title: Patient/family views on data sharing in rare diseases: study in the European LeukoTreat project.
abstract: The purpose of this study was to explore patient and family views on the sharing of their medical data in the context of compiling a European leukodystrophies database. A survey questionnaire was delivered with help from referral centers and the European Leukodystrophies Association, and the questionnaires returned were both quantitatively and qualitatively analyzed. This study found that patients/families were strongly in favor of participating. Patients/families hold great hope and trust in the development of this type of research. They have a strong need for information and transparency on database governance, the conditions framing access to data, all research conducted, partnerships with the pharmaceutical industry, and they also need access to results. Our findings bring ethics-driven arguments for a process combining initial broad consent with ongoing information. On both, we propose key item-deliverables to database participants.
authors.affiliation: Ethique médicale - EA 4569 - Université Paris Descartes, Sorbonne Paris Cité, Faculté de Médecine, Paris, France.
authors.forename: Sylviane
authors.initials: S
authors.lastname: Darquy
authors.affiliation: Assistance Publique-Hôpitaux de Paris, HEGP-Hôpital Corentin Celton, Unité de Médecine Sociale, Issy-les-Moulineaux, France.
authors.forename: Grégoire
authors.initials: G
authors.lastname: Moutel
authors.affiliation: Ethique médicale - EA 4569 - Université Paris Descartes, Sorbonne Paris Cité, Faculté de Médecine, Paris, France.
authors.forename: Anne-Sophie
authors.initials: AS
authors.lastname: Lapointe
authors.affiliation: Ethique médicale - EA 4569 - Université Paris Descartes, Sorbonne Paris Cité, Faculté de Médecine, Paris, France.
authors.forename: Diane
authors.initials: D
authors.lastname: D'Audiffret
authors.affiliation: Ethique médicale - EA 4569 - Université Paris Descartes, Sorbonne Paris Cité, Faculté de Médecine, Paris, France.
authors.forename: Julie
authors.initials: J
authors.lastname: Champagnat
authors.affiliation: Clermont Université, Université d'Auvergne, Faculté de médecine, Clermont-Ferrand, France.
authors.forename: Samia
authors.initials: S
authors.lastname: Guerroui
authors.affiliation: Assistance Publique-Hôpitaux de Paris, Hôpital Robert Debré, Centre de Reference maladies rares « leucodystrophies », Service de Neuropédiatrie et Maladies Métaboliques, Paris, France.
authors.forename: Marie-Louise
authors.initials: ML
authors.lastname: Vendeville
authors.affiliation: Assistance Publique-Hôpitaux de Paris, Hôpital Robert Debré, Centre de Reference maladies rares « leucodystrophies », Service de Neuropédiatrie et Maladies Métaboliques, Paris, France.|Université Paris Diderot- Sorbonne Paris Cité, DHU Protect, INSERM U 1141, Hôpital Robert Debré, Paris, France.
authors.forename: Odile
authors.initials: O
authors.lastname: Boespflug-Tanguy
authors.affiliation: Ethique médicale - EA 4569 - Université Paris Descartes, Sorbonne Paris Cité, Faculté de Médecine, Paris, France.
authors.forename: Nathalie
authors.initials: N
authors.lastname: Duchange
mesh_terms: D000328:Adult; D035843:Biomedical Research; D002986:Clinical Trials as Topic; D016208:Databases, Factual; D005060:Europe; D005190:Family; D006282:Health Personnel; D006801:Humans; D033181:Information Dissemination; D056784:Leukoencephalopathies / Q000628:therapy*; D008875:Middle Aged; D035583:Rare Diseases / Q000628:therapy*; D012108:Research Personnel