Premarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies
Mooghali M, Wallach J, Ross J, Ramachandran R. Premarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies. JAMA Network Open 2024, 7: e2430486. PMID: 39190303, PMCID: PMC11350476, DOI: 10.1001/jamanetworkopen.2024.30486.Peer-Reviewed Original ResearchConceptsFood and Drug Administration tableFood and Drug AdministrationSurrogate end pointsSurrogate markerPostmarketing studiesEnd pointsTraditional approvalCross-sectional studyClinical benefitAccelerated approvalTherapy designClinically significant end pointsReview of therapeuticsPrimary end pointUS Food and Drug AdministrationTrial end pointsSignificant end pointsPostmarketing requirementsPreliminary clinical evidenceApproval pathwayPivotal trialsClinical evidenceBreakthrough therapiesDrug AdministrationTherapyCharacterization of accelerated approval status, trial endpoints and results, and recommendations in guidelines for oncology drug treatments from the National Comprehensive Cancer Network: cross sectional study
Mooghali M, Mitchell A, Skydel J, Ross J, Wallach J, Ramachandran R. Characterization of accelerated approval status, trial endpoints and results, and recommendations in guidelines for oncology drug treatments from the National Comprehensive Cancer Network: cross sectional study. BMJ Medicine 2024, 3: e000802. PMID: 38596814, PMCID: PMC11002412, DOI: 10.1136/bmjmed-2023-000802.Peer-Reviewed Original ResearchNational Comprehensive Cancer NetworkNational Comprehensive Cancer Network guidelinesComprehensive Cancer NetworkFood and Drug AdministrationCancer NetworkUS Food and Drug AdministrationCategories of evidenceAccelerated approvalCross sectional studyDrug treatmentConfirmatory trialsClinical benefitTrial resultsOncological indicationsTraditional approvalGuideline recommendationsOutcome measuresSectional studyApproval statusFDA decisionRecommended alignmentOncologyUpdate recommendationsPostapproval trialsGuidelines