2023
Therapeutic value of first versus supplemental indications of drugs in US and Europe (2011-20): retrospective cohort study
Vokinger K, Glaus C, Kesselheim A, Serra-Burriel M, Ross J, Hwang T. Therapeutic value of first versus supplemental indications of drugs in US and Europe (2011-20): retrospective cohort study. The BMJ 2023, 382: e074166. PMID: 37407074, PMCID: PMC10320829, DOI: 10.1136/bmj-2022-074166.Peer-Reviewed Original ResearchConceptsEuropean Medicines AgencyRetrospective cohort studyCohort studyTherapeutic valueSupplemental indicationsIndication approvalsHigh therapeutic valueAvailable treatmentsDrug AdministrationUS FoodMedicines AgencyFDA approvalHealth authoritiesCancer disordersSimilar findingsTherapeutic ratingFirst indicationDrugsIndicationsApprovalFDAPatientsLarge subsetProportionPhysicians
2021
Registration, 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 devicesAssessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020
Johnston JL, Dhruva SS, Ross JS, Rathi VK. Assessment of FDA Approval for New High-risk Therapeutic Devices Not Meeting Pivotal Study Primary End Points, 2016-2020. JAMA Internal Medicine 2021, 181: 1409-1412. PMID: 34152383, PMCID: PMC8218229, DOI: 10.1001/jamainternmed.2021.3042.Peer-Reviewed Original ResearchClinical 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 ResearchEvaluation of Drug Trials in High-, Middle-, and Low-Income Countries and Local Commercial Availability of Newly Approved Drugs
Miller JE, Mello MM, Wallach JD, Gudbranson EM, Bohlig B, Ross JS, Gross CP, Bach PB. Evaluation of Drug Trials in High-, Middle-, and Low-Income Countries and Local Commercial Availability of Newly Approved Drugs. JAMA Network Open 2021, 4: e217075. PMID: 33950209, PMCID: PMC8100865, DOI: 10.1001/jamanetworkopen.2021.7075.Peer-Reviewed Original ResearchConceptsFDA approvalHigh-income countriesDrug approvalIncome countriesNovel drugsDrug Administration (FDA) drug approvalsRegulatory agency websitesCross-sectional analysisProportion of drugPrimary outcomeFDA drug approvalsProportion of countriesDrug trialsMAIN OUTCOMEUS FoodClinical researchCountry income levelLow-income countriesMarketing approvalTrialsGoogle ScholarDrugsDrug companiesApprovalMedianReporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics
Varma T, Wallach JD, Miller JE, Schnabel D, Skydel JJ, Zhang AD, Dinan MA, Ross JS, Gross CP. Reporting of Study Participant Demographic Characteristics and Demographic Representation in Premarketing and Postmarketing Studies of Novel Cancer Therapeutics. JAMA Network Open 2021, 4: e217063. PMID: 33877309, PMCID: PMC8058642, DOI: 10.1001/jamanetworkopen.2021.7063.Peer-Reviewed Original ResearchConceptsNovel cancer therapeuticsPostmarketing studyBlack patientsCancer therapeuticsDemographic dataOlder adultsUS cancer populationUS Cancer StatisticsCross-sectional studyRace/ethnicityParticipants' demographic characteristicsPercentage of trialsPatient sexCancer populationClinical studiesCancer statisticsMAIN OUTCOMEDrug AdministrationFDA approvalStudy participantsCancer typesDemographic characteristicsDemographic informationStudy samplePatients
2020
Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study
Hwang TJ, Ross JS, Vokinger KN, Kesselheim AS. Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study. The BMJ 2020, 371: m3434. PMID: 33028575, PMCID: PMC7537471, DOI: 10.1136/bmj.m3434.Peer-Reviewed Original ResearchConceptsEuropean Medicines AgencyRetrospective cohort studyTherapeutic valueHigh therapeutic valueNew drugsCohort studyLow therapeutic valueEMA approvalUS FoodDrug AdministrationMedicines AgencyFDA approvalDrugsFDARegulatory approvalApproval programsNew medicinesAssociationGreater proportionApprovalProportionAdministrationAssessment 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 ResearchConceptsPivotal 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
2019
Sharing of clinical trial data and results reporting practices among large pharmaceutical companies: cross sectional descriptive study and pilot of a tool to improve company practices
Miller J, Ross JS, Wilenzick M, Mello MM. Sharing of clinical trial data and results reporting practices among large pharmaceutical companies: cross sectional descriptive study and pilot of a tool to improve company practices. The BMJ 2019, 366: l4217. PMID: 31292127, PMCID: PMC6614834, DOI: 10.1136/bmj.l4217.Peer-Reviewed Original ResearchConceptsClinical trial dataTrial dataDrug approvalSectional descriptive studyPharmaceutical companiesFDA drug approvalsLarge pharmaceutical companiesClinical trialsDrug levelsNew drug approvalsUS FoodDrug AdministrationNew drug applicationsFDA approvalPatientsNovel drugsDescriptive studyDrug applicationTrialsDrugsExternal investigatorsSharing scoreDrug companiesTrial transparencyStudy measuresPostmarketing commitments for novel drugs and biologics approved by the US Food and Drug Administration: a cross-sectional analysis
Wallach JD, Luxkaranayagam AT, Dhruva SS, Miller JE, Ross JS. Postmarketing commitments for novel drugs and biologics approved by the US Food and Drug Administration: a cross-sectional analysis. BMC Medicine 2019, 17: 117. PMID: 31203816, PMCID: PMC6572730, DOI: 10.1186/s12916-019-1344-3.Peer-Reviewed Original ResearchConceptsNew clinical trialsClinical trialsPostmarketing commitmentFDA approvalPeer-reviewed journalsNovel drugsPharmaceutical companiesCross-sectional analysisRate of completionRates of registrationClinical studiesPublic reporting requirementsFirst approvalTherapeutic evaluationUS FoodDrug AdministrationTrialsNew drugsFDA documentsBiologicsFDADrugsFDA recordsApprovalStatus
2018
Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval
Shaw DL, Dhruva SS, Ross JS. Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval. Journal Of Managed Care & Specialty Pharmacy 2018, 24: 10.18553/jmcp.2018.24.12.1230. PMID: 30479199, PMCID: PMC10397592, DOI: 10.18553/jmcp.2018.24.12.1230.Peer-Reviewed Original ResearchConceptsNovel therapeutic agentsPrescription drug plansOrphan drug statusFDA approvalYear of approvalTherapeutic agentsNovel therapeuticsPrior authorizationNovel therapiesPriority reviewStep therapyMedicare prescription drug plansDrug plansDrug coverageNational InstituteDrug statusPart D prescription drug benefitMedicaid ServicesDrug characteristicsTherapeutic areasCross-sectional studyDrug benefit coverageHealth insurance payerBlue Cross Blue Shield AssociationPrescription drug benefitRegistration, 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 drugsDrugsEvidence supporting FDA approval and CMS national coverage determinations for novel medical products, 2005 through 2016
Roginiel AC, Dhruva SS, Ross JS. Evidence supporting FDA approval and CMS national coverage determinations for novel medical products, 2005 through 2016. Medicine 2018, 97: e12715. PMID: 30290675, PMCID: PMC6200488, DOI: 10.1097/md.0000000000012715.Peer-Reviewed Original ResearchConceptsOriginal clinical trialsCMS national coverage determinationsNational Coverage DeterminationClinical trialsFDA approvalControl armFDA approval documentsQuality of trialsCross-sectional studySignificant differencesCoverage determinationMedian ageMedian timePivotal trialsQuantity of evidenceInclusion criteriaTrial participantsMedicare populationCMS coverageDrug AdministrationStudy sizeNovel therapeuticsMedicaid ServicesMarketing approvalTrialsSpeed of Adoption of Immune Checkpoint Inhibitors of Programmed Cell Death 1 Protein and Comparison of Patient Ages in Clinical Practice vs Pivotal Clinical Trials
O’Connor J, Fessele KL, Steiner J, Seidl-Rathkopf K, Carson KR, Nussbaum NC, Yin ES, Adelson KB, Presley CJ, Chiang AC, Ross JS, Abernethy AP, Gross CP. Speed of Adoption of Immune Checkpoint Inhibitors of Programmed Cell Death 1 Protein and Comparison of Patient Ages in Clinical Practice vs Pivotal Clinical Trials. JAMA Oncology 2018, 4: e180798-e180798. PMID: 29800974, PMCID: PMC6143052, DOI: 10.1001/jamaoncol.2018.0798.Peer-Reviewed Original ResearchMeSH KeywordsAgedAntibodies, Monoclonal, HumanizedCarcinoma, Non-Small-Cell LungCarcinoma, Renal CellClinical Trials as TopicFemaleFollow-Up StudiesHumansKidney NeoplasmsLung NeoplasmsMaleMiddle AgedNivolumabPractice Patterns, Physicians'PrognosisProgrammed Cell Death 1 ReceptorRetrospective StudiesConceptsNon-small cell lung cancerPivotal clinical trialsRenal cell carcinomaCell death 1 proteinEligible patientsImmune checkpoint inhibitorsDeath 1 proteinClinical trialsClinical practiceFDA approvalAgent treatmentCheckpoint inhibitorsAnti-PD-1 agentsFlatiron Health networkRetrospective cohort studyAge of patientsCell lung cancerClinical trial participantsCohort studyPatient ageYounger patientsMore patientsSuch patientsTrial evidenceCell carcinomaAvailability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs
Puthumana J, Miller JE, Kim J, Ross JS. Availability of Investigational Medicines Through the US Food and Drug Administration’s Expanded Access and Compassionate Use Programs. JAMA Network Open 2018, 1: e180283-e180283. PMID: 30646072, PMCID: PMC6324420, DOI: 10.1001/jamanetworkopen.2018.0283.Peer-Reviewed Original ResearchConceptsCompassionate use programInvestigational medicinesFDA approvalNew drug application submissionAccess programExpanded AccessUS FoodAvailable FDA documentsLife-threatening conditionCross-sectional studyEvidence of safetyProtection of patientsUse programClinical trialsMAIN OUTCOMEDrug AdministrationInfectious diseasesDrug accessClinical development periodsFDA documentsApplication submissionPatientsDiseaseApprovalMonthsPostmarket studies required by the US Food and Drug Administration for new drugs and biologics approved between 2009 and 2012: cross sectional analysis
Wallach JD, Egilman AC, Dhruva SS, McCarthy ME, Miller JE, Woloshin S, Schwartz LM, Ross JS. Postmarket studies required by the US Food and Drug Administration for new drugs and biologics approved between 2009 and 2012: cross sectional analysis. The BMJ 2018, 361: k2031. PMID: 29794072, PMCID: PMC5967364, DOI: 10.1136/bmj.k2031.Peer-Reviewed Original ResearchConceptsProspective cohort studyCohort studyClinical trialsPostmarketing requirementsTimeliness of registrationNew drugsUS FoodDrug AdministrationFDA approvalStudy designNumber of patientsSectional analysisPostmarket studiesFirst approvalRegistrySecondary analysisTrialsBiologicsDrugsComparator typeAdministrationApprovalFDAFDA's authorityComparison of treatment effect sizes from pivotal and postapproval trials of novel therapeutics approved by the FDA based on surrogate markers of disease: a meta-epidemiological study
Wallach JD, Ciani O, Pease AM, Gonsalves GS, Krumholz HM, Taylor RS, Ross JS. Comparison of treatment effect sizes from pivotal and postapproval trials of novel therapeutics approved by the FDA based on surrogate markers of disease: a meta-epidemiological study. BMC Medicine 2018, 16: 45. PMID: 29562926, PMCID: PMC5863466, DOI: 10.1186/s12916-018-1023-9.Peer-Reviewed Original ResearchConceptsPostapproval trialsPivotal trialsActual clinical effectSurrogate markerTrial endpointsLarge treatment effectsPrimary endpointNovel therapeuticsNovel drugsTreatment effectsFDA approvalPatient-relevant outcomesMeta-epidemiological studyStandardized mean differenceTreatment effect sizeClinical effectsResultsBetween 2005Odds ratioDrug trialsSame indicationDrug AdministrationEvidence of differencesMean differenceU.S. FoodDisease
2017
Measuring clinical trial transparency: an empirical analysis of newly approved drugs and large pharmaceutical companies
Miller JE, Wilenzick M, Ritcey N, Ross JS, Mello MM. Measuring clinical trial transparency: an empirical analysis of newly approved drugs and large pharmaceutical companies. BMJ Open 2017, 7: e017917. PMID: 29208616, PMCID: PMC5728266, DOI: 10.1136/bmjopen-2017-017917.Peer-Reviewed Original ResearchThe FDA Unapproved Drugs Initiative: An Observational Study of the Consequences for Drug Prices and Shortages in the United States.
Gupta R, Dhruva SS, Fox ER, Ross JS. The FDA Unapproved Drugs Initiative: An Observational Study of the Consequences for Drug Prices and Shortages in the United States. Journal Of Managed Care & Specialty Pharmacy 2017, 23: 1066-1076. PMID: 28944731, PMCID: PMC10397719, DOI: 10.18553/jmcp.2017.23.10.1066.Peer-Reviewed Original ResearchConceptsUnapproved Drugs InitiativeClinical evidenceAverage wholesale priceFDA approvalPrescription drugsNew clinical trial evidenceMedian shortage durationClinical trial evidenceNew clinical evidenceDrug shortagesNumber of drugsEvidence of safetyUtah Drug Information ServiceHealth-System PharmacistsAssociation 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