2024
Premarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies
Mooghali M, Wallach J, Ross J, Ramachandran R. Premarket Pivotal Trial End Points and Postmarketing Requirements for FDA Breakthrough Therapies. JAMA Network Open 2024, 7: e2430486. PMID: 39190303, PMCID: PMC11350476, DOI: 10.1001/jamanetworkopen.2024.30486.Peer-Reviewed Original ResearchConceptsFood and Drug Administration tableFood and Drug AdministrationSurrogate end pointsSurrogate markerPostmarketing studiesEnd pointsTraditional approvalCross-sectional studyClinical benefitAccelerated approvalTherapy designClinically significant end pointsReview of therapeuticsPrimary end pointUS Food and Drug AdministrationTrial end pointsSignificant end pointsPostmarketing requirementsPreliminary clinical evidenceApproval pathwayPivotal trialsClinical evidenceBreakthrough therapiesDrug AdministrationTherapyPremarket Evidence and Postmarketing Requirements for Real-Time Oncology Review Indication Approvals
Mooghali M, Mohammad A, Wallach J, Mitchell A, Ross J, Ramachandran R. Premarket Evidence and Postmarketing Requirements for Real-Time Oncology Review Indication Approvals. JAMA Network Open 2024, 7: e249233. PMID: 38691363, PMCID: PMC11063797, DOI: 10.1001/jamanetworkopen.2024.9233.Peer-Reviewed Original Research
2023
Pivotal Trial Demographic Representation and Clinical Development Times for Oncology Therapeutics
Wong A, Miller J, Mooghali M, Ramachandran R, Ross J, Wallach J. Pivotal Trial Demographic Representation and Clinical Development Times for Oncology Therapeutics. JAMA 2023, 330: 2392-2394. PMID: 38079163, PMCID: PMC10714278, DOI: 10.1001/jama.2023.21958.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 ResearchApproval of Drugs With Unmet Trial End Points—In Reply
Johnston J, Ross J, Ramachandran R. Approval of Drugs With Unmet Trial End Points—In Reply. JAMA Internal Medicine 2023, 183: 889-890. PMID: 37307009, DOI: 10.1001/jamainternmed.2023.2240.Peer-Reviewed Original ResearchTherapeutic 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 subsetProportionPhysiciansExtending the US Food and Drug Administration’s Postmarket Authorities
Lynch H, Sachs R, Lee S, Herder M, Ross J, Ramachandran R. Extending the US Food and Drug Administration’s Postmarket Authorities. JAMA Health Forum 2023, 4: e231313. PMID: 37294583, DOI: 10.1001/jamahealthforum.2023.1313.Commentaries, Editorials and LettersUS Food and Drug Administration Review Time of Supplemental New Indication Approvals of Drugs and Biologics, 2017 to 2019
Dhodapkar M, Ross J, Ramachandran R. US Food and Drug Administration Review Time of Supplemental New Indication Approvals of Drugs and Biologics, 2017 to 2019. JAMA Network Open 2023, 6: e2318889. PMID: 37358857, PMCID: PMC10293905, DOI: 10.1001/jamanetworkopen.2023.18889.Peer-Reviewed Original ResearchUS 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 Administration
2022
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
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 resultsApprovals and Timing of New Formulations of Novel Drugs Approved by the US Food and Drug Administration Between 1995 and 2010 and Followed Through 2021
Gupta R, Morten CJ, Zhu AY, Ramachandran R, Shah ND, Ross JS. Approvals and Timing of New Formulations of Novel Drugs Approved by the US Food and Drug Administration Between 1995 and 2010 and Followed Through 2021. JAMA Health Forum 2022, 3: e221096. PMID: 35977259, PMCID: PMC9123500, DOI: 10.1001/jamahealthforum.2022.1096.Peer-Reviewed Original ResearchConceptsCross-sectional studyNovel drugsTherapeutic valueUS FoodDrug AdministrationWorld Health Organization Model ListGeneric approvalCapsule formMultivariable analysisAccelerated approvalClinical usefulnessMAIN OUTCOMEPrescription drugsBrand-name drugsModel ListEssential medicinesDrugsApproval statusMedicine inclusionFDANovel tabletBlockbuster statusApprovalAdministrationDrug sales
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 ResearchMeSH KeywordsDrug ApprovalHumansPeer ReviewPubMedResearch ReportUnited StatesUnited States Food and Drug AdministrationConceptsHigh-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 ResearchComparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018
Wallach JD, Ramachandran R, Bruckner T, Ross JS. Comparison of Duration of Postapproval vs Pivotal Trials for Therapeutic Agents Granted US Food and Drug Administration Accelerated Approval, 2009-2018. JAMA Network Open 2021, 4: e2133601. PMID: 34751764, PMCID: PMC8579231, DOI: 10.1001/jamanetworkopen.2021.33601.Peer-Reviewed Original ResearchTransparency of Regulatory Data across the European Medicines Agency, Health Canada, and US Food and Drug Administration.
Egilman AC, Kapczynski A, McCarthy ME, Luxkaranayagam AT, Morten CJ, Herder M, Wallach JD, Ross JS. Transparency of Regulatory Data across the European Medicines Agency, Health Canada, and US Food and Drug Administration. The Journal Of Law, Medicine & Ethics 2021, 49: 456-485. PMID: 34665102, DOI: 10.1017/jme.2021.67.Peer-Reviewed Original ResearchEarly experience with the FDA’s regulatory review of novel gene therapies
Puthumana J, Egilman AC, Ramachandran R, Naushad N, Shah N, Ross J. Early experience with the FDA’s regulatory review of novel gene therapies. BMJ Evidence-Based Medicine 2021, 27: 195-198. PMID: 34635478, DOI: 10.1136/bmjebm-2021-111720.Peer-Reviewed Original ResearchClinical studies sponsored by digital health companies participating in the FDA’s Precertification Pilot Program: A cross-sectional analysis
Bartlett VL, Dhruva SS, Shah ND, Ross JS. Clinical studies sponsored by digital health companies participating in the FDA’s Precertification Pilot Program: A cross-sectional analysis. Clinical Trials 2021, 19: 119-122. PMID: 34632824, PMCID: PMC9888320, DOI: 10.1177/17407745211048493.Peer-Reviewed Original ResearchFulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway
Gyawali B, Ross JS, Kesselheim AS. Fulfilling the Mandate of the US Food and Drug Administration’s Accelerated Approval Pathway. JAMA Internal Medicine 2021, 181: 1275-1276. PMID: 34254981, DOI: 10.1001/jamainternmed.2021.4604.Commentaries, Editorials and LettersImplementation of 21st Century Cures Act Expanded Access Policies Requirements
Kang S, Chang S, Ross JS, Miller JE. Implementation of 21st Century Cures Act Expanded Access Policies Requirements. Clinical Pharmacology & Therapeutics 2021, 110: 1579-1584. PMID: 34431083, DOI: 10.1002/cpt.2401.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 Research