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Delphi approach to select rare diseases for a European representative survey: the BURQOL-RD study

Linertová, Renata, Serrano-Aguilar, Pedro, Posada-de-la-Paz, Manuel, Hens-Pérez, Manuel, Kanavos, Panos ORCID: 0000-0001-9518-3089, Taruscio, Domenica, Schieppati, Arrigo, Stefanov, Rumen, Péntek, Márta, Delgado, Claudia, Graf von der Schulenburg, Johann-Matthias, Persson, Ulf, Chevreul, Karine, Fattore, Giovanni, Worbes-Cerezo, Melany, Sefton, Mark and López-Bastida, Julio (2012) Delphi approach to select rare diseases for a European representative survey: the BURQOL-RD study. Health Policy, 108 (1). pp. 19-26. ISSN 0168-8510

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Identification Number: 10.1016/j.healthpol.2012.08.001


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.

Item Type: Article
Official URL:
Additional Information: © 2012 Elsevier Ireland
Divisions: European Institute
Social Policy
LSE Health
Subjects: H Social Sciences > HC Economic History and Conditions
R Medicine > R Medicine (General)
JEL classification: I - Health, Education, and Welfare > I1 - Health > I12 - Health Production: Nutrition, Mortality, Morbidity, Suicide, Substance Abuse and Addiction, Disability, and Economic Behavior
Date Deposited: 11 Sep 2012 09:14
Last Modified: 15 Feb 2024 05:09

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