2024
Reporting of surrogate endpoints in randomised controlled trial protocols (SPIRIT-Surrogate): extension checklist with explanation and elaboration
Manyara A, Davies P, Stewart D, Weir C, Young A, Blazeby J, Butcher N, Bujkiewicz S, Chan A, Dawoud D, Offringa M, Ouwens M, Hróbjartsson A, Amstutz A, Bertolaccini L, Bruno V, Devane D, Faria C, Gilbert P, Harris R, Lassere M, Marinelli L, Markham S, Powers J, Rezaei Y, Richert L, Schwendicke F, Tereshchenko L, Thoma A, Turan A, Worrall A, Christensen R, Collins G, Ross J, Taylor R, Ciani O. Reporting of surrogate endpoints in randomised controlled trial protocols (SPIRIT-Surrogate): extension checklist with explanation and elaboration. The BMJ 2024, 386: e078525. PMID: 38981624, PMCID: PMC11231880, DOI: 10.1136/bmj-2023-078525.Peer-Reviewed Original ResearchMeSH KeywordsBiomarkersChecklistClinical Trial Protocols as TopicHumansRandomized Controlled Trials as TopicResearch DesignConceptsTrial protocolRandomised controlled trial protocolTarget outcomesReduce research wasteStandard Protocol ItemsRandomised controlled trialsIntervention effectsProtocol ItemsInterpretation of findingsResearch wasteImprove reportingPrimary outcome(sIntervention trialsControlled trialsChecklistSurrogate endpointsInterventionItemsIncreasing callsTrialsGuidelinesOutcomesLimited informationOutcome(sHarmReporting of surrogate endpoints in randomised controlled trial reports (CONSORT-Surrogate): extension checklist with explanation and elaboration
Manyara A, Davies P, Stewart D, Weir C, Young A, Blazeby J, Butcher N, Bujkiewicz S, Chan A, Dawoud D, Offringa M, Ouwens M, Hróbjartsson A, Amstutz A, Bertolaccini L, Bruno V, Devane D, Faria C, Gilbert P, Harris R, Lassere M, Marinelli L, Markham S, Powers J, Rezaei Y, Richert L, Schwendicke F, Tereshchenko L, Thoma A, Turan A, Worrall A, Christensen R, Collins G, Ross J, Taylor R, Ciani O. Reporting of surrogate endpoints in randomised controlled trial reports (CONSORT-Surrogate): extension checklist with explanation and elaboration. The BMJ 2024, 386: e078524. PMID: 38981645, PMCID: PMC11231881, DOI: 10.1136/bmj-2023-078524.Peer-Reviewed Original ResearchConceptsRandomised controlled trial reportsTrial reportsRandomised controlled trialsControlled trial reportsIntervention treatment effectsResearch wasteTrial findingsTreatment effectsControlled trialsTarget outcomesChecklistAdequate informationSurrogate endpointsInterventionCONSORTItemsTrialsImprove transparencyHarmReportsGuidelinesOutcomesEndpoint
2023
Assessing the use of observational methods and real-world data to emulate ongoing randomized controlled trials
Wallach J, Deng Y, Polley E, Dhruva S, Herrin J, Quinto K, Gandotra C, Crown W, Noseworthy P, Yao X, Jeffery M, Lyon T, Ross J, McCoy R. Assessing the use of observational methods and real-world data to emulate ongoing randomized controlled trials. Clinical Trials 2023, 20: 689-698. PMID: 37589143, PMCID: PMC10843567, DOI: 10.1177/17407745231193137.Peer-Reviewed Original ResearchMeSH KeywordsHumansLongitudinal StudiesMyocardial InfarctionPandemicsRandomized Controlled Trials as TopicResearch DesignConceptsBaseline participant characteristicsParticipant characteristicsPrimary endpointSecondary endpointsTrial publicationsMajor adverse cardiovascular eventsPropensity score-matched participantsFirst major adverse cardiovascular eventAdverse cardiovascular eventsBaseline patient characteristicsNonfatal myocardial infarctionOptumLabs Data WarehouseElectronic health record dataRepresentative patient populationHealth record dataCardiovascular eventsClinical characteristicsPatient characteristicsPatient populationMyocardial infarctionExclusion criteriaDrug effectivenessTrialsRecord dataEndpointUS 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 ResearchMeSH KeywordsDrug ApprovalHumansPharmaceutical PreparationsResearch DesignUnited StatesUnited States Food and Drug AdministrationEvidence of publication bias in multiple sclerosis clinical trials: a comparative analysis of published and unpublished studies registered in ClinicalTrials.gov
Rivero-de-Aguilar A, Pérez-Ríos M, Ruano-Raviña A, Candal-Pedreira C, Puente-Hernandez M, Ross J, Varela-Lema L. Evidence of publication bias in multiple sclerosis clinical trials: a comparative analysis of published and unpublished studies registered in ClinicalTrials.gov. Journal Of Neurology Neurosurgery & Psychiatry 2023, 94: 597-604. PMID: 36977551, DOI: 10.1136/jnnp-2023-331132.Peer-Reviewed Original ResearchConceptsClinical trialsPeer-reviewed journalsTrial publicationsPublication biasMultivariate logistic regression analysisMultiple sclerosis clinical trialsFavorable primary outcomeMS clinical researchPhase IIILogistic regression analysisMultiple sclerosis drugsCase-control designTreatment tolerabilityMore patientsPrimary outcomeUnpublished trialsMS drugsTreatment decisionsLower oddsStudy design characteristicsMultivariate analysisClinical researchUnpublished studiesTrialsGoogle Scholar
2022
Consistency between trials presented at conferences, their subsequent publications and press releases
Rowhani-Farid A, Hong K, Grewal M, Reynolds J, Zhang A, Wallach J, Ross J. Consistency between trials presented at conferences, their subsequent publications and press releases. BMJ Evidence-Based Medicine 2022, 28: 95-102. PMID: 36357160, PMCID: PMC10086295, DOI: 10.1136/bmjebm-2022-111989.Peer-Reviewed Original ResearchConceptsClinical trialsConference abstractsTrial resultsEndpoint definitionsPrimary efficacy end pointEnd pointEfficacy end pointPrimary efficacy endpointClinical Trials RegistryMedical conferencesMultiple logistic regressionCross-sectional analysisSafety endpointEfficacy endpointPeer-reviewed journalsTrial abstractsTrials RegistryTrial characteristicsConsistency of reportingSample sizePrimary analysisSecondary analysisInternational medical conferencesLogistic regressionStudy designProtocol for the development of SPIRIT and CONSORT extensions for randomised controlled trials with surrogate primary endpoints: SPIRIT-SURROGATE and CONSORT-SURROGATE
Manyara AM, Davies P, Stewart D, Weir CJ, Young A, Butcher NJ, Bujkiewicz S, Chan AW, Collins GS, Dawoud D, Offringa M, Ouwens M, Ross JS, Taylor RS, Ciani O. Protocol for the development of SPIRIT and CONSORT extensions for randomised controlled trials with surrogate primary endpoints: SPIRIT-SURROGATE and CONSORT-SURROGATE. BMJ Open 2022, 12: e064304. PMID: 36220321, PMCID: PMC9557267, DOI: 10.1136/bmjopen-2022-064304.Peer-Reviewed Original ResearchMeSH KeywordsConsensusHumansPublicationsRandomized Controlled Trials as TopicResearch DesignResearch ReportTreatment OutcomeConceptsSurrogate endpointsPeer-reviewed publicationsSurrogate primary endpointOpen-access peer-reviewed publicationPATIENTS/PARTICIPANTSCompleteness of reportingTranslation of effectsPhase 1Primary endpointPrimary outcomeTrial findingsEthical approvalCONSORT extensionSuch trialsEthics CommitteeEndpointHealth benefitsTrialsPhase 3Final outcomePhase 2Transparent reportingOutcomesPhase 4Additional items
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 OUTCOMEMisreporting of Results of Research in Psychiatry
Bowcut J, Levi L, Livnah O, Ross JS, Knable M, Davidson M, Davis JM, Weiser M. Misreporting of Results of Research in Psychiatry. Schizophrenia Bulletin 2021, 47: 1254-1260. PMID: 33860793, PMCID: PMC8379531, DOI: 10.1093/schbul/sbab040.Peer-Reviewed Original ResearchCharacteristics and Reporting of Number Needed to Treat, Number Needed to Harm, and Absolute Risk Reduction in Controlled Clinical Trials, 2001-2019
Elliott MH, Skydel JJ, Dhruva SS, Ross JS, Wallach JD. Characteristics and Reporting of Number Needed to Treat, Number Needed to Harm, and Absolute Risk Reduction in Controlled Clinical Trials, 2001-2019. JAMA Internal Medicine 2021, 181: 282-284. PMID: 33226398, PMCID: PMC7684521, DOI: 10.1001/jamainternmed.2020.4799.Peer-Reviewed Original Research
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 interpretationOutcomesMarkersInterventionConsistency of trial reporting between ClinicalTrials.gov and corresponding publications: one decade after FDAAA
Talebi R, Redberg RF, Ross JS. Consistency of trial reporting between ClinicalTrials.gov and corresponding publications: one decade after FDAAA. Trials 2020, 21: 675. PMID: 32703252, PMCID: PMC7376878, DOI: 10.1186/s13063-020-04603-9.Peer-Reviewed Original Research
2019
Age-treatment subgroup analyses in Cochrane intervention reviews: a meta-epidemiological study
Liu P, Ioannidis JPA, Ross JS, Dhruva SS, Luxkaranayagam AT, Vasiliou V, Wallach JD. Age-treatment subgroup analyses in Cochrane intervention reviews: a meta-epidemiological study. BMC Medicine 2019, 17: 188. PMID: 31639007, PMCID: PMC6805640, DOI: 10.1186/s12916-019-1420-8.Peer-Reviewed Original ResearchConceptsCochrane intervention reviewsFormal interaction testingSubgroup analysisIntervention reviewsClinical practice resourcesInsufficient trial dataPotential subgroup differencesMeta-epidemiological studyIndividual subgroup analysisCochrane reviewAppropriate statistical testsClinical significanceClinical careSubgroup findingsTrial dataClinical resourcesHealthcare interventionsBiological rationaleAnalysis of ageTrialsIndividual trialsDemographic characteristicsPractice resourcesSubgroup differencesClinical translationCommunicating, and Justifying, Nonefficacy Benefits for New Drugs Approved on the Basis of Noninferiority Trials
Ross JS, Wee CC. Communicating, and Justifying, Nonefficacy Benefits for New Drugs Approved on the Basis of Noninferiority Trials. JAMA Internal Medicine 2019, 179: 721-722. PMID: 30830205, DOI: 10.1001/jamainternmed.2018.8019.Commentaries, Editorials and Letters
2018
Registration, results reporting, and publication bias of clinical trials supporting FDA approval of neuropsychiatric drugs before and after FDAAA: a retrospective cohort study
Zou CX, Becker JE, Phillips AT, Garritano JM, Krumholz HM, Miller JE, Ross JS. Registration, results reporting, and publication bias of clinical trials supporting FDA approval of neuropsychiatric drugs before and after FDAAA: a retrospective cohort study. Trials 2018, 19: 581. PMID: 30352601, PMCID: PMC6199729, DOI: 10.1186/s13063-018-2957-0.Peer-Reviewed Original ResearchConceptsRetrospective cohort studyPublication biasNeuropsychiatric indicationsCohort studyClinical trialsRelative riskEfficacy trialsFDA approvalPositive trialsFisher's exact testRecent FDA approvalDrug Administration Amendments ActClinical trial publicationsTRIAL REGISTRATIONMAIN OUTCOMEProportion of trialsNeuropsychiatric drugsNew drug approvalsTrial publicationsExact testMedical interventionsTrialsDrug approvalNew drugsDrugsAdherence to the International Committee of Medical Journal Editors’ (ICMJE) prospective registration policy and implications for outcome integrity: a cross-sectional analysis of trials published in high-impact specialty society journals
Gopal AD, Wallach JD, Aminawung JA, Gonsalves G, Dal-Ré R, Miller JE, Ross JS. Adherence to the International Committee of Medical Journal Editors’ (ICMJE) prospective registration policy and implications for outcome integrity: a cross-sectional analysis of trials published in high-impact specialty society journals. Trials 2018, 19: 448. PMID: 30134950, PMCID: PMC6106722, DOI: 10.1186/s13063-018-2825-y.Peer-Reviewed Original ResearchConceptsPrimary outcome dataOutcome dataCross-sectional analysisUnregistered trialsTrial characteristicsClinical trialsFavorable findingsProfessional medical societiesMedical societiesIndustry-funded trialsFrequency of registrationPrimary outcomeRegistered trialsEnrollment siteProspective fashionChi-square analysisProportion of trialsOriginal research reportsInitial ascertainmentTrialsHigh-impact journalsMedical Journal EditorsRegistration timingRetrospective registrationLower ratesAge of Data at the Time of Publication of Contemporary Clinical Trials
Welsh J, Lu Y, Dhruva SS, Bikdeli B, Desai NR, Benchetrit L, Zimmerman CO, Mu L, Ross JS, Krumholz HM. Age of Data at the Time of Publication of Contemporary Clinical Trials. JAMA Network Open 2018, 1: e181065-e181065. PMID: 30646100, PMCID: PMC6324269, DOI: 10.1001/jamanetworkopen.2018.1065.Peer-Reviewed Original ResearchMeSH KeywordsCross-Sectional StudiesJournal Impact FactorPublishingRandomized Controlled Trials as TopicResearch DesignTime FactorsConceptsClinical trialsFinal data collectionParticipant enrollmentInternal medicineMultivariable linear regression analysisFirst participant enrollmentPrimary end pointMultivariable regression analysisContemporary clinical trialsClinical trial dataJAMA Internal MedicineRegression analysisCross-sectional analysisTime of publicationMedian timeTrial characteristicsOutcome measuresMAIN OUTCOMENew England JournalClinical practiceLinear regression analysisTrial dataEnd pointTrial resultsTrialsFactors Associated With Postmarketing Research for Approved Indications for Novel Medicines Approved by Both the FDA and EMA Between 2005 and 2010: A Multivariable Analysis
Zeitoun J, Ross JS, Atal I, Vivot A, Downing NS, Baron G, Ravaud P. Factors Associated With Postmarketing Research for Approved Indications for Novel Medicines Approved by Both the FDA and EMA Between 2005 and 2010: A Multivariable Analysis. Clinical Pharmacology & Therapeutics 2018, 104: 1000-1007. PMID: 29377075, DOI: 10.1002/cpt.1038.Peer-Reviewed Original Research
2017
Post‐marketing research and its outcome for novel anticancer agents approved by both the FDA and EMA between 2005 and 2010: A cross‐sectional study
Zeitoun J, Baron G, Vivot A, Atal I, Downing NS, Ross JS, Ravaud P. Post‐marketing research and its outcome for novel anticancer agents approved by both the FDA and EMA between 2005 and 2010: A cross‐sectional study. International Journal Of Cancer 2017, 142: 414-423. PMID: 28929484, DOI: 10.1002/ijc.31061.Peer-Reviewed Original ResearchConceptsPost-marketing trialsPrimary outcomeNovel anticancer agentsPost-market researchAnticancer agentsSupplemental indicationsCross-sectional studyPost-marketing researchEuropean Medicines AgencyCross-sectional analysisTypes of cancerPost-market trialClinical endpointsHematologic malignanciesKidney cancerSolid cancersSurrogate endpointsUS FoodDrug AdministrationMedicines AgencyOverall populationCancerTrialsPublication rateOutcomesAssociation of the FDA Amendment Act with trial registration, publication, and outcome reporting
Phillips AT, Desai NR, Krumholz HM, Zou CX, Miller JE, Ross JS. Association of the FDA Amendment Act with trial registration, publication, and outcome reporting. Trials 2017, 18: 333. PMID: 28720112, PMCID: PMC5516301, DOI: 10.1186/s13063-017-2068-3.Peer-Reviewed Original ResearchConceptsCardiovascular diseaseTRIAL REGISTRATIONFDA approvalNew drugsClinical trial registrationFDA Amendments ActDrug Administration Amendments ActAccessible trial registryClinical trial publicationsTrials RegistryResultsBetween 2005Efficacy trialsFDA reviewersTrial publicationsOutcome reportingMedical literatureReviewer interpretationsTrialsReporting of findingsDiseaseFDA documentsDrugsDiabetesFDAAAReviewers