Tag: orphan drug development

Clinician- and Patient-reported Endpoints in CNS Orphan Drug Clinical Trials: ISCTM Position Paper on Best Practices for Endpoint Selection, Validation, Training, and Standardization

Clinician- and Patient-reported Endpoints in CNS Orphan Drug Clinical Trials: ISCTM Position Paper on Best Practices for Endpoint Selection, Validation, Training, and Standardization

| October 1, 2021

by Joan Busner, PhD; Gahan Pandina, PhD; Silvia Zaragoza domingo PhD; Anna-Karin Berger PhD; Maria T. Acosta, MD; Nahome Fisseha, PharmD; Joseph Horrigan, MD; Jelena Ivkovic, MD; William Jacobson, PhD;┬áDennis Revicki, PhD; and Victoria Villalta-Gil, PhD, MSc All authors are members of the ISCTM Working Group for Rare Disease/Orphan Drug Development; Drs. Busner and Pandina […]

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