Delphi approach to select rare diseases for a European representative survey. The BURQOL-RD study

被引：39

作者：

Linertova, Renata ^{[1
,2
]}

Serrano-Aguilar, Pedro ^{[2
,3
]}

Posada-de-la-Paz, Manuel ^{[4
]}

Hens-Perez, Manuel ^{[4
]}

Kanavos, Panos ^{[5
]}

Taruscio, Domenica ^{[6
]}

Schieppati, Arrigo ^{[7
]}

Stefanov, Rumen ^{[8
]}

Pentek, Marta ^{[9
]}

Delgado, Claudia ^{[10
]}

von der Schulenburg, Johann-Matthias Graf ^{[11
]}

Persson, Ulf ^{[12
]}

Chevreul, Karine ^{[13
]}

Fattore, Giovanni ^{[14
]}

Worbes-Cerezo, Melany ^{[15
]}

Sefton, Mark ^{[16
]}

Lopez-Bastida, Julio ^{[1
,2
,17
]}

机构：

[1] Hosp Univ Gran Canaria Dr Negrin, Canary Isl Fdn Hlth & Res FUNCIS, Las Palmas Gran Canaria 35010, Spain

[2] Red Invest Serv Sanitarios Enfermedades Cron REDI, Madrid, Spain

[3] Canary Isl Hlth Serv, Evaluat & Planning Serv, Santa Cruz De Tenerife 38004, Spain

[4] Inst Salud Carlos III, Res Inst Rare Dis IIER, Madrid 28029, Spain

[5] Univ London London Sch Econ & Polit Sci, Hlth Res Ctr, London WC2A 2AE, England

[6] ISS, Natl Ctr Rare Dis, I-00161 Rome, Italy

[7] Mario Negri Inst Pharmacol Res, Ctr Ric Clin Malattie Rare Aldo & Cele Dacco, I-20156 Milan, Italy

[8] Bulgarian Assoc Promot Educ & Sci, ICRDOD, Plovdiv 4017, Bulgaria

[9] Ctr Publ Affairs Studies Fdn, Hlth Econ & Technol Assessment Res Ctr, H-1093 Budapest, Hungary

[10] FEDER, Madrid 28020, Spain

[11] Leibniz Univ Hannover, Ctr Hlth Econ, Inst Risk & Insurance, D-30167 Hannover, Germany

[12] Swedish Inst Hlth Econ, S-22002 Lund, Sweden

[13] Univ Paris Est, F-94000 Creteil, France

[14] Univ Commerciale Luigi Bocconi, Ctr Res Hlth & Social Care CERGAS, I-20136 Milan, Italy

[15] CIBERESP, Madrid, Spain

[16] Biomedred, Madrid 28010, Spain

[17] Univ Castilla La Mancha, E-13071 Ciudad Real, Spain

来源：

HEALTH POLICY | 2012年 / 108卷 / 01期

关键词：

Rare diseases; Delphi approach; Health-related quality of life; Burden of illness; QUALITY-OF-LIFE; SOCIAL-ECONOMIC COSTS; INTERNATIONAL CONSENSUS; APPROPRIATENESS; SCLEROSIS; PANEL;

D O I：

10.1016/j.healthpol.2012.08.001

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

摘要：

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. 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. (C) 2012 Elsevier Ireland Ltd. All rights reserved.

引用

页码：19 / 26

页数：8

共 41 条

[1]

Ali AK, 2005, QUAL REP, V10, P718

[2] ACR Appropriateness Criteria® Multiple Gestations [J].

Allison, Sandra O. DeJesus ;

Javitt, Marcia C. ;

Glanc, Phyllis ;

Andreotti, Rochelle F. ;

Bennett, Genevieve L. ;

Brown, Douglas L. ;

Dubinsky, Theodore ;

Harisinghani, Mukesh G. ;

Harris, Robert D. ;

Mitchell, Donald G. ;

Pandharipande, Pari V. ;

Pannu, Harpreet K. ;

Podrasky, Ann E. ;

Shipp, Thomas D. ;

Siegel, Cary Lynn ;

Simpson, Lynn ;

Wong-You-Cheong, Jade J. ;

Zelop, Carolyn M. .

ULTRASOUND QUARTERLY, 2012, 28 (02) :149-155

[3]

[Anonymous], 2006, EUR WHAT IS RAR DIS

[4]

[Anonymous], 2009, EUR VOIC 12000 PAT

[5]

[Anonymous], 2002, The Delphi Method

[6]

[Anonymous], 1996, Gazing Into the Oracle: The Delphi Method and Its Application to Social Policy and Public Health

[7] The effect of panel membership and feedback on ratings in a two-round Delphi survey - Results of a randomized controlled trial [J].

Campbell, SM ;

Hann, M ;

Roland, MO ;

Quayle, JA ;

Shekelle, PG .

MEDICAL CARE, 1999, 37 (09) :964-968

[8] Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure [J].

de Ru, Minke H. ;

Teunissen, Quirine G. A. ;

van der Lee, Johanna H. ;

Beck, Michael ;

Bodamer, Olaf A. ;

Clarke, Lorne A. ;

Hollak, Carla E. ;

Lin, Shuan-Pei ;

Rojas, Maria-Veronica Munoz ;

Pastores, Gregory M. ;

Raiman, Julian A. ;

Scarpa, Maurizio ;

Treacy, Eileen P. ;

Tylki-Szymanska, Anna ;

Wraith, J. Edmond ;

Zeman, Jiri ;

Wijburg, Frits A. .

ORPHANET JOURNAL OF RARE DISEASES, 2012, 7

[9] The use of consensus methods and expert panels in pharmacoeconomic studies - Practical applications and methodological shortcomings [J].

Evans, C .

PHARMACOECONOMICS, 1997, 12 (02) :121-129

[10]

FEDER (Federacion Espanola de Enfermedades Raras), 2009, EST SIT NEC SOC PERS

← 1 2 3 4 5 →