Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017–2019
Poddar A, Raggio M, Concato J. Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017–2019. Therapeutic Innovation & Regulatory Science 2023, 58: 214-221. PMID: 37926768, PMCID: PMC10764372, DOI: 10.1007/s43441-023-00589-z.Peer-Reviewed Original ResearchMeSH KeywordsDrug ApprovalDrug DevelopmentDrugs, InvestigationalHumansPharmaceutical PreparationsRare DiseasesConceptsBreakthrough therapy statusTherapy statusInsufficient treatment effectNon-oncology drugsReview of requestsUnmet medical needInadequate study designBreakthrough therapy designationConclusionsThis analysisCommon reasonInvestigational drugsUS FoodMedical needOrphan diseaseStudy designDrug evaluationMarketing approvalCorresponding drugsDrugsApproval statusTreatment effectsDrug developmentFDA processDesignation programStatusUse of Real‐World Evidence in Neuroscience‐Related New Drug and Biologics License Applications for Novel Therapeutics
Bloomfield‐Clagett B, Rahman M, Smith K, Concato J. Use of Real‐World Evidence in Neuroscience‐Related New Drug and Biologics License Applications for Novel Therapeutics. Clinical Pharmacology & Therapeutics 2023, 114: 1002-1005. PMID: 37548904, DOI: 10.1002/cpt.3018.Peer-Reviewed Original ResearchMeSH KeywordsBiological FactorsBiological ProductsDrug ApprovalHumansPharmaceutical PreparationsResearch DesignUnited States