Introduction/Aims Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States–approved treatment for patients with DMD and exon 51 skip‐amenable mutations. Previous analyses have shown that eteplirsen is associated with a statistically significant attenuation of pulmonary decline. In this study we evaluate the effect of eteplirsen treatment from newly available data sources on pulmonary function over time in patients with DMD. Methods We used a post hoc pooled analysis to compare the percentage of predicted forced vital capacity (FVC%p) and projected time with pulmonary function milestones in patients with DMD and exon 51 skip‐amenable mutations receiving eteplirsen (Studies 204 and 301) or standard of care (SoC; Cooperative International Neuromuscular Research Group Duchenne Natural History Study). A mixed model for repeated‐measures framework was applied to evaluate the impact of eteplirsen. Results An average annual rate of FVC%p decline for eteplirsen‐treated patients was estimated to be 3.47%, a statistically significant attenuation from the 5.95% rate of decline estimated in SoC patients (P = .0001). Using linear extrapolations of the model‐estimated decline in FVC%p, the attenuation in FVC%p decline for eteplirsen‐treated patients corresponded to a delay of 5.72 years in time to needing continuous ventilation, 3.31 years in time to needing nighttime ventilation, and 2.11 years in time to needing a cough assist device compared with SoC patients. Discussion The attenuation of FVC%p decline suggests that eteplirsen‐treated patients had statistically significant and clinically meaningful attenuations in pulmonary decline compared with SoC patients.

There is limited data on the cost-effectiveness of continuous-flow left ventricular assist devices (LVAD) in the United States particularly for the bridge-to-transplant indication. Our objective is to study the cost-effectiveness of a small intrapericardial centrifugal LVAD compared with medical management (MM) and subsequent heart transplantation using the respective clinical trial data. We developed a Markov economic framework. Clinical inputs for the LVAD arm were based on prospective trials employing the HeartWare centrifugal-flow ventricular assist device system. To better assess survival in the MM arm, and in the absence of contemporary trials randomizing patients to LVAD and MM, estimates from the Seattle Heart Failure Model were used. Costs inputs were calculated based on Medicare claim analyses and when appropriate prior published literature. Time horizon was lifetime. Costs and benefits were appropriately discounted at 3% per year. The deterministic cost-effectiveness analyses resulted in $69,768 per Quality Adjusted Life Year and $56,538 per Life Year for the bridge-to-transplant indication and $102,587 per Quality Adjusted Life Year and $87,327 per Life Year for destination therapy. These outcomes signify a substantial improvement compared with prior studies and re-open the discussion around the cost-effectiveness of LVADs.

Any opinions expressed in this paper are those of the author(s) and not those of IZA. Research published in this series may include views on policy, but IZA takes no institutional policy positions. The IZA research network is committed to the IZA Guiding Principles of Research Integrity. The IZA Institute of Labor Economics is an independent economic research institute that conducts research in labor economics and offers evidence-based policy advice on labor market issues. Supported by the Deutsche Post Foundation, IZA runs the world's largest network of economists, whose research aims to provide answers to the global labor market challenges of our time. Our key objective is to build bridges between academic research, policymakers and society. IZA Discussion Papers often represent preliminary work and are circulated to encourage discussion. Citation of such a paper should account for its provisional character. A revised version may be available directly from the author.

We exploit a highly competitive environment in which elite‐female athletes are exposed to the presence of men, but without being in direct competition with them. Specifically, we use variation in how fast the fastest man runs in the New York City Marathon to identify the potential influence of men on female performance while holding constant female‐runners' marginal incentives to perform. Our results suggest that as the men overtake the female runners, the performance of female runners declines differentially across ability, with the largest declines concentrated among lower ability runners.