Cost analysis of plasma-derived factor VIII/von Willebrand factor versus recombinant factor VIII for treatment of previously untreated patients with severe hemophilia A in the United States

Background: Inhibitor development to factor VIII (FVIII) hemophilia therapy results in increased complications and substantial economic costs. The SIPPET study, the first randomized controlled trial to compare the immunogenicity of plasma-derived FVIII (pdFVIII)/von Willebrand factor (VWF) and recombinant-DNA-derived FVIII (rFVIII), demonstrated higher inhibitor rates in previously untreated patients (PUPs) treated with rFVIII than in PUPs treated with pdFVIII/VWF.

Objective: To quantify the economic impact of treating PUPs with pdFVIII/VWF vs rFVIII.

Methods: An Excel-based clinical and economic model was developed from a US healthcare payer perspective and run over a 5-year period. The analysis utilized a cohort approach to model patient treatment and outcomes over a monthly cycle to quantify differences in costs of FVIII, bypassing agents, and hospitalizations for serious bleeds. Rates of high-titer inhibitor development were obtained from the SIPPET study. Patients developing high-titer inhibitors were treated with immune tolerance induction (ITI). Patients who developed low-titer inhibitors and those who did not develop inhibitors continued their usual FVIII treatment. Patients who were successfully treated with ITI returned to FVIII treatment, while unsuccessfully treated patients received bypassing agents. Total costs per treated patient were estimated and a one-way sensitivity analysis was conducted to quantify the impact of parameter uncertainty on the model outcomes.

Results: Total cumulative costs per patient over 5 years were $834,621 for pdFVIII/VWF patients and $1,237,163 for rFVIII patients, representing a total saving of $402,542 per patient over the 5-year period, for an average annual saving of $80,508 per patient.

Conclusions: Based on data from the SIPPET study, this analysis found that initiating FVIII treatment in severe hemophilia A PUPs with pdFVIII/VWF has the potential to offer substantial cost savings to healthcare payers, amounting to a one-third reduction in costs.     

Duality theory for robust utility maximisation

In this paper, we present a duality theory for the robust utility maximisation problem in continuous time for utility functions defined on the positive real line. Our results are inspired by – and can be seen as the robust analogues of – the seminal work of Kramkov and Schachermayer (Ann. Appl. Probab. 9:904–950, 1999). Namely, we show that if the set of attainable trading outcomes and the set of pricing measures satisfy a bipolar relation, then the utility maximisation problem is in duality with a conjugate problem. We further discuss the existence of optimal trading strategies. In particular, our general results include the case of logarithmic and power utility, and they apply to drift and volatility uncertainty.

     

Remote Leadership,Communication Effectiveness and Leader Performance

As remote work arrangements have gained in popularity, workforce dispersion has become increasingly widespread. Little research to date has examined how physical distance influences leader–follower communication effectiveness or leader performance. Building on top of transformational leadership theory, this paper explores how perceived leader performance is influenced by leadership style, physical distance, and communication effectiveness between leaders and followers. A survey of 138 followers, reporting to a total of 41 leaders, was conducted and data were analyzed at the individual follower-level using the Partial Least Squares (PLS) technique. Our model explained 45% of the variance in communication effectiveness and 67% of the variance in perceived leader performance. Consistent with past empirical findings, transformational leadership was associated more strongly with perceived leader performance than transactional contingent reward leadership. Communication effectiveness was also a strong predictor of leader performance, and furthermore acted as a mediator of leadership behavior on performance. Surprisingly, distance had no influence on either communication effectiveness or perceived leader performance. Implications for theory and practice are discussed.     

TO ADMIT OR NOT TO ADMIT: THE QUESTION OF RESEARCH PARK SIZE

A theoretical model is used to explore the determinants of the optimum size of a private research park and the effect of university affiliation on that optimum size. Parks are assumed to operate as cooperatives where costs are equally shared among the member firms, and optimality occurs when the firms’ average net benefits are maximized. To achieve this, existing members of a park will limit the park's size, denying entry to firms who wish to join and are willing to share the costs. University affiliation may either increase or decrease the optimum size of a park.     

Community of Practice Behaviors and Individual Learning Outcomes

The community of practice (CoP) concept has grown in popularity, yet remains under-studied. In particular, we have not developed a sufficient understanding of the individual outcomes associated with CoP engagement. This paper offers a fresh research model that identifies three practice-based concepts described in the CoP literature—shared repertoire, joint enterprise, and mutual engagement—and links them to individual learning outcomes. Survey measures are developed using a card sorting procedure, a research model is pilot tested using survey data collected from 53 graduate students in a large Canadian university, and then the model is field-tested using interview and survey data collected from 59 employees in a non-profit organization. The paper offers a new set of distinct CoP measures, and examines how they are associated with learning. A discussion of practical implications and future research directions is provided.     

James Delbourgo. A Most Amazing Scene of Wonders: Electricity and Enlightenment in Early America

James Delbourgo, chair of a program in the history and philosophyof science at McGill University, examines the disparate viewson electricity held in the eighteenth and early nineteenth centuriesby a broad variety of individuals including scientists, showmen,clergymen, physicians, and medical quacks. Delbourgo sometimessucceeds in connecting these views on electricity with othercultural and political ideas of the time. Not primarily aboutthe history of science, this book is more about cultural andpopular intellectual history. The     

Falling Water: The Origins of Direct Federal Participation in the US Electric Utility Industry, 1902–1933

The federal government's role as a direct producer in a non-defense, non-distressed American industry is unique to electric power. This participation arose during the first three decades of the twentieth century; it was politically controversial then and remains so today. We seek to explain how and why the federal government got into the electric utility business. The convergence of technological, economic, and political forces paved the way for federal participation. The growth of long-distance transmission networks and the success of the Progressive movement were important factors. Because so many dam sites with hydroelectric potential were on public lands in western states, and because hydroelectricity could help pay for those dams, even conservative Republicans like Herbert Hoover came to accept the involvement of the federal government. After the election of Franklin D. Roosevelt in 1932, the federal government accepted the task with enthusiasm as part of a plan for the economic development of entire river basins.     

Super‐replication in fully incomplete markets

In this work, we introduce the notion of fully incomplete markets. We prove that for these markets, the super‐replication price coincides with the model‐free super‐replication price. Namely, the knowledge of the model does not reduce the super‐replication price. We provide two families of fully incomplete models: stochastic volatility models and rough volatility models. Moreover, we give several computational examples. Our approach is purely probabilistic.