‘We are the engine’: a focus group study on clinical practice guideline development with European patient advocates for rare congenital malformations and/or intellectual disability
Understanding the perspectives of patient advocates is essential to develop CPGs that meet the life-long and complex care needs of individuals and families living with rare conditions. Identified challenges include balancing the urgency of information needs with thorough guideline development processes, as well as the integration and interpretation of different types of knowledge.
Authors
- Chloé Aymée de Mortier (ORCID: https://orcid.org/0000-0002-5911-7698)
- Charlotte M. W. Gaasterland (ORCID: https://orcid.org/0000-0003-3501-8211)
- Martina C. Cornel (ORCID: https://orcid.org/0000-0002-5397-5544)
- Mirthe J Klein Haneveld (ORCID: https://orcid.org/0000-0002-0453-2608)
- Agnies M. van Eeghen (ORCID: https://orcid.org/0000-0001-8149-8645)
- Anne Hugon
Institutions
- Royal Dutch Pharmacists Association (NL)
- Emma Kinderziekenhuis (NL)
- Department of Health (TW)
- Hôpital Robert-Debré (FR)
- Department of Health Services (NP)
- Public Health Service of Amsterdam (NL)
- Amsterdam University Medical Centers (NL)
- Société Francophone du Diabète (FR)
- 's Heeren Loo (NL)
- Amsterdam University of Applied Sciences (NL)
- Vrije Universiteit Amsterdam (NL)
- University of Amsterdam (NL)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-04-09
- DOI
- https://doi.org/10.1186/s13023-025-03673-9
- Citations
- 3
- Primary Topic
- Ethics in Clinical Research
- Type
- article
- Field-Weighted Citation Impact
- 5.64
Funders
- European Commission