2024
Direct oral anticoagulant approvals by four major regulatory agencies: a cross-sectional analysis of premarket and postmarket evidence
Mooghali M, Zhou T, Ross J. Direct oral anticoagulant approvals by four major regulatory agencies: a cross-sectional analysis of premarket and postmarket evidence. BMJ Open 2024, 14: e090376. PMID: 39461853, PMCID: PMC11529451, DOI: 10.1136/bmjopen-2024-090376.Peer-Reviewed Original ResearchConceptsPhase 2 trialPhase 3 trialBox warningAtrial fibrillation patientsPostmarketing evidenceEvidence of safetyOral anticoagulantsFibrillation patientsPostmarketing studiesCross-sectional analysisDegree of concordanceStroke preventionStudy requirementsPostmarketingEfficacy evidenceRegulatory agenciesDrugPostmarketing requirementsInterpretation of resultsEfficacyHealth CanadaSample sizeSafety evidenceConcordanceEndpointPremarket 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 ResearchMeSH KeywordsBiomarkersClinical Trials as TopicCross-Sectional StudiesDrug ApprovalEndpoint DeterminationHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationConceptsFood 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 AdministrationTherapyPremarket Evidence and Postmarketing Requirements for Real-Time Oncology Review Indication Approvals
Mooghali M, Mohammad A, Wallach J, Mitchell A, Ross J, Ramachandran R. Premarket Evidence and Postmarketing Requirements for Real-Time Oncology Review Indication Approvals. JAMA Network Open 2024, 7: e249233. PMID: 38691363, PMCID: PMC11063797, DOI: 10.1001/jamanetworkopen.2024.9233.Peer-Reviewed Original Research
2023
Unique Device Identifiers for Medical Devices at 10 Years
Dhruva S, Ross J, Wilson N. Unique Device Identifiers for Medical Devices at 10 Years. JAMA Internal Medicine 2023, 183: 1045-1046. PMID: 37603351, DOI: 10.1001/jamainternmed.2023.3572.Commentaries, Editorials and LettersMeSH KeywordsHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationAvailability of Unique Device Identifiers for Class I Medical Device Recalls From 2018 to 2022
Mooghali M, Ross J, Kadakia K, Dhruva S. Availability of Unique Device Identifiers for Class I Medical Device Recalls From 2018 to 2022. JAMA Internal Medicine 2023, 183: 735-737. PMID: 37184854, PMCID: PMC10186206, DOI: 10.1001/jamainternmed.2023.0727.Peer-Reviewed Original ResearchMeSH KeywordsDevice ApprovalHumansMedical Device RecallsProduct Surveillance, PostmarketingRisk FactorsUnited StatesUnited States Food and Drug AdministrationUS Food and Drug Administration regulatory reviewer disagreements and postmarket safety actions among new therapeutics
Eadie A, MacGregor A, Wallach J, Ross J, Herder M. US Food and Drug Administration regulatory reviewer disagreements and postmarket safety actions among new therapeutics. BMJ Evidence-Based Medicine 2023, 28: 151-156. PMID: 36944478, DOI: 10.1136/bmjebm-2022-112005.Peer-Reviewed Original ResearchMeSH KeywordsCommunicationCross-Sectional StudiesHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationConceptsNovel therapeuticsNew therapeuticsUS FoodReviewer disagreementTherapeutic approvalsFDA safety communicationCross-sectional studyBlack box warningPatient populationBox warningDrug AdministrationDrug approvalFDA reviewSafety actionsRegulatory approvalTherapeuticsFDAApprovalMarket approvalFDA reviewersReviewersInstances of disagreementReviewActionAdministration
2022
Spontaneous reporting of post-market safety signals: what evidence should support regulatory action?
Dhodapkar MM, Ross JS, Ramachandran R. Spontaneous reporting of post-market safety signals: what evidence should support regulatory action? The BMJ 2022, 379: o2409. PMID: 36198410, DOI: 10.1136/bmj.o2409.Commentaries, Editorials and LettersAdverse Drug Reaction Reporting SystemsDatabases, FactualDrug-Related Side Effects and Adverse ReactionsHumansProduct Surveillance, PostmarketingRenewing the Call for Reforms to Medical Device Safety—The Case of Penumbra
Kadakia KT, Beckman AL, Ross JS, Krumholz HM. Renewing the Call for Reforms to Medical Device Safety—The Case of Penumbra. JAMA Internal Medicine 2022, 182: 59-65. PMID: 34842892, DOI: 10.1001/jamainternmed.2021.6626.Commentaries, Editorials and LettersMeSH KeywordsDevice ApprovalEquipment and SuppliesEquipment SafetyHumansProduct Surveillance, PostmarketingRisk FactorsUnited StatesUnited States Food and Drug AdministrationConceptsReperfusion catheterClinical evidenceClass IPenumbra reperfusion catheterSingle-arm trialUser Facility Device Experience (MAUDE) databaseAdverse event reportsPostmarket surveillanceHealth policy makersMedical device reportsPenumbra deviceClinical evaluationPatient deathDevice safetyMedicine recommendationsAnimal dataCatheterFDA databaseFDA medical device regulationsClinical literatureSmall sample sizeDevice reportsEvent reportsPublic healthExperience database
2021
Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval
Wallach JD, Zhang AD, Skydel JJ, Bartlett VL, Dhruva SS, Shah ND, Ross JS. Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval. JAMA Network Open 2021, 4: e2133667. PMID: 34751763, PMCID: PMC8579227, DOI: 10.1001/jamanetworkopen.2021.33667.Peer-Reviewed Original ResearchMeSH KeywordsCross-Sectional StudiesData ScienceDrug ApprovalFeasibility StudiesHumansPragmatic Clinical Trials as TopicProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationComparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018
Wallach JD, Ramachandran R, Bruckner T, Ross JS. Comparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018. JAMA Network Open 2021, 4: e2133601. PMID: 34751764, PMCID: PMC8579231, DOI: 10.1001/jamanetworkopen.2021.33601.Peer-Reviewed Original ResearchMeSH KeywordsClinical Trials as TopicDrug ApprovalDrug IndustryHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationFulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway
Gyawali B, Ross JS, Kesselheim AS. Fulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway. JAMA Internal Medicine 2021, 181: 1275-1276. PMID: 34254981, DOI: 10.1001/jamainternmed.2021.4604.Commentaries, Editorials and LettersCharacteristics of Preapproval and Postapproval Studies of Vaccines Granted Accelerated Approval by the US Food and Drug Administration
Egilman A, Wallach JD, Puthumana J, Zhang AD, Schwartz JL, Ross JS. Characteristics of Preapproval and Postapproval Studies of Vaccines Granted Accelerated Approval by the US Food and Drug Administration. Journal Of General Internal Medicine 2021, 36: 3281-3284. PMID: 34131876, PMCID: PMC8205312, DOI: 10.1007/s11606-021-06943-x.Peer-Reviewed Original ResearchMeSH KeywordsDrug ApprovalHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationVaccinesUS Food and Drug Administration utilization of postmarketing requirements and postmarketing commitments, 2009–2018
Skydel JJ, Zhang AD, Dhruva SS, Ross JS, Wallach JD. US Food and Drug Administration utilization of postmarketing requirements and postmarketing commitments, 2009–2018. Clinical Trials 2021, 18: 488-499. PMID: 33863236, PMCID: PMC8292154, DOI: 10.1177/17407745211005044.Peer-Reviewed Original ResearchMeSH KeywordsCross-Sectional StudiesDrug ApprovalHumansProduct Surveillance, PostmarketingProspective StudiesRetrospective StudiesUnited StatesUnited States Food and Drug AdministrationConceptsClinical studiesPostmarketing requirementsUS FoodDrug AdministrationNew therapeuticsClinical evidenceNew prospective cohort studyProspective cohort studyCross-sectional studyDrug Administration approvalCohort studyRetrospective studyUnapproved indicationsAdministration approvalClinical indicationsClinical trialsBACKGROUND/Median numberDisease populationTherapeutic safetyTherapeutic indicationsSecondary analysisNovel therapeuticsSmall molecule drugsOriginal approvalReporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics
Varma T, Wallach JD, Miller JE, Schnabel D, Skydel JJ, Zhang AD, Dinan MA, Ross JS, Gross CP. Reporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics. JAMA Network Open 2021, 4: e217063. PMID: 33877309, PMCID: PMC8058642, DOI: 10.1001/jamanetworkopen.2021.7063.Peer-Reviewed Original ResearchConceptsNovel cancer therapeuticsPostmarketing studyBlack patientsCancer therapeuticsDemographic dataOlder adultsUS cancer populationUS Cancer StatisticsCross-sectional studyRace/ethnicityParticipants' demographic characteristicsPercentage of trialsPatient sexCancer populationClinical studiesCancer statisticsMAIN OUTCOMEDrug AdministrationFDA approvalStudy participantsCancer typesDemographic characteristicsDemographic informationStudy samplePatients
2020
Clinical Evidence Supporting US Food and Drug Administration Clearance of Novel Therapeutic Devices via the De Novo Pathway Between 2011 and 2019
Johnston JL, Dhruva SS, Ross JS, Rathi VK. Clinical Evidence Supporting US Food and Drug Administration Clearance of Novel Therapeutic Devices via the De Novo Pathway Between 2011 and 2019. JAMA Internal Medicine 2020, 180: 1701-1703. PMID: 33044513, PMCID: PMC7551221, DOI: 10.1001/jamainternmed.2020.3214.Peer-Reviewed Original ResearchMeSH KeywordsCross-Sectional StudiesDevice ApprovalEquipment and SuppliesEquipment SafetyHumansProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationMandatory Registration and Results Reporting of Real-World Evidence Studies of FDA-Regulated Medical Products
Dhruva SS, Shah ND, Ross JS. Mandatory Registration and Results Reporting of Real-World Evidence Studies of FDA-Regulated Medical Products. Mayo Clinic Proceedings 2020, 95: 2609-2611. PMID: 33289654, DOI: 10.1016/j.mayocp.2020.04.013.Commentaries, Editorials and LettersAssociation between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study
Hwang TJ, Ross JS, Vokinger KN, Kesselheim AS. Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study. The BMJ 2020, 371: m3434. PMID: 33028575, PMCID: PMC7537471, DOI: 10.1136/bmj.m3434.Peer-Reviewed Original ResearchMeSH KeywordsDrug ApprovalDrug EvaluationDrug Information ServicesEuropeHumansInformation DisseminationProduct Surveillance, PostmarketingProgram DevelopmentQuality ImprovementRetrospective StudiesUnited StatesConceptsEuropean Medicines AgencyRetrospective cohort studyTherapeutic valueHigh therapeutic valueNew drugsCohort studyLow therapeutic valueEMA approvalUS FoodDrug AdministrationMedicines AgencyFDA approvalDrugsFDARegulatory approvalApproval programsNew medicinesAssociationGreater proportionApprovalProportionAdministrationPostmarket Clinical Evidence for High-Risk Therapeutic Medical Devices Receiving Food and Drug Administration Premarket Approval in 2010 and 2011
Rathi VK, Krumholz HM, Masoudi FA, Ross JS. Postmarket Clinical Evidence for High-Risk Therapeutic Medical Devices Receiving Food and Drug Administration Premarket Approval in 2010 and 2011. JAMA Network Open 2020, 3: e2014496. PMID: 32857145, PMCID: PMC7455850, DOI: 10.1001/jamanetworkopen.2020.14496.Peer-Reviewed Original ResearchMeSH KeywordsClinical Trials as TopicCross-Sectional StudiesDevice ApprovalHumansMedical Device LegislationProduct Surveillance, PostmarketingProstheses and ImplantsUnited StatesUnited States Food and Drug AdministrationEarly experience with the FDA’s Breakthrough Devices program
Johnston JL, Dhruva SS, Ross JS, Rathi VK. Early experience with the FDA’s Breakthrough Devices program. Nature Biotechnology 2020, 38: 933-938. PMID: 32704171, DOI: 10.1038/s41587-020-0636-7.Peer-Reviewed Original ResearchDevice ApprovalEquipment and SuppliesHumansMedical Device LegislationProduct Surveillance, PostmarketingUnited StatesUnited States Food and Drug AdministrationDisagreements Within the US Food and Drug Administration Regarding Approval of Novel Therapeutic Agents, 2011-2015
MacGregor A, Zhang AD, Wallach JD, Ross JS, Herder M. Disagreements Within the US Food and Drug Administration Regarding Approval of Novel Therapeutic Agents, 2011-2015. JAMA Network Open 2020, 3: e209498. PMID: 32706379, PMCID: PMC7382003, DOI: 10.1001/jamanetworkopen.2020.9498.Peer-Reviewed Original ResearchMeSH KeywordsCross-Sectional StudiesDrug ApprovalDrugs, InvestigationalLegislation, DrugPharmaceutical PreparationsProduct Surveillance, PostmarketingRegistriesUnited StatesUnited States Food and Drug Administration