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 ResearchConceptsTrial 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
A framework for the definition and interpretation of the use of surrogate endpoints in interventional trials
Ciani O, Manyara A, Davies P, Stewart D, Weir C, Young A, Blazeby J, Butcher N, Bujkiewicz S, Chan A, Dawoud D, Offringa M, Ouwens M, Hróbjartssson 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. A framework for the definition and interpretation of the use of surrogate endpoints in interventional trials. EClinicalMedicine 2023, 65: 102283. PMID: 37877001, PMCID: PMC10590868, DOI: 10.1016/j.eclinm.2023.102283.Peer-Reviewed Original ResearchSurrogate endpointsInterventional trialsIntermediate outcomesHealth technology assessment expertsPrimary outcome measureMeasures of functionTreatment effectsE-Delphi studyOutcome measuresConsensus meetingBetter HealthTarget outcomesTrialsEndpointLack of consensusOutcomesPatientsFuture reportingCurrent definitionUrgent needSymptomsCliniciansSettingBiomarkers
2022
Protocol 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 ResearchConceptsSurrogate 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
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
2019
Biomarkers as Surrogate Endpoints: Ongoing Opportunities for Validation
Zhang AD, Ross JS. Biomarkers as Surrogate Endpoints: Ongoing Opportunities for Validation. The Journal Of Law, Medicine & Ethics 2019, 47: 393-395. PMID: 31560627, DOI: 10.1177/1073110519876170.Commentaries, Editorials and Letters
2018
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
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 rateOutcomesTwo Decades of Cardiovascular Trials With Primary Surrogate Endpoints: 1990–2011
Bikdeli B, Punnanithinont N, Akram Y, Lee I, Desai NR, Ross JS, Krumholz HM. Two Decades of Cardiovascular Trials With Primary Surrogate Endpoints: 1990–2011. Journal Of The American Heart Association 2017, 6: e005285. PMID: 28325713, PMCID: PMC5524035, DOI: 10.1161/jaha.116.005285.Peer-Reviewed Original ResearchConceptsClinical outcome trialsOutcome trialsEndpoint trialsPrimary endpointCardiovascular trialsClinical outcome studiesClinical outcomesSurrogate markerPatient's perspectiveSurrogate endpointsOutcome studiesNew England JournalSample cohortTrial resultsTrialsSurrogate trialsEndpointPositive resultsAmerican MedicalInterventionHigh-impact journalsCohort