2024
Associations Between Surrogate Markers and Clinical Outcomes for Nononcologic Chronic Disease Treatments
Wallach J, Yoon S, Doernberg H, Glick L, Ciani O, Taylor R, Mooghali M, Ramachandran R, Ross J. Associations Between Surrogate Markers and Clinical Outcomes for Nononcologic Chronic Disease Treatments. JAMA 2024, 331: 1646-1654. PMID: 38648042, PMCID: PMC11036312, DOI: 10.1001/jama.2024.4175.Peer-Reviewed Original ResearchConceptsMeta-analysesChronic diseasesFood and Drug AdministrationMeta-regression analysisMeta-analyses of observational studiesSurrogate markerClinical outcomesClinical trialsMeta-analysisEvidence of associationPublished meta-analysesStrength of associationMeta-analyses of clinical trialsChronic disease treatmentFood and Drug Administration tableResults of meta-regression analysisOutcome pairsFood and Drug Administration approvalMain OutcomesData extractionSystematic reviewStatistically significant resultsObservational studyPooled analysisDrug AdministrationReporting of Demographics & Subgroup Analyses in Premarketing Studies of FDA Approved High-Risk Cardiovascular Devices, 2014–2022
Swanson M, Uyeki C, Yoder S, Dhruva S, Miller J, Ross J. Reporting of Demographics & Subgroup Analyses in Premarketing Studies of FDA Approved High-Risk Cardiovascular Devices, 2014–2022. Medical Devices Evidence And Research 2024, 17: 165-172. PMID: 38707869, PMCID: PMC11067925, DOI: 10.2147/mder.s457152.Peer-Reviewed Original ResearchFood and Drug AdministrationHigh-risk cardiovascular devicesSubgroup analysisPremarketing studiesUS Food and Drug AdministrationResults of subgroup analysisParticipation of older adultsStudy participantsClinical trialsSocioeconomic positionTrial populationDrug AdministrationStudy populationConduction of subgroup analysisDemographic dataOlder adultsReporting of demographicsSubgroupsPatients' socioeconomic position.PatientsTrials
2023
Geographic and Demographic Representation in Industry-Sponsored, US-Based Clinical Trials of Systemic Lupus Erythematosus Therapies
Skydel J, Ramachandran R, Suttiratana S, Ross J, Burns C, Wallach J. Geographic and Demographic Representation in Industry-Sponsored, US-Based Clinical Trials of Systemic Lupus Erythematosus Therapies. The Journal Of Rheumatology 2023, 51: jrheum.2023-0920. PMID: 38101910, PMCID: PMC10922605, DOI: 10.3899/jrheum.2023-0920.Peer-Reviewed Original ResearchFeasibility of Emulating Clinical Trials Supporting US FDA Supplemental Indication Approvals of Drugs and Biologics
Janda G, Wallach J, Dhodapkar M, Ramachandran R, Ross J. Feasibility of Emulating Clinical Trials Supporting US FDA Supplemental Indication Approvals of Drugs and Biologics. JAMA Internal Medicine 2023, 183: 1271-1273. PMID: 37782514, PMCID: PMC10546285, DOI: 10.1001/jamainternmed.2023.4073.Peer-Reviewed Original ResearchEvidence 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
Comparison of Clinical Study Results Reported in medRxiv Preprints vs Peer-reviewed Journal Articles
Janda G, Khetpal V, Shi X, Ross J, Wallach J. Comparison of Clinical Study Results Reported in medRxiv Preprints vs Peer-reviewed Journal Articles. JAMA Network Open 2022, 5: e2245847. PMID: 36484989, PMCID: PMC9856222, DOI: 10.1001/jamanetworkopen.2022.45847.Peer-Reviewed Original ResearchConceptsPrimary end pointPrimary end point resultsPeer-reviewed journalsClinical studiesEnd pointClinical trialsObservational studyStudy characteristicsEnd point resultsClinical study resultsMost clinical studiesCross-sectional studyHealth-related outcomesSample sizeMAIN OUTCOMEPeer-reviewed publicationsMedRxivEffect estimatesStudy interpretationDiscordant pairsCOVID-19TrialsPopulation-Level Strategies for Nirmatrelvir/Ritonavir Prescribing—A Cost-effectiveness Analysis
Savinkina A, Paltiel A, Ross J, Gonsalves G. Population-Level Strategies for Nirmatrelvir/Ritonavir Prescribing—A Cost-effectiveness Analysis. Open Forum Infectious Diseases 2022, 9: ofac637. PMID: 36589482, PMCID: PMC9792084, DOI: 10.1093/ofid/ofac637.Peer-Reviewed Original ResearchCost-effectiveness analysisTrade-offs implicitSevere diseaseDecision analytic modelAllocation decisionsPay thresholdsPolicy strategiesUnvaccinated patientsVaccination statusHigh riskTreatment effectivenessHigh-risk individualsAllocation strategyPopulation-level strategiesWillingnessFurther priorityDrug costsSensitivity analysisCostRitonavir treatmentUnvaccinated personsVaccinated adultsPatient populationClinical trialsLower riskConsistency 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 designSpending by the Centers for Medicare & Medicaid Services Before and After Confirmation of Benefit for Drugs Granted US Food and Drug Administration Accelerated Approval, 2012 to 2017
Skydel JJ, Egilman AC, Wallach JD, Ramachandran R, Gupta R, Ross JS. Spending by the Centers for Medicare & Medicaid Services Before and After Confirmation of Benefit for Drugs Granted US Food and Drug Administration Accelerated Approval, 2012 to 2017. JAMA Health Forum 2022, 3: e221158. PMID: 35977252, PMCID: PMC9142876, DOI: 10.1001/jamahealthforum.2022.1158.Peer-Reviewed Original ResearchConceptsSurrogate end pointsCross-sectional studyClinical benefitConfirmation of benefitEnd pointOriginal indicationClinical outcomesUS FoodConfirmatory trialsMedicaid ServicesPrimary end pointUnproven clinical benefitsTrial end pointsClinical trial resultsTypes of drugsPostapproval trialsAccelerated approvalClinical trialsMAIN OUTCOMEDrug AdministrationMedicare Part BStandard approvalConversion statusSupplemental indicationsTrial results
2021
Heart Watch Study: protocol for a pragmatic randomised controlled trial
Dhruva SS, Shah ND, Vemulapalli S, Deshmukh A, Beatty AL, Gamble GM, Freeman JV, Hummel JP, Piccini JP, Akar JG, Ervin K, Arges KL, Emanuel L, Noseworthy PA, Hu T, Bartlett V, Ross JS. Heart Watch Study: protocol for a pragmatic randomised controlled trial. BMJ Open 2021, 11: e054550. PMID: 35234659, PMCID: PMC8719216, DOI: 10.1136/bmjopen-2021-054550.Peer-Reviewed Original ResearchConceptsHealthcare utilisationSecondary outcomesAtrial fibrillationAtrial flutterDuke University Health SystemRoutine clinical care settingsAtrial Fibrillation EffectLife global scoresTime of cardioversionAdditional secondary outcomesPopulation of patientsPatient-reported outcomesClinical care settingsUniversity Health SystemQuality of lifeInstitutional review boardMulticentre pragmaticPeer-reviewed journalsPrimary outcomePatients' qualityTrial protocolClinical trialsMayo ClinicECG featuresCare settingsStrengthening the FDA’s Enforcement of ClinicalTrials.gov Reporting Requirements
Ramachandran R, Morten CJ, Ross JS. Strengthening the FDA’s Enforcement of ClinicalTrials.gov Reporting Requirements. JAMA 2021, 326: 2131-2132. PMID: 34766971, DOI: 10.1001/jama.2021.19773.Commentaries, Editorials and LettersMeSH KeywordsActivin Receptors, Type IIAntineoplastic AgentsAxitinibCarcinoma, Renal CellClinical Trials as TopicClinical Trials, Phase II as TopicDrug IndustryHumansImmunoglobulin Fc FragmentsKidney NeoplasmsLaw EnforcementMandatory ReportingNational Institutes of Health (U.S.)Recombinant Fusion ProteinsRegistriesUnited StatesUnited States Food and Drug AdministrationRegistration, publication, and outcome reporting among pivotal clinical trials that supported FDA approval of high-risk cardiovascular devices before and after FDAAA
Swanson MJ, Johnston JL, Ross JS. Registration, publication, and outcome reporting among pivotal clinical trials that supported FDA approval of high-risk cardiovascular devices before and after FDAAA. Trials 2021, 22: 817. PMID: 34789308, PMCID: PMC8597303, DOI: 10.1186/s13063-021-05790-9.Peer-Reviewed Original ResearchConceptsHigh-risk cardiovascular devicesPrimary efficacy outcomeFDA Amendments ActPivotal clinical trialsClinical trialsEfficacy outcomesFDA approvalTrial interpretationPivotal clinical studiesPrimary clinical evidenceFDA summariesPivotal trialsClinical evidenceResultsBetween 2005Clinical studiesHigh-risk medical devicesOutcome reportingUS FoodDrug AdministrationTrialsReporting of findingsInterpretation of findingsPeer-reviewed literatureOutcomesCardiovascular devicesFeasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval
Wallach JD, Zhang AD, Skydel JJ, Bartlett VL, Dhruva SS, Shah ND, Ross JS. Feasibility of Using Real-world Data to Emulate Postapproval Confirmatory Clinical Trials of Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval. JAMA Network Open 2021, 4: e2133667. PMID: 34751763, PMCID: PMC8579227, DOI: 10.1001/jamanetworkopen.2021.33667.Peer-Reviewed Original ResearchAccess to COVID-19 Vaccines in High-, Middle-, and Low-Income Countries Hosting Clinical Trials
Ramachandran R, Ross JS, Miller JE. Access to COVID-19 Vaccines in High-, Middle-, and Low-Income Countries Hosting Clinical Trials. JAMA Network Open 2021, 4: e2134233. PMID: 34792596, PMCID: PMC8603077, DOI: 10.1001/jamanetworkopen.2021.34233.Peer-Reviewed Original ResearchReal-world Cardiovascular Outcomes Associated With Degarelix vs Leuprolide for Prostate Cancer Treatment
Wallach JD, Deng Y, McCoy RG, Dhruva SS, Herrin J, Berkowitz A, Polley EC, Quinto K, Gandotra C, Crown W, Noseworthy P, Yao X, Shah ND, Ross JS, Lyon TD. Real-world Cardiovascular Outcomes Associated With Degarelix vs Leuprolide for Prostate Cancer Treatment. JAMA Network Open 2021, 4: e2130587. PMID: 34677594, PMCID: PMC8536955, DOI: 10.1001/jamanetworkopen.2021.30587.Peer-Reviewed Original ResearchConceptsMajor adverse cardiovascular eventsSecondary end pointsMyocardial infarctionClinical trialsCardiovascular diseaseProstate cancerCardiovascular eventsEnd pointRisk of MACELarge US administrative claims databasePropensity-matched cohort studyUS administrative claims databasePropensity score-matched patientsAdverse cardiovascular eventsPrimary end pointAdministrative claims databaseProportional hazards regressionRandomized clinical trialsAdministrative claims dataTrial eligibility criteriaMedicare Advantage beneficiariesProstate cancer treatmentReal-world evidenceElectronic health recordsCardiovascular outcomesClinical trial transparency and data sharing among biopharmaceutical companies and the role of company size, location and product type: a cross-sectional descriptive analysis
Axson S, Mello MM, Lincow D, Yang C, Gross C, Ross JS, Miller J. Clinical trial transparency and data sharing among biopharmaceutical companies and the role of company size, location and product type: a cross-sectional descriptive analysis. BMJ Open 2021, 11: e053248. PMID: 34281933, PMCID: PMC8291313, DOI: 10.1136/bmjopen-2021-053248.Peer-Reviewed Original ResearchDrug labeling changes and pediatric hematology/oncology prescribing: Measuring the impact of U.S. legislation
Benning TJ, Shah ND, Inselman JW, Van Houten HK, Ross JS, Wyatt KD. Drug labeling changes and pediatric hematology/oncology prescribing: Measuring the impact of U.S. legislation. Clinical Trials 2021, 18: 732-740. PMID: 34269090, PMCID: PMC10119689, DOI: 10.1177/17407745211030683.Peer-Reviewed Original ResearchConceptsPediatric Research Equity ActClinical trialsBest PharmaceuticalsOncology drugsDrug utilizationPediatric indicationsLabel changesPediatric-specific evidenceAdministrative claims databasePediatric Hematology/OncologyQuality of evidenceDrug labelling changesHematology/oncologyDrug utilization ratesNew pediatric indicationsLow methodological rigorClaims databaseComparator groupPediatric utilizationTrial characteristicsBACKGROUND/Pediatric useMonthly utilization ratesDrug safetyStudy designEvaluation of Trials Comparing Single-Enantiomer Drugs to Their Racemic Precursors
Long AS, Zhang AD, Meyer CE, Egilman AC, Ross JS, Wallach JD. Evaluation of Trials Comparing Single-Enantiomer Drugs to Their Racemic Precursors. JAMA Network Open 2021, 4: e215731. PMID: 33956134, PMCID: PMC8103227, DOI: 10.1001/jamanetworkopen.2021.5731.Peer-Reviewed Original ResearchConceptsRandomized clinical trialsSafety end pointEnd pointClinical trialsDrug pairsPrimary end point resultsEnd point resultsImproved efficacyEfficacy end pointCochrane Central RegistrySeparate end pointsSingle-enantiomer drugsSingle enantiomer formulationPrimary efficacySecondary efficacyControlled TrialsOvid EmbaseOvid MEDLINESuperior efficacyDrug AdministrationCentral RegistrySystematic reviewMore efficacyTrialsRacemic drugsUS 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 approvalMisreporting 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 Research