2021
Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019
Dhodapkar M, Zhang AD, Puthumana J, Downing NS, Shah ND, Ross JS. Characteristics of Clinical Studies Used for US Food and Drug Administration Supplemental Indication Approvals of Drugs and Biologics, 2017 to 2019. JAMA Network Open 2021, 4: e2113224. PMID: 34110392, PMCID: PMC8193429, DOI: 10.1001/jamanetworkopen.2021.13224.Peer-Reviewed Original ResearchConceptsPrimary efficacy end pointEfficacy end pointPivotal trialsIndication approvalsActive comparatorClinical outcomesSupplemental indicationsUS FoodEnd pointOriginal approvalTherapeutic areasPivotal efficacy trialsCross-sectional studyAdditional clinical dataDrug Administration approvalNew indication approvalsStrength of evidenceAdministration approvalMonths durationClinical dataClinical studiesEfficacy trialsMedian numberCancer indicationsMAIN OUTCOME
2020
Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017
Zhang AD, Puthumana J, Downing NS, Shah ND, Krumholz HM, Ross JS. Assessment of Clinical Trials Supporting US Food and Drug Administration Approval of Novel Therapeutic Agents, 1995-2017. JAMA Network Open 2020, 3: e203284. PMID: 32315070, PMCID: PMC7175081, DOI: 10.1001/jamanetworkopen.2020.3284.Peer-Reviewed Original ResearchConceptsPivotal trialsProportion of indicationsIndication approvalsNew drugsUS FoodTrial durationPrimary end pointTherapeutic areasPivotal efficacy trialsCross-sectional studyDrug Administration approvalFast track designationCommon therapeutic areasRecent FDA approvalOrphan designationLonger trial durationAccelerated approvalAdministration approvalClinical trialsMonths durationEfficacy trialsMAIN OUTCOMETherapeutic safetyDrug AdministrationFDA approval
2018
Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval
Shaw DL, Dhruva SS, Ross JS. Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval. Journal Of Managed Care & Specialty Pharmacy 2018, 24: 10.18553/jmcp.2018.24.12.1230. PMID: 30479199, PMCID: PMC10397592, DOI: 10.18553/jmcp.2018.24.12.1230.Peer-Reviewed Original ResearchConceptsNovel therapeutic agentsPrescription drug plansOrphan drug statusFDA approvalYear of approvalTherapeutic agentsNovel therapeuticsPrior authorizationNovel therapiesPriority reviewStep therapyMedicare prescription drug plansDrug plansDrug coverageNational InstituteDrug statusPart D prescription drug benefitMedicaid ServicesDrug characteristicsTherapeutic areasCross-sectional studyDrug benefit coverageHealth insurance payerBlue Cross Blue Shield AssociationPrescription drug benefit
2017
Postmarket Safety Events Among Novel Therapeutics Approved by the US Food and Drug Administration Between 2001 and 2010
Downing NS, Shah ND, Aminawung JA, Pease AM, Zeitoun JD, Krumholz HM, Ross JS. Postmarket Safety Events Among Novel Therapeutics Approved by the US Food and Drug Administration Between 2001 and 2010. JAMA 2017, 317: 1854-1863. PMID: 28492899, PMCID: PMC5815036, DOI: 10.1001/jama.2017.5150.Peer-Reviewed Original ResearchConceptsNovel therapeuticsSafety eventsAccelerated approvalTherapeutic characteristicsUS FoodDrug AdministrationFDA approvalInitial regulatory approvalClinician decision makingNew safety risksMedian followCohort studyMedian timeMultivariable analysisBoxed warningDrug classesMAIN OUTCOMEClinical practicePostmarket periodPsychiatric diseasesPriority reviewTherapeutic areasOrphan statusPsychiatric therapeuticsBiologics