2020
Non-inferiority trials using a surrogate marker as the primary endpoint: An increasing phenotype in cardiovascular trials
Bikdeli B, Caraballo C, Welsh J, Ross JS, Kaul S, Stone GW, Krumholz HM. Non-inferiority trials using a surrogate marker as the primary endpoint: An increasing phenotype in cardiovascular trials. Clinical Trials 2020, 17: 723-728. PMID: 32838556, PMCID: PMC8088773, DOI: 10.1177/1740774520949157.Peer-Reviewed Original ResearchConceptsNon-inferiority trialPrimary endpointClinical outcome trialsNon-inferiority marginSurrogate markerNon-inferiority designCardiovascular trialsOutcome trialsClinical outcomesDefinitive clinical outcome trialsNon-inferiority criteriaStudy protocolSurrogate outcomesBACKGROUND/Median numberSurrogate endpointsPrimary analysisCardiovascular interventionsCardiovascular medicineTrialsEndpointClinical interpretationOutcomesMarkersIntervention
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