Delphi approach to select rare diseases for a European representative survey

The BURQOL-RD study

authored by

BURQOL-RD Research Network , Renata Linertová, Pedro Serrano-Aguilar, Manuel Posada-de-la-Paz, Manuel Hens-Pérez, Panos Kanavos, Domenica Taruscio, Arrigo Schieppati, Rumen Stefanov, Márta Péntek, Claudia Delgado, Johann Matthias Graf von der Schulenburg, Ulf Persson, Karine Chevreul, Giovanni Fattore, Melany Worbes-Cerezo, Mark Sefton, Julio López-Bastida

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.

Organisation(s)

Institute of Insurance Business Administration

External Organisation(s)

Hospital de Gran Canaria Dr. Negrin
Red de Investigación en Servicios Sanitarios en Enfermedades Crónicas (REDISSEC)
Canary Islands Health Service
Institute of Health Carlos III (ISCIII)
London School of Economics and Political Science
Istituto Superiore di Sanita
Institute of Pharmacological Research Mario Negri IRCCS
Bulgarian Association for Promotion of Education and Science
Health Economics and Health Technology Assessment Research Centre (HunHTA)
Federación Española de Enfermedades Raras (FEDER)
The Swedish Institute for Health Economics
Université Paris-Est Créteil Val-de-Marne (UPEC)
Università Commerciale Luigi Bocconi
Centros de Investigacion Biomedica en Red - CIBER
BiomedRed
Universidad de Castilla-La Mancha

Type

Article

Journal

HEALTH POLICY

Volume

108

Pages

19-26

No. of pages

8

ISSN

0168-8510

Publication date

11.2012

Publication status

Published

Peer reviewed

Yes

ASJC Scopus subject areas

Health Policy

Sustainable Development Goals

SDG 3 - Good Health and Well-being

Electronic version(s)

https://doi.org/10.1016/j.healthpol.2012.08.001 (Access: Closed)