2023
US Food and Drug Administration Approval of Drugs Not Meeting Pivotal Trial Primary End Points, 2018-2021
Johnston J, Ross J, Ramachandran R. US Food and Drug Administration Approval of Drugs Not Meeting Pivotal Trial Primary End Points, 2018-2021. JAMA Internal Medicine 2023, 183: 376-380. PMID: 36780148, PMCID: PMC9926353, DOI: 10.1001/jamainternmed.2022.6444.Peer-Reviewed Original ResearchUsing real-world data from health systems to evaluate the safety and effectiveness of a catheter to treat ischemic ventricular tachycardia
Dhruva S, Zhang S, Chen J, Noseworthy P, Doshi A, Agboola K, Herrin J, Jiang G, Yu Y, Cafri G, Farr K, Mbwana M, Ross J, Coplan P, Drozda J. Using real-world data from health systems to evaluate the safety and effectiveness of a catheter to treat ischemic ventricular tachycardia. Journal Of Interventional Cardiac Electrophysiology 2023, 66: 1817-1825. PMID: 36738387, DOI: 10.1007/s10840-023-01496-x.Peer-Reviewed Original ResearchConceptsIschemic ventricular tachycardiaVentricular tachycardiaHealth system dataVT ablationSTSF catheterComposite outcomeHealth systemRisk differencePre-specified performance goalComposite safety outcomeReal-world safetyRoutine clinical practiceDrug Administration approvalAverage risk differenceThromboembolic eventsTotal patientsHeart failureProcedural complicationsMethodsAmong patientsAdministration approvalMayo ClinicSafety outcomesEffectiveness outcomesClinical practiceCatheter
2021
Comparison 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 ResearchAssessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020
Johnston JL, Dhruva SS, Ross JS, Rathi VK. Assessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020. JAMA Internal Medicine 2021, 181: 1409-1412. PMID: 34152383, PMCID: PMC8218229, DOI: 10.1001/jamainternmed.2021.3042.Peer-Reviewed Original ResearchCharacteristics 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 OUTCOMEUS 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 ResearchConceptsClinical 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 approval
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
2017
Inclusion of Demographic-Specific Information in Studies Supporting US Food & Drug Administration Approval of High-Risk Medical Devices
Dhruva SS, Mazure CM, Ross JS, Redberg RF. Inclusion of Demographic-Specific Information in Studies Supporting US Food & Drug Administration Approval of High-Risk Medical Devices. JAMA Internal Medicine 2017, 177: 1390-1391. PMID: 28738116, PMCID: PMC5818834, DOI: 10.1001/jamainternmed.2017.3148.Peer-Reviewed Original ResearchAdoption of immunotherapy into real-world practice: Insights from the use of checkpoint inhibitors.
O'Connor J, Seidl-Rathkopf K, You P, Nussbaum N, Torres A, Fessele K, Darius K, Adelson K, Yin E, Presley C, Chiang A, Ross J, Abernethy A, Gross C. Adoption of immunotherapy into real-world practice: Insights from the use of checkpoint inhibitors. Journal Of Clinical Oncology 2017, 35: e14583-e14583. DOI: 10.1200/jco.2017.35.15_suppl.e14583.Peer-Reviewed Original ResearchElectronic health record databaseReal-world practiceCheckpoint inhibitorsAdvanced non-small cell lung cancerFDA approvalNon-small cell lung cancerDeath-1 checkpoint inhibitorsMetastatic renal cell carcinomaAdoption of immunotherapyGeneralizability of trialsCell lung cancerRenal cell carcinomaDrug Administration approvalHealth record databaseChi-square testStudy drugOlder patientsSystemic therapyAdvanced melanomaMedian ageTherapy linesCell carcinomaLung cancerAdministration approvalClinical trialsClinical Evidence Supporting US Food and Drug Administration Approval of Otolaryngologic Prescription Drug Indications, 2005‐2014
Rathi VK, Wang B, Ross JS, Downing NS, Kesselheim AS, Gray ST. Clinical Evidence Supporting US Food and Drug Administration Approval of Otolaryngologic Prescription Drug Indications, 2005‐2014. Otolaryngology 2017, 156: 683-692. PMID: 28116974, DOI: 10.1177/0194599816689666.Peer-Reviewed Original ResearchConceptsPivotal studiesOriginal indicationPrimary endpointClinical evidenceDrug indicationsSupplemental indicationsUS FoodFDA approvalAvailable FDA documentsDouble-blinded studyDrug Administration approvalInformed treatment decisionsPivotal clinical studiesPremarket evidenceOtolaryngologic diseaseMedian enrollmentSurrogate markerAdministration approvalClinical studiesTreatment decisionsMost indicationsDrug AdministrationInitial approvalMultidisciplinary teamPrescription drugs
2016
Trends of Anti-Vascular Endothelial Growth Factor Use in Ophthalmology Among Privately Insured and Medicare Advantage Patients
Parikh R, Ross JS, Sangaralingham LR, Adelman RA, Shah ND, Barkmeier AJ. Trends of Anti-Vascular Endothelial Growth Factor Use in Ophthalmology Among Privately Insured and Medicare Advantage Patients. Ophthalmology 2016, 124: 352-358. PMID: 27890437, DOI: 10.1016/j.ophtha.2016.10.036.Peer-Reviewed Original ResearchMeSH KeywordsAdultAgedAged, 80 and overAngiogenesis InhibitorsBevacizumabCohort StudiesDatabases, FactualDiabetic RetinopathyDrug UtilizationFemaleHumansInsurance, HealthIntravitreal InjectionsMacular DegenerationMaleMedicare Part CMiddle AgedOphthalmologyPrivate SectorRanibizumabReceptors, Vascular Endothelial Growth FactorRecombinant Fusion ProteinsRetinal Vein OcclusionRetrospective StudiesUnited StatesVascular Endothelial Growth Factor AYoung AdultConceptsAge-related macular degenerationDiabetic retinal diseaseAnti-VEGF injectionsIntravitreal anti-VEGF injectionsRetinal vein occlusionAdministrative claims dataRetinal diseasesOphthalmic diseasesVein occlusionClaims dataUse of bevacizumabCurrent Procedural Terminology codesPatients 18 yearsRetrospective cohort studyMedicare Advantage patientsOptumLabs Data WarehouseDrug Administration approvalProcedural Terminology codesGrowth factor useRanibizumab useBevacizumab useAvailable medicationsCohort studyMedication useCommon medicationsParticipation of the elderly, women, and minorities in pivotal trials supporting 2011–2013 U.S. Food and Drug Administration approvals
Downing NS, Shah ND, Neiman JH, Aminawung JA, Krumholz HM, Ross JS. Participation of the elderly, women, and minorities in pivotal trials supporting 2011–2013 U.S. Food and Drug Administration approvals. Trials 2016, 17: 199. PMID: 27079511, PMCID: PMC4832528, DOI: 10.1186/s13063-016-1322-4.Peer-Reviewed Original ResearchMeSH KeywordsAdultAge FactorsAgedClinical Trials as TopicCross-Sectional StudiesDatabases, FactualDrug ApprovalEthnicityFemaleHealth Status DisparitiesHealthcare DisparitiesHumansMaleMiddle AgedMinority GroupsPatient SelectionSex FactorsTime FactorsUnited StatesUnited States Food and Drug AdministrationConceptsSubgroup efficacy analysesPivotal trialsEfficacy analysisNovel therapeuticsSubgroup analysisTrial participantsU.S. FoodMean proportionAvailable FDA documentsCross-sectional studyDrug Administration approvalBasis of approvalYears of ageTrial patientsElderly patientsMedian ageBlack patientsAsian patientsAdministration approvalClinical studiesFDA reviewersPatientsHispanic participantsTrialsDemographic characteristicsRevisiting Essure—Toward Safe and Effective Sterilization
Dhruva S, Ross J, Gariepy A. Revisiting Essure—Toward Safe and Effective Sterilization. Obstetrical & Gynecological Survey 2016, 71: 86-87. DOI: 10.1097/01.ogx.0000479905.52201.55.Commentaries, Editorials and Letters