2022
Sex Differences in PrEsentation, Risk Factors, Drug and Interventional Therapies, and OUtcomes of Elderly PatientS with Pulmonary Embolism: Rationale and design of the SERIOUS-PE study
Bikdeli B, Piazza G, Jimenez D, Muriel A, Wang Y, Khairani CD, Rosovsky RP, Mehdipoor G, O'Donoghue ML, Spagnolo P, Dreyer RP, Bertoletti L, López-Jiménez L, Núñez MJ, Blanco-Molina Á, Bates SM, Gerhard-Herman M, Goldhaber SZ, Monreal M, Krumholz HM. Sex Differences in PrEsentation, Risk Factors, Drug and Interventional Therapies, and OUtcomes of Elderly PatientS with Pulmonary Embolism: Rationale and design of the SERIOUS-PE study. Thrombosis Research 2022, 214: 122-131. PMID: 35537232, DOI: 10.1016/j.thromres.2022.04.019.Peer-Reviewed Original ResearchMeSH KeywordsAgedFemaleHumansMaleMedicarePharmaceutical PreparationsPulmonary EmbolismRegistriesRisk FactorsSex CharacteristicsUnited StatesVenous ThromboembolismConceptsRisk factorsElderly patientsUS Medicare feeInterventional therapyMedicare feeInternational registryService beneficiariesSex differencesNational US databasePrognostic prediction rulePulmonary embolism studyLarge international registryPrincipal discharge diagnosisSex-based disparitiesAssessment of outcomesPotential sex differencesSex-specific findingsPE presentationPulmonary embolismVenous thromboembolismTreatment patternsDischarge diagnosisAvailable therapiesDisease presentationTreatment-based differences
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 ResearchMeSH KeywordsBiological ProductsClinical Trials as TopicCross-Sectional StudiesDrug ApprovalHumansPharmaceutical PreparationsUnited StatesUnited States Food and Drug AdministrationConceptsPivotal 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
2014
Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012
Downing NS, Aminawung JA, Shah ND, Krumholz HM, Ross JS. Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012. JAMA 2014, 311: 368-377. PMID: 24449315, PMCID: PMC4144867, DOI: 10.1001/jama.2013.282034.Peer-Reviewed Original ResearchConceptsPivotal efficacy trialsNovel therapeutic agentsClinical trial evidencePivotal trialsEfficacy trialsTherapeutic agentsEnd pointTrial evidenceMedian numberAvailable FDA documentsSingle pivotal trialTrial end pointsSurrogate end pointsNumber of patientsLength of treatmentCross-sectional analysisPrimary outcomeClinical outcomesTrial completion ratesClinical benefitPlacebo comparatorSurrogate outcomesMAIN OUTCOMEDrug AdministrationUS Food