll diagram was used to generate consensus in the selection of the 10 RDs among the project participants.
Results
The two Delphi rounds yielded a prioritised list, to which the Carroll diagram was applied, taking into account three determinants: prevalence, availability of effective treatment and need for carer. The final set of RD to be studied was obtained: cystic fibrosis, Prader-Willi syndrome, haemophilia, duchenne muscular dystrophy, epidermolysis bullosa, fragile X syndrome, scleroderma, mucopolysaccharidosis, juvenile idiopathic arthritis and histiocytosis.
Conclusions
This methodology permitted the generation of an equilibrated set of RDs for the pilot study of BURQOL-RD project. The model will be suitable for application in a wide range of RDs.