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
2017
Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review
Pease AM, Krumholz HM, Downing NS, Aminawung JA, Shah ND, Ross JS. Postapproval studies of drugs initially approved by the FDA on the basis of limited evidence: systematic review. The BMJ 2017, 357: j1680. PMID: 28468750, PMCID: PMC5421452, DOI: 10.1136/bmj.j1680.Peer-Reviewed Original ResearchConceptsSingle pivotal trialPivotal trialsPostapproval studiesSurrogate markerPrimary endpointNovel drugsClinical outcomesClinical studiesLimited evidenceSystematic reviewDouble-blind studyMedian total numberClinical evidenceSuperior efficacyBlind studyDrug AdministrationOriginal FDATrial approvalDiseaseTrialsDrugsFDAEndpointEfficacyMarkers