2024
Characterization of accelerated approval status, trial endpoints and results, and recommendations in guidelines for oncology drug treatments from the National Comprehensive Cancer Network: cross sectional study
Mooghali M, Mitchell A, Skydel J, Ross J, Wallach J, Ramachandran R. Characterization of accelerated approval status, trial endpoints and results, and recommendations in guidelines for oncology drug treatments from the National Comprehensive Cancer Network: cross sectional study. BMJ Medicine 2024, 3: e000802. PMID: 38596814, PMCID: PMC11002412, DOI: 10.1136/bmjmed-2023-000802.Peer-Reviewed Original ResearchNational Comprehensive Cancer NetworkNational Comprehensive Cancer Network guidelinesComprehensive Cancer NetworkFood and Drug AdministrationCancer NetworkUS Food and Drug AdministrationCategories of evidenceAccelerated approvalCross sectional studyDrug treatmentConfirmatory trialsClinical benefitTrial resultsOncological indicationsTraditional approvalGuideline recommendationsOutcome measuresSectional studyApproval statusFDA decisionRecommended alignmentOncologyUpdate recommendationsPostapproval trialsGuidelines
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 ResearchWant to make drugs, including insulin, affordable? End patent abuse.
Skydel J, Ramachandran R. Want to make drugs, including insulin, affordable? End patent abuse. The Baltimore Sun. 03/03/2023. https://www.baltimoresun.com/opinion/op-ed/bs-ed-op-0306-pharmaceutical-patent-abuse-20230303-i5m23qxza5dkfd2oji5fv3v4ru-story.htmlPublications for non-academic audiences
2022
Accelerated approval: unfulfilled promises for FDA’s expedited review program
Skydel J, Ross J, Ramachandran R. Accelerated approval: unfulfilled promises for FDA’s expedited review program. The Hill. 05/27/2022. https://thehill.com/blogs/congress-blog/3504591-accelerated-approval-unfulfilled-promises-for-fdas-expedited-review-program/Publications for non-academic audiencesAgreement of treatment effects from observational studies and randomized controlled trials evaluating hydroxychloroquine, lopinavir-ritonavir, or dexamethasone for covid-19: meta-epidemiological study
Moneer O, Daly G, Skydel JJ, Nyhan K, Lurie P, Ross JS, Wallach JD. Agreement of treatment effects from observational studies and randomized controlled trials evaluating hydroxychloroquine, lopinavir-ritonavir, or dexamethasone for covid-19: meta-epidemiological study. The BMJ 2022, 377: e069400. PMID: 35537738, PMCID: PMC9086409, DOI: 10.1136/bmj-2021-069400.Peer-Reviewed Original ResearchConceptsCOVID-19 treatmentObservational studyMeta-epidemiological studyEfficacy outcomesLopinavir-ritonavirLiving reviewTreatment effectsCOVID-19 treatment guidelinesSame interventionCOVID-19Continuous outcomesMeta-analyze evidenceTrial sequential analysisSummary treatment effectsDistribution of sexTreatment guidelinesIndividual RCTsSelection of studiesEvidence DatabasePlacebo comparatorClinical dataStudy demographicsRCTsDichotomous outcomesTherapeutic interventionsSpending 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
Feasibility 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 ResearchUS 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 approvalReporting 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 samplePatientsCharacteristics and Reporting of Number Needed to Treat, Number Needed to Harm, and Absolute Risk Reduction in Controlled Clinical Trials, 2001-2019
Elliott MH, Skydel JJ, Dhruva SS, Ross JS, Wallach JD. Characteristics and Reporting of Number Needed to Treat, Number Needed to Harm, and Absolute Risk Reduction in Controlled Clinical Trials, 2001-2019. JAMA Internal Medicine 2021, 181: 282-284. PMID: 33226398, PMCID: PMC7684521, DOI: 10.1001/jamainternmed.2020.4799.Peer-Reviewed Original ResearchPublishing at any cost: a cross-sectional study of the amount that medical researchers spend on open access publishing each year
Ellingson MK, Shi X, Skydel JJ, Nyhan K, Lehman R, Ross JS, Wallach JD. Publishing at any cost: a cross-sectional study of the amount that medical researchers spend on open access publishing each year. BMJ Open 2021, 11: e047107. PMID: 33526505, PMCID: PMC7852964, DOI: 10.1136/bmjopen-2020-047107.Peer-Reviewed Original Research
2019
Analysis of Postapproval Clinical Trials of Therapeutics Approved by the US Food and Drug Administration Without Clinical Postmarketing Requirements or Commitments
Skydel JJ, Luxkaranayagam AT, Dhruva SS, Ross JS, Wallach JD. Analysis of Postapproval Clinical Trials of Therapeutics Approved by the US Food and Drug Administration Without Clinical Postmarketing Requirements or Commitments. JAMA Network Open 2019, 2: e193410. PMID: 31074812, PMCID: PMC6512282, DOI: 10.1001/jamanetworkopen.2019.3410.Peer-Reviewed Original ResearchConceptsPostapproval clinical trialsClinical trialsPrimary completion datePostmarketing requirementsNovel therapeuticsDrug AdministrationUS FoodNew indicationsPharmaceutical companiesNew clinical studiesCross-sectional analysisClinical outcomesStudy design elementsPatient populationUnapproved indicationsEfficacy dataClinical studiesStudy completionMAIN OUTCOMEFirst approvalMost trialsTrialsFDATherapeutic approvalsTherapeutics