Clara Cavero-Carbonell1, Elena Gras-Colomer1, Rosana Guaita-Calatrava2, Carmen López-Briones3, Rubén Amorós3, Ignacio Abaitua4, Manuel Posada4, Oscar Zurriaga5. 1. Rare Diseases Research Area, FISABIO-Public Health, Valencia, Spain. 2. Public Health Center, Valencia, Spain. 3. Inequalities Research Area, FISABIO-Public Health, Valencia, Spain. 4. Institute of Rare Diseases Research (IIER), Madrid, Spain SpainRDR and CIBERER, Madrid, Spain Instituto de Salud Carlos III (ISCIII), Madrid, Spain. 5. Rare Diseases Research Area, FISABIO-Public Health, Valencia, Spain Subdirección General de Epidemiología y Vigilancia de la Salud, Conselleria de Sanitat, Valencia, Spain Spanish Consortium of Epidemiology and Public Health Research (CIBERESP), Madrid, Spain.
Abstract
BACKGROUND: Patient registries (PRs) are important tools for public-health surveillance and rare-disease research. The purpose of this study is to identify the most important criteria for the creation of a rare-disease PR that could be used by public-health authorities to develop health policies. METHODS: A consensus-development Delphi study was used, with participants selected for their expertize in rare diseases and registries. Participants were asked to complete a questionnaire on the most important criteria for creating PRs. Three rounds were performed. RESULTS: Agreement was reached on half the questions in the first round and on 89% of questions in the final round, with a total expert participation rate of around 60% by the final stage. This study made it possible to reach a broader consensus starting from experts' initial assessment of the features that should be considered for the creation of a rare-disease PR. CONCLUSION: The consensus method used made it possible to define the characteristics of a PR based on expert opinion within a rare-disease framework. This study may serve as a guide for helping other researchers plan and build a rare-disease PR.
BACKGROUND:Patient registries (PRs) are important tools for public-health surveillance and rare-disease research. The purpose of this study is to identify the most important criteria for the creation of a rare-disease PR that could be used by public-health authorities to develop health policies. METHODS: A consensus-development Delphi study was used, with participants selected for their expertize in rare diseases and registries. Participants were asked to complete a questionnaire on the most important criteria for creating PRs. Three rounds were performed. RESULTS: Agreement was reached on half the questions in the first round and on 89% of questions in the final round, with a total expert participation rate of around 60% by the final stage. This study made it possible to reach a broader consensus starting from experts' initial assessment of the features that should be considered for the creation of a rare-disease PR. CONCLUSION: The consensus method used made it possible to define the characteristics of a PR based on expert opinion within a rare-disease framework. This study may serve as a guide for helping other researchers plan and build a rare-disease PR.
Authors: M K Javaid; L Forestier-Zhang; L Watts; A Turner; C Ponte; H Teare; D Gray; N Gray; R Popert; J Hogg; J Barrett; R Pinedo-Villanueva; C Cooper; R Eastell; N Bishop; R Luqmani; P Wordsworth; J Kaye Journal: Orphanet J Rare Dis Date: 2016-11-08 Impact factor: 4.123