2023
Use of Expedited Regulatory Programs and Clinical Development Times for FDA-Approved Novel Therapeutics
Wong A, Mooghali M, Ramachandran R, Ross J, Wallach J. Use of Expedited Regulatory Programs and Clinical Development Times for FDA-Approved Novel Therapeutics. JAMA Network Open 2023, 6: e2331753. PMID: 37651145, PMCID: PMC10472182, DOI: 10.1001/jamanetworkopen.2023.31753.Peer-Reviewed Original Research
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
Postmarketing 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
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 drugsDrugsPostmarket 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 authority
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 ResearchAssociation 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 documentsDrugsDiabetesFDAAAReviewersAssessing the potential clinical impact of reciprocal drug approval legislation on access to novel therapeutics in the USA: a cohort study
Larochelle M, Downing NS, Ross JS, David FS. Assessing the potential clinical impact of reciprocal drug approval legislation on access to novel therapeutics in the USA: a cohort study. BMJ Open 2017, 7: e014582. PMID: 28179418, PMCID: PMC5306516, DOI: 10.1136/bmjopen-2016-014582.Peer-Reviewed Original ResearchConceptsEuropean Medicines AgencyCohort studyClinical impactRare diseaseHealth CanadaNew therapeuticsLikely clinical impactPotential clinical impactUS patientsAvailable therapiesNovel mechanismDrug AdministrationMedicines AgencyNovel therapeuticsUS approvalNew drugsDrugsFDA submissionsReview outcomesTherapeuticsSafety concernsDiseaseFDAMechanistic noveltyAffected population
2015
Clinical trial registration, reporting, publication and FDAAA compliance: a cross-sectional analysis and ranking of new drugs approved by the FDA in 2012
Miller JE, Korn D, Ross JS. Clinical trial registration, reporting, publication and FDAAA compliance: a cross-sectional analysis and ranking of new drugs approved by the FDA in 2012. BMJ Open 2015, 5: e009758. PMID: 26563214, PMCID: PMC4654354, DOI: 10.1136/bmjopen-2015-009758.Peer-Reviewed Original ResearchEfficacy And Safety Concerns Are Important Reasons Why The FDA Requires Multiple Reviews Before Approval Of New Drugs
Ross JS, Dzara K, Downing NS. Efficacy And Safety Concerns Are Important Reasons Why The FDA Requires Multiple Reviews Before Approval Of New Drugs. Health Affairs 2015, 34: 681-688. PMID: 25847652, DOI: 10.1377/hlthaff.2014.1160.Peer-Reviewed Original Research