2023
Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017–2019
Poddar A, Raggio M, Concato J. Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017–2019. Therapeutic Innovation & Regulatory Science 2023, 58: 214-221. PMID: 37926768, PMCID: PMC10764372, DOI: 10.1007/s43441-023-00589-z.Peer-Reviewed Original ResearchConceptsBreakthrough therapy statusTherapy statusInsufficient treatment effectNon-oncology drugsReview of requestsUnmet medical needInadequate study designBreakthrough therapy designationConclusionsThis analysisCommon reasonInvestigational drugsUS FoodMedical needOrphan diseaseStudy designDrug evaluationMarketing approvalCorresponding drugsDrugsApproval statusTreatment effectsDrug developmentFDA processDesignation programStatus
2000
Randomized, Controlled Trials, Observational Studies, and the Hierarchy of Research Designs
Concato J, Shah N, Horwitz R. Randomized, Controlled Trials, Observational Studies, and the Hierarchy of Research Designs. New England Journal Of Medicine 2000, 342: 1887-1892. PMID: 10861325, PMCID: PMC1557642, DOI: 10.1056/nejm200006223422507.Peer-Reviewed Original ResearchConceptsCase-control studyObservational studyBacille Calmette-Guérin (BCG) vaccineCalmette-Guérin vaccineRandomized clinical trialsClinical topicsIndividual observational studiesEffect of vaccinationEffect of treatmentMEDLINE data baseActive tuberculosisControlled TrialsPercent confidence intervalsClinical trialsOdds ratioRelative riskSummary estimatesMajor medical journalsSame interventionTrialsResearch designConfidence intervalsHigh gradeTreatment effectsOriginal report