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Ensuring Stakeholder Feedback in the Design and Conduct of Clinical Trials for Rare Diseases: ISCTM Position Paper of the Orphan Disease Working Group.

Autor: Pandina GJ; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Pandina is with Janssen Research & Development in Titusville, New Jersey., Busner J; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Busner is with Signant Health in Blue Bell, Pennsylvania and Department of Psychiatry, Virginia Commonwealth University School of Medicine in Richmond, Virginia., Kempf L; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Kempf is with Parexel in Washington, DC., Fallon J; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Fallon is with Curemark in Rye Brook, New York., Alphs LD; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Alphs is with Denovo Pharmaceuticals in Princeton, New Jersey., Acosta MT; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Acosta is with the National Institutes of Health in Bethesda, Maryland., Berger AK; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Berger is with H. Lundbeck A/S in Valby, Denmark., Day S; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Day is with Clinical Trials Consulting & Training in Buckingham, United Kingdom., Dunn J; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Dunn is with Evolution Research Group in Boston, Massachusetts., Villalta-Gil V; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Villalta-Gil is with WCG Clinical in Durham, North Carolina., Grabb MC; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Grabb is with the National Institute of Mental Health in Rockville, Maryland., Horrigan JP; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Horrigan is with AMO Pharma in Wonersh, United Kingdom and Duke University in Durham, North Carolina., Jacobson W; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Jacobson is with Harmony Biosciences in Mundelein, Illinois., Kando JC; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Kando is with Karuna Therapeutics in Boston, Massachusetts., Macek TA; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Macek is with Novartis Pharmaceuticals in Bannockburn, Illinois., Singh MK; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Singh is with Stanford University School of Medicine in Stanford, California., Stanford AD; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Stanford is with Bristol Myers Squibb in Cambridge, Massachusetts., Domingo SZ; All authors are members of the International Society for Central Nervous System Clinical Trials and Methodology (ISCTM) Working Group for Rare Disease/Orphan Drug Development. Drs. Pandina and Busner are Co-Chairs.; Dr. Domingo is with Neuropsynchro in Barcelona, Spain.

Publikováno v: Innovations in clinical neuroscience [Innov Clin Neurosci] 2024 Mar 01; Vol. 21 (1-3), pp. 52-60. Date of Electronic Publication: 2024 Mar 01 (Print Publication: 2024).

A Call for Objective Dose Selection to Increase Success in Pediatric Clinical Trials: A Perspective From NICHD and NIMH Program Staff.

Autor: Giacoia G; Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, USA., Grabb MC; National Institute of Mental Health (NIMH), Bethesda, Maryland, USA., Pawlyk AC; Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, USA., Ren Z; Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, USA., Samedy-Bates L; Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, USA., Taylor-Zapata P; Obstetric and Pediatric Pharmacology and Therapeutics Branch, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Bethesda, Maryland, USA.

Publikováno v: Journal of clinical pharmacology [J Clin Pharmacol] 2021 Jun; Vol. 61 Suppl 1, pp. S9-S12.