Recommendations from the IRDiRC Working Group on methodologies to assess the impact of diagnoses and therapies on rare disease patients

Autor: Galliano Zanello, Chun-Hung Chan, David A. Pearce, IRDiRC Working Group

Publikováno v: Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-10 (2022)

Abstract Rare disease patients face many challenges including diagnostic delay, misdiagnosis and lack of therapies. However, early access to diagnosis and therapies can modify the management and the progression of diseases, which in return positively

Externí odkaz: https://doaj.org/article/0b5144fbb40947c9b552b841b285b8ce

Orphan and paediatric medical devices in Europe: recommendations to support their availability for on-label and off-label clinical indications.

Autor: Melvin T; Department of Medical Gerontology, School of Medicine, Trinity College Dublin, Dublin, Ireland., Dooms MM; IRDiRC Working Group on MedTech for Rare Diseases, Ivry-sur-Seine, France.; Centre of Clinical Pharmacology, University Hospitals Leuven, Leuven, Belgium., Koletzko B; Department of Paediatrics, Dr. von Hauner Children's Hospital, LMU University Hospital, Munich, Germany.; German Center for Child and Adolescent Health (DZKJ), Site, Munich Germany.; European Academy of Paediatrics, Brussels, Belgium., Turner MA; Institute of Life course and Medical Sciences, University of Liverpool, Liverpool, UK.; conect4children Stichting, Utrecht, Netherlands., Kenny D; Children's Health Ireland at Crumlin, Dublin, Ireland., Fraser AG; Department of Cardiology, University Hospital of Wales, Cardiff, UK., Gewillig M; Paediatric Cardiology, University of Leuven, Leuven, Belgium., Jonker AH; University of Twente, Enschede, The Netherlands.

Publikováno v: Expert review of medical devices [Expert Rev Med Devices] 2024 Oct; Vol. 21 (10), pp. 893-901. Date of Electronic Publication: 2024 Sep 20.