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Toward European harmonization of national myasthenia gravis registries: modified Delphi procedure-based expert consensus on collectable data
A. Slioui, G. Tammam, F. Vanoli, AD. Marina, S. Vohanka, NE. Gilhus, I. Moroni, MI. Leite, F. Piehl, C. Antozzi, J. Pini, F. Stascheit, S. Attarian, E. Santos, J. Verschuuren, L. Canonge, J. Garcia, C. Perriard, E. Cortés-Vicente, R. Mantegazza,...
Language English Country England, Great Britain
Document type Journal Article
NLK
BioMedCentral
from 2006-12-01
BioMedCentral Open Access
from 2006
Directory of Open Access Journals
from 2006
Free Medical Journals
from 2006
PubMed Central
from 2006
Europe PubMed Central
from 2006
ProQuest Central
from 2009-01-01
Open Access Digital Library
from 2006-01-01
Open Access Digital Library
from 2006-01-01
Medline Complete (EBSCOhost)
from 2006-01-01
Health & Medicine (ProQuest)
from 2009-01-01
ROAD: Directory of Open Access Scholarly Resources
from 2006
Springer Nature OA/Free Journals
from 2006-12-01
- MeSH
- Delphi Technique * MeSH
- Consensus MeSH
- Humans MeSH
- Myasthenia Gravis * therapy diagnosis MeSH
- Registries * MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Geographicals
- Europe MeSH
BACKGROUND: Myasthenia gravis (MG) is a rare autoimmune disorder. Several new treatment concepts have emerged in recent years, but access to these treatments varies due to differing national reimbursement regulations, leading to disparities across Europe. This highlights the need for high-quality data collection by stakeholders to establish MG registries. A European MG registry could help bridge the treatment access gap across different countries, offering critical data to support regulatory decisions, foster international collaborations, and enhance clinical and epidemiological research. Several national MG registries already exist or are in development. To avoid duplication and ensure harmonization in data collection, a modified Delphi procedure was implemented to identify essential data elements for inclusion in national registries. RESULTS: Following a literature review, consultations with patient associations and pharmaceutical companies, and input from multiple European MG experts, 100 data elements were identified. Of these, 62 reached consensus for inclusion and classification, while only 1 item was agreed for exclusion. 30 items failed to reach the ≥ 80% agreement threshold and were excluded. Among the 62 accepted items, 21 were classified as mandatory data elements, 32 optional, and 9 items pertained to the informed consent form. CONCLUSIONS: Through a modified Delphi procedure, consensus was successfully achieved. This consensus-based approach represents a crucial step toward harmonizing MG registries across Europe. The resulting dataset will facilitate the sharing of knowledge and enhance European collaborations. Furthermore, the harmonized data may assist in regulatory or reimbursement decisions regarding novel therapies, as well as address treatment access disparities between European countries.
Department of Brain and Behavioral Sciences University of Pavia IRCCS Mondino Foundation Pavia Italy
Department of Clinical Medicine University of Bergen Bergen Norway
Department of Human Neurosciences Sapienza University of Rome Rome Italy
Department of Neurology Faculty of Medicine University Hospital Brno Masaryk University Brno Czechia
Department of Neurology Leiden University Medical Center Leiden the Netherlands
Department of Pediatric Neurosciences Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy
ESIEE PARIS School Gustave Eiffel University Paris France
Neurology Department Centro Hospitalar Universitário de Santo António
Neuromuscular Diseases Unit Hospital de La Santa Creu 1 Sant Pau Barcelona Spain
References provided by Crossref.org
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