2023
Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States
Ito S, Chetlapalli K, Potnis K, Richmond R, Wang D, Lee A, Goshua G. Setting Cost-Effective Price Thresholds before FDA Approval: Cost-Effectiveness of Iptacopan Monotherapy Versus Standard-of-Care Anti-C5 Therapy in Transfusion-Dependent, Treatment-Experienced Adult Patients with Paroxysmal Nocturnal Hemoglobinuria in the United States. Blood 2023, 142: 5042. DOI: 10.1182/blood-2023-188063.Peer-Reviewed Original ResearchTreatment-experienced patientsParoxysmal nocturnal hemoglobinuriaIncremental net monetary benefitIncremental cost-effectiveness ratioNursing timeExtravascular hemolysisNocturnal hemoglobinuriaPhase III study resultsTreatment-experienced adult patientsTransfusion-associated adverse eventsCost-effective therapeutic optionAnti-C5 therapyComplement inhibition therapyComplement inhibitor therapyHealth resource utilizationCare of patientsIndirect medical costsComplement component 5Clinical trial dataLife-threatening hematological disorderCost-effectiveness ratioBreakthrough therapy designationProbabilistic sensitivity analysesFirst cost-effectiveness analysisCost-effectiveness analysisCost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States
Ito S, Wang D, Purcell A, Chetlapalli K, Lee A, Cuker A, Goshua G. Cost-Effectiveness of Sutimlimab Versus Standard-of-Care in Transfusion Dependent Patients with Primary Cold Agglutinin Disease in the United States. Blood 2023, 142: 2316. DOI: 10.1182/blood-2023-187270.Peer-Reviewed Original ResearchPrimary cold agglutinin diseaseCold agglutinin diseaseIncremental cost-effectiveness ratioTransfusion-dependent patientsIncremental net monetary benefitProbabilistic sensitivity analysesDistributional cost-effectiveness analysisAnemia severityBody weightPhase 3 clinical studiesHealth resource utilizationRed blood cell destructionDisease-specific mortalityHistory of transfusionAutoimmune hemolytic anemiaMarkov state transition modelCare of patientsHumanized monoclonal antibodyLower body weightBlood cell destructionCost-effectiveness ratioFDA package insertsCold-reactive antibodiesBase-case analysisCost-effectiveness analysis
2021
Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Goshua G, Sinha P, Hendrickson J, Tormey C, Bendapudi PK, Lee AI. Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood 2021, 137: 969-976. PMID: 33280030, PMCID: PMC7918179, DOI: 10.1182/blood.2020006052.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAgedClinical Trials, Phase II as TopicClinical Trials, Phase III as TopicCombined Modality TherapyCost-Benefit AnalysisDecision TreesDrug CostsDrug Therapy, CombinationFemaleFibrinolytic AgentsHemorrhageHumansImmunosuppressive AgentsLength of StayMaleMarkov ChainsMiddle AgedModels, EconomicMulticenter Studies as TopicPlasma ExchangePurpura, Thrombotic ThrombocytopenicRecurrenceRituximabSingle-Domain AntibodiesStandard of CareUnited StatesYoung AdultConceptsIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraTherapeutic plasma exchangeVon Willebrand factorRelapse rateThrombocytopenic purpuraClinical trialsMajor randomized clinical trialsThrombotic microangiopathy leadingEnd-organ damageWillebrand factorPlatelet count recoveryRandomized clinical trialsHealth system costsOne-way sensitivity analysesCost-effectiveness ratioLife-threatening diseaseProbabilistic sensitivity analysesCost-effectiveness analysisHospital lengthCount recoveryPlasma exchangeTPE treatmentTTP patientsImmunomodulatory agents
2020
Cost Effectiveness of Caplacizumab in Acquired Thrombotic Thrombocytopenic Purpura
Goshua G, Sinha P, Hendrickson J, Tormey C, Bendapudi P, Lee A. Cost Effectiveness of Caplacizumab in Acquired Thrombotic Thrombocytopenic Purpura. Blood 2020, 136: 18-19. DOI: 10.1182/blood-2020-138515.Peer-Reviewed Original ResearchIncremental cost-effectiveness ratioThrombotic thrombocytopenic purpuraQuality-adjusted life yearsIntensive care unitTherapeutic plasma exchangeHERCULES trialCost-effectiveness analysisCaplacizumab treatmentRituximab useTITAN trialHospital lengthTPE sessionsThrombocytopenic purpuraTreatment armsAcquired Thrombotic Thrombocytopenic PurpuraLong-term disease courseThrombotic microangiopathy leadingEnd-organ damagePlatelet count recoveryLong-term followHealth system perspectiveOne-way sensitivity analysesPotential confounding factorsCost-effectiveness ratioLife-threatening disease