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
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 results
2021
Comparison 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 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 LettersConceptsAccelerated approval pathwayUS FoodPostapproval trialsApproval pathwayDrug AdministrationPathwayAdministration
2018
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
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