Study evaluates the conventional and distributional cost-effectiveness of prophylaxis with efanesoctocog alfa versus standard-care factor VIII (standard half-life and extended half-life) for patients living with severe hemophilia A in the United States. Read more at

Excited to see this article with @GeorgeGoshuaMD out in @BulletinAtomic, where we explore how adding a health equity lens to valuation can help harness the potential of gene therapies around the world @YaleMed @YaleIMed @TradIMYale @MarkDSiegel1

Thank you @YaleCancer -> our trainees = why we do this: 4 most recent (May & June 2024) first-author trainee 🌟s @YaleMed @YaleIMed @HopkinsHeme & @NIHCritCare @WaldronCJ @SatokoItoMDPhD #cecilyallen #danielwang @AyeshaButtMD @RhysRichmond @hmkyale @HannyAlSamkari @CukerMd

We are grateful for the support of @MGHCancerCenter @YaleMed @YaleIMed @YaleCancer @YaleHematology

14/This enormous benefit is mediated by reduction in the need for RBC and iron supplementation, hemostatic procedures, hospitalizations, and emergency department encounters, adding meaningful health benefits while saving patients valuable time

13/ In summary, we find longitudinal bevacizumab therapy added to the SOC is a cost-saving intervention that improves quality-adjusted life expectancy across a range of accepted WTP thresholds for patients with HHT with moderate-to-severe bleeding

12/ One-way sensitivity analysis revealed no parameter change which favored SOC alone over bevacizumab; in probabilistic sensitivity analysis bevacizumab was favored in 100% of 10,000 Monte Carlo iterations, across a range of WTP thresholds, with 99.6% being cost-saving

11/ Bevacizumab continues to be cost-saving when base-case and scenario analyses are conducted from a societal perspective, incorporating caregivers’ productivity loss to total costs

10/ Scenario analyses demonstrate that bevacizumab remains cost-saving across a range of IV iron formulations (carboxymaltose - base-case; ferumoxytol iNMB = $354,000; iron dextran iNMB = $316,000) and when the adverse events rate from RBC and iron supplementation is held to 0

9/ Longitudinal bevacizumab is also predicted to save patients an estimated 133 hours of time per year of life lived compared to SOC alone by reducing the need for RBC and iron supplementation, hemostatic surgeries, emergency room visits, and hospitalizations

8/ Aside: the net-monetary-benefit (NMB) is well-established summary measure of costs and health outcomes, found by deducting costs from the product of WTP and effectiveness ( https://t.co/csdrcNnpVH) with larger NMB indicating the more cost-effective alternative

7/ We found that, over a lifetime time horizon and at a willingness-to-pay (WTP) of $150,000/QALY, bevacizumab is not only cost-effective but cost-saving compared to SOC alone – saving costs while adding QALYs, and accruing an incremental net-monetary-benefit (iNMB) of $433,000

6/ To address this, we conducted the first cost-effectiveness analysis of longitudinal IV bevacizumab therapy added to SOC versus SOC alone, developing a Markov cohort model of patients with HHT with moderate-to-severe bleeding utilizing data from the InHIBIT-Beed clinical study

5/ Bevacizumab, a monoclonal anti-VEGF antibody, has shown great promise as a disease-modifying therapeutic targeting bleeding pathophysiology. However, the cost-effectiveness of this expensive biologic agent in moderate-to-severe HHT-associated bleeding is not known

4/ The current standard-of-care (SOC) – consisting of iron and red blood cell (RBC) supplementation and local hemostatic procedures (i.e. nasal and gastrointestinal) – help manage symptoms but do not target underlying disease pathogenesis

3/ As such HHT is associated with substantial morbidity, mortality, and significant decreases in both quality-of-life and life expectancy. Unlike hemophilia or von Willebrand disease, no FDA- or EMA-approved therapies exist for HHT-associated bleeding

2/ Hereditary hemorrhagic telangiectasia (HHT) is the second-most-common inherited bleeding disorder worldwide, affecting 1 in 5,000 persons, with recurrent mucosal bleeding and disease progression leading to iron deficiency anemia in most patients

🚨Fourth, in @BloodAdvances, a Yale-MGH collaboration @HannyAlSamkari: cost-effectiveness of bevacizumab therapy in the care of patients with hereditary hemorrhagic telangiectasia https://t.co/PtutUMt1R2

.@GeorgeGoshuaMD accepts the prestigious NOMIS and @ScienceMagazine Young Explorer Award in Zurich, Switzerland. Read more about Dr. Goshua and his winning essay "Napkin math can change the world." https://t.co/tRr3FkLtQl @SmilowCancer @YaleMed @YNHH @YaleHematology

Cost-effectiveness of prophylaxis with recombinant vs plasma-derived VWF in severe von Willebrand disease in the United States https://t.co/JAuuie1C4t #clinicaltrialsandobservations #thrombosisandhemostasis