Details
| Original language | English |
|---|---|
| Pages (from-to) | 19-26 |
| Number of pages | 8 |
| Journal | HEALTH POLICY |
| Volume | 108 |
| Issue number | 1 |
| Early online date | 2 Sept 2012 |
| Publication status | Published - Nov 2012 |
Abstract
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.
Keywords
- Burden of illness, Delphi approach, Health-related quality of life, Rare diseases
ASJC Scopus subject areas
- Medicine(all)
- Health Policy
Sustainable Development Goals
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In: HEALTH POLICY, Vol. 108, No. 1, 11.2012, p. 19-26.
Research output: Contribution to journal › Article › Research › peer review
}
TY - JOUR
T1 - Delphi approach to select rare diseases for a European representative survey
T2 - The BURQOL-RD study
AU - BURQOL-RD Research Network
AU - Linertová, Renata
AU - Serrano-Aguilar, Pedro
AU - Posada-de-la-Paz, Manuel
AU - Hens-Pérez, Manuel
AU - Kanavos, Panos
AU - Taruscio, Domenica
AU - Schieppati, Arrigo
AU - Stefanov, Rumen
AU - Péntek, Márta
AU - Delgado, Claudia
AU - Graf von der Schulenburg, Johann Matthias
AU - Persson, Ulf
AU - Chevreul, Karine
AU - Fattore, Giovanni
AU - Worbes-Cerezo, Melany
AU - Sefton, Mark
AU - López-Bastida, Julio
PY - 2012/11
Y1 - 2012/11
N2 - 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.
AB - 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.
KW - Burden of illness
KW - Delphi approach
KW - Health-related quality of life
KW - Rare diseases
UR - http://www.scopus.com/inward/record.url?scp=84867336987&partnerID=8YFLogxK
U2 - 10.1016/j.healthpol.2012.08.001
DO - 10.1016/j.healthpol.2012.08.001
M3 - Article
C2 - 22947412
AN - SCOPUS:84867336987
VL - 108
SP - 19
EP - 26
JO - HEALTH POLICY
JF - HEALTH POLICY
SN - 0168-8510
IS - 1
ER -