Jeffrey S. Stonebraker

A study of variations in the reported haemophilia A prevalence around the world

Summary.  The objectives of this paper were to study the reported haemophilia A prevalence (per 100 000 males) on a country‐by‐country basis and address the following: Does the reported prevalence of haemophilia A vary by national economies? We collected prevalence data for 106 countries from the World Federation of Hemophilia (WFH) annual global surveys and the literature. We found that the reported haemophilia A prevalence varied considerably among countries, even among the wealthiest of countries. The prevalence (per 100 000 males) for high income countries was 12.8 ± 6.0 (mean ± SD) whereas it was 6.6 ± 4.8 for the rest of the world. Within a country, there was a strong trend of increasing prevalence over time – the prevalence for Canada ranged from 10.2 in 1989 to 14.2 in 2008 (R = 0.94 and P < 0.001) and for the United Kingdom it ranged from 9.3 in 1974 to 21.6 in 2006 (R = 0.94 and P < 0.001). Prevalence data reported from the WFH compared well with prevalence data from the literature. Patient registries generally provided the highest quality of prevalence data. The lack of accurate country‐specific prevalence data has constrained planning efforts for the treatment and care of people with haemophilia A. With improved information, healthcare agencies can assess budgetary needs to develop better diagnostic and treatment facilities for affected patients and families and work to ensure adequate supplies of factor VIII concentrates for treatment. In addition, this information can help manufacturers plan the production of concentrates and prevent future shortages.

A STUDY OF REPORTED FACTOR IX USE AROUND THE WORLD

Summary.  Replacement therapy has significantly improved the life expectancy and lifestyle of people with haemophilia. The objectives of this article were to study the reported factor IX (FIX) use on a country‐by‐country basis and address the following question: Does the reported FIX use vary by national economies? We obtained data on the reported number of international units (IUs) of FIX used for 90 countries from the Marketing Research Bureau and the World Federation of Hemophilia. Results show that the reported FIX use varies considerably across national economies, even among the wealthiest of countries.Trends suggest that the reported FIX usage increases with increasing economic capacity and has been increasing over time. Trends also suggest that consumption of FIX has been increasing at a greater rate in high income countries. Given these trends, there will likely be an overall increase in the amount of FIX concentrates used in the treatment of haemophilia B. We also found that FIX use both in terms of IUs per capita and IUs per person provide a complete picture of the level of haemophilia care within a country. Such information is critical for planning efforts of national healthcare agencies to determine realistic budget priorities and pharmaceutical manufacturers to determine adequate production levels of FIX concentrates. By improving the data collection and surveillance of FIX use for the treatment of people with haemophilia B, we can identify trends and needs of patients and highlight best treatment practices among countries.

A study of variations in the reported haemophilia B prevalence around the world: A STUDY OF REPORTED HAEMOPHILIA B PREVALENCE

Summary.  The objectives of this article were to study the reported prevalence of haemophilia B (HB) on a country‐by‐country basis and to analyse whether the prevalence of HB varied by national economy. The prevalence of HB is the proportion of diagnosed, reported cases of HB in a population at a specific point of time. We collected data on the HB prevalence for 105 countries from the World Federation of Hemophilia annual global surveys. Our results showed that the HB prevalence varied considerably among countries, even among the wealthiest of countries. The HB prevalence (per 100 000 males) for the highest income countries was 2.69 ± 1.61 (mean ± SD), whereas the prevalence for the rest of the world was 1.20 ± 1.33 (mean ± SD). Ireland had the highest reported HB prevalence of 8.07 per 100 000 males. There was a strong trend of increasing HB prevalence (per 100 000 males) over time. Prevalence data reported from the WFH compared well with prevalence data from the literature. The WFH annual global surveys have some limitations, but they are the best available source of worldwide haemophilia data. Prevalence data are extremely valuable information for the planning efforts of national healthcare agencies in setting priorities and allocating resources for the treatment of HB.

Formulating and solving sequential decision analysis models with continuous variables

This paper presents a new decision analysis approach for modeling decision problems with continuous decision and/or random variables, and applies the approach to a research and development (R&D) planning problem. The approach allows for compact, natural formulation for classes of decision problems that are less appropriately addressed with standard discrete-variable decision analysis methods. Thus it provides a useful alternative analysis approach for problems that are often addressed in practice using simulation risk analysis methods. An illustrative application is presented to energy system R&D planning. The continuous-variable version of this model more directly represents the structure of the decision than a discrete approximation, and the resulting model can be efficiently solved using standard nonlinear optimization methods.

Measuring the quality of haemophilia care across different settings : a set of performance indicators derived from demographics data

Haemophilia is a rare disease for which quality of care varies around the world. We propose data‐driven indicators as surrogate measures for the provision of haemophilia care across countries and over time.

Practice Summaries: American Airlines Uses Should-Cost Modeling to Assess the Uncertainty of Bids for Its Full-Truckload Shipment Routes

We used decision analysis to develop a probabilistic model to help American Airlines assess the uncertainty of bid quotes for its full-truckload (FTL) point-to-point freight shipments of maintenance equipment and in-flight service items in the United States. The model reduced the airlines risk of overpaying an FTL supplier.

Product-Generation Transition Decision Making for Bayer's Hemophilia Drugs: Global Capacity Expansion Under Uncertainty with Supply-Demand Imbalances

We present an application of decision analysis to global production capacity expansion under uncertainty for Bayer Groups proposed new biotechnology drug to treat hemophilia A. This decision analysis developed an improved approach to Bayers decision for product-generation transition and global production capacity expansion that more realistically addresses potential regional supply shortages and overages due to demand and supply uncertainties that can result in supply-demand imbalances. With the added confidence provided by this more realistic approach, Bayers executive leadership team acted on the recommendation from the decision analysis in contrast to an earlier analysis that had not resulted in management action. The paper makes two major contributions: First, it describes the details of what is involved in conducting applied decision analysis in a real-world setting in more detail than what is typically in textbooks on decision analysis. Second, the paper illustrates the important role of economic...

Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire

Objective To assess the psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire. Methods This study was a cross-sectional, multinational study. Participants were enrolled if they were more than 10 years old and people with haemophilia A or B or people without a bleeding disorder. Participants were invited through non-governmental patient organisations in 21 countries between 01/27/2016 and 02/23/2017. The following psychometric properties: missing data, floor and ceiling effects, exploratory factor analysis and internal consistency reliability were examined. A PROBE Score was derived and assessed for its convergent and known groups validity. Results The study analysed the data on 916 participants with median age of 37.0 (IQR 27.0 to 48.0) years, 74.8% male. In the domain assessing patient-reported outcomes (PROs), more than 15% of participants presented a ceiling effect for all items but two, and a floor effect for one item. Factor analysis identified three factors explaining the majority of the variance. Cronbach’s alpha coefficient indicated good internal consistency reliability (0.84). PROBE items showed moderate to strong correlations with corresponding EuroQol five dimension 5-level instrument (EQ-5D-5L) domains. The PROBE Score has a strong correlation (r=0.67) with EQ-5D-5L utility index score. The PROBE Score has a known groups validity among various groups. Conclusions The results of this study suggest that PROBE is a valid questionnaire for evaluating PROs in people with haemophilia as well as control population. The known-group property of PROBE will allow its use in future clinical trials, longitudinal studies, health technology assessment studies, routine clinical care or registries. Additional studies are needed to test responsiveness and sensitivity to change. Trial registration number NCT02439710; Results.

Empirically Investigating the Portfolio Management Process: Findings from a Large Pharmaceutical Company

This exploratory study analyzes cross-sectional project data from the enterprise information system at a large pharmaceutical company in order to gain insight into the company’s portfolio decision process and determinants of the ways in which decision analytic tools were applied in practice. Statistical measures based on the economic parameters describe individual projects and various partitions of the company’s portfolio of projects. Other statistics, such as number of scenarios, describe aspects of the structured decision process. The study found significant differences across project groups, and results in suggesting reasons for these differences. More generally, obtainable empirical data proved to be useful for studying the patterns in the use of portfolio decision analysis.

Advances in Decision Analysis: Balancing Environmental and Operational Objectives in Nuclear Refueling Strategies

The New York Power Authority (NYPA) wanted to develop a 10-year schedule for refueling its Indian Point 3 Nuclear Power Plant (IP3) that balanced fish protection, which occurs when IP3 is shutdown for nuclear refueling, and the costs of buying and loading fuel. We developed a decision analysis model to compare alternative strategies for refueling. It explicitly considered key uncertainties associated with future operation: how well IP3 operates, how long it takes to refuel, and when New York State is likely to deregulate the electric utility industry. The NYPA decision-makers used the model to reinforce their choice of a refueling strategy. They were not surprised that more fish protection occurred with strategies that restricted the starting date for refueling to the last full week in May, rather than allowing the starting date to float throughout the period from May through August. However, the NYPA decision-makers were surprised that the more restrictive strategies also resulted in lower costs. Advances: Nuclear Refueling Page 2 of 32 Ch 27 060520 V06 CONTENTS Problem Structuring – Designing the Project for Success Mission Statement Decision Pyramid Objectives Hierarchy Objective—Minimize Total Cost Objective—Maximize Fish Protection Strategies Uncertainties Modeling – Linking Choices to Outcomes Data Collection – Developing Reliable Inputs Evaluation – Performing Analyses to Generate New Insights Stakeholder Reaction Reflections Advances: Nuclear Refueling Page 3 of 32 Ch 27 060520 V06 The New York Power Authority (NYPA) is the nation’s largest nonfederal public power organization, providing about one-fourth of the electricity used in New York State. The NYPA owns 12 power projects. The Indian Point 3 Nuclear Power Plant (IP3), located on the Hudson River, generates approximately one-fifth of its electrical power. Since 1975, IP3 has saved electric power users in Westchester County and New York City more than