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Toward responsible clinical n-of-1 strategies for rare diseases.
Defelippe, Victoria M; J M W van Thiel, Ghislaine; Otte, Willem M; Schutgens, Roger E G; Stunnenberg, Bas; Cross, Helen J; O'Callaghan, Finbar; De Giorgis, Valentina; Jansen, Floor E; Perucca, Emilio; Brilstra, Eva H; Braun, Kees P J.
Afiliación
  • Defelippe VM; Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology,
  • J M W van Thiel G; Department of Medical Humanities, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.
  • Otte WM; Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands.
  • Schutgens REG; Van Creveldkliniek, Benign Hematology Center, University Medical Center Utrecht, Heidelberglaan 100, 3584 CX Utrecht, the Netherlands; European Reference Network for Oncological and non-oncological Rare Hematological Diseases (EuroBloodNet), Hôpital St Louis / Université Paris 7, 1 Avenue Claude Vel
  • Stunnenberg B; Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Donders Center for Medical Neuroscience, Radboud University Medical Center, Thomas van Aquinostraat 4, 6525 GD Nijmegen, the Netherlands.
  • Cross HJ; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; University College London (UCL) Great Ormond Street,
  • O'Callaghan F; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Paediatric Neuroscience, UCL Great Ormond Street, In
  • De Giorgis V; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Fondazione Mondino National Institute of Neurology,
  • Jansen FE; Department of Child Neurology, UMCU Brain Center, University Medical Center Utrecht, Universiteitsweg 100, 3584 CG Utrecht, the Netherlands; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology,
  • Perucca E; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Medicine, University of Melbourne (Aus
  • Brilstra EH; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Genetics, Center for Molecular Medicin
  • Braun KPJ; European Reference Network for Rare and Complex Epilepsies (EpiCare), Department of Paediatric Clinical Epileptology, Sleep Disorders and Functional Neurology, c/o Pr Arzimanoglou, Hôpital Femme Mère Enfant, 59 Boulevard Pinel, 69677 Bron, France; Department of Child Neurology, UMCU Brain Center, Un
Drug Discov Today ; 28(10): 103688, 2023 10.
Article en En | MEDLINE | ID: mdl-37356616
N-of-1 strategies can provide high-quality evidence of treatment efficacy at the individual level and optimize evidence-based selection of off-label treatments for patients with rare diseases. Given their design characteristics, n-of-1 strategies are considered to lay at the intersection between medical research and clinical care. Therefore, whether n-of-1 strategies should be governed by research or care regulations remains a debated issue. Here, we delineate differences between medical research and optimized clinical care, and distinguish the regulations which apply to either. We also set standards for responsible optimized clinical n-of-1 strategies with (off-label) treatments for rare diseases. Implementing clinical n-of-1 strategies as defined here could aid in optimized treatment selection for such diseases.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades Raras Tipo de estudio: Guideline Límite: Humans Idioma: En Revista: Drug Discov Today Asunto de la revista: FARMACOLOGIA / TERAPIA POR MEDICAMENTOS Año: 2023 Tipo del documento: Article Pais de publicación: Reino Unido
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Enfermedades Raras Tipo de estudio: Guideline Límite: Humans Idioma: En Revista: Drug Discov Today Asunto de la revista: FARMACOLOGIA / TERAPIA POR MEDICAMENTOS Año: 2023 Tipo del documento: Article Pais de publicación: Reino Unido