@article{curtis_wu_iorio_frick_nichol_noone_o'mahony_page_stonebraker_kucher_et al._2024, title={Test-retest reliability of a mobile application of the patient reported outcomes burdens and experiences (PROBE) study}, ISSN={["1365-2516"]}, DOI={10.1111/hae.14969}, abstractNote={Abstract Introduction The Patient Reported Outcomes, Burdens, and Experiences (PROBE) questionnaire is a patient‐reported outcome tool that assesses quality of life and disease burden in people with haemophilia (PWH). Aim To assesses the test–retest reliability of PROBE when completed using the mobile phone application. Methods We recruited PWH, including carriers, and individuals with no bleeding disorders who attended haemophilia‐related workshops or via social media. Participants completed PROBE three times (twice on the app: T1 and T2, and once on the web, T3). Test–retest reliability was analysed for T1 versus T2 (app to app, time period one) and T2 versus T3 (app to web, time period two). Results We enrolled 48 participants (median age = 56 [range 27–78] years). Eighteen participants (37.5%) were PWH and seven (14.6%) were carriers. On general health domain questions, we found almost perfect agreement, except for a question on the frequency of use of pain medication in the last 12 months [Kappa coefficient ( κ ) .72 and .37 for time period one and two, respectively] and any use of pain medications ( κ .75) for time period two. For haemophilia‐related questions, we found substantial to perfect agreement, except for the questions on the number of joint bleeds in the previous 6 months for time period one ( κ .49) and the number of bleeds in the previous two weeks for time period two ( κ .34). Conclusions The results demonstrate the reliability of the PROBE app. The app can be used interchangeably with the paper and web platforms for PROBE administration.}, journal={HAEMOPHILIA}, author={Curtis, Randall and Wu, Joanne and Iorio, Alfonso and Frick, Neil and Nichol, Michael and Noone, Declan and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey and Kucher, Alexandra and et al.}, year={2024}, month={Mar} }
 @article{stonebraker_2023, title={Case Article—Bayer New Drug Development Decision Making}, url={https://doi.org/10.1287/ited.2021.0262ca}, DOI={10.1287/ited.2021.0262ca}, abstractNote={The interactive case study requires student teams to engage with the instructor using a structured decision analysis process in deciding whether to develop a new drug to treat blood clots in legs. There is role-playing in the interactive case study where student teams are decision consultants and the instructor serves as the decision maker, subject matter expert (SME), and coach. Student teams are responsible for managing the analytical process, framing the decision, collecting data from the SME (instructor), constructing the Excel model, assessing probabilities for the most-sensitive uncertainties from the SME, evaluating the Excel-based decision-tree model, and presenting evaluation results and recommendations to the decision maker (instructor). The goal of the case is to improve the analytical, modeling, and consulting skills of the students. The interactive case study is the culmination of a semester-long elective MBA course, entitled Decision Making Under Uncertainty. Since 2010, I have taught this course 31 times to 870 graduate students.}, journal={INFORMS Transactions on Education}, author={Stonebraker, Jeffrey S.}, year={2023}, month={May} }
 @article{stonebraker_2023, title={Case—Bayer New Drug Development Decision Making}, url={https://doi.org/10.1287/ited.2021.0262cs}, DOI={10.1287/ited.2021.0262cs}, abstractNote={History: This paper has been accepted for the INFORMS Transactions on Education Special Section on Cases Based on Real-World Projects from the INFORMS Journal on Applied Analytics.}, journal={INFORMS Transactions on Education}, author={Stonebraker, Jeffrey S.}, year={2023}, month={May} }
 @article{stonebraker_iorio_lavin_rezende_srivastava_pierce_coffin_tootoonchian_makris_2023, title={Reported prevalence of von Willebrand disease worldwide in relation to income classification}, volume={6}, ISSN={["1365-2516"]}, url={https://doi.org/10.1111/hae.14810}, DOI={10.1111/hae.14810}, abstractNote={The diagnosis of von Willebrand disease (VWD) is complex and challenging, especially when diagnostic resources are limited. This results in a lack of consistency in identifying and reporting the number of people with VWD and variations in the VWD prevalence worldwide.}, journal={HAEMOPHILIA}, author={Stonebraker, Jeffrey S. and Iorio, Alfonso and Lavin, Michelle and Rezende, Suely M. and Srivastava, Alok and Pierce, Glenn F. and Coffin, Donna and Tootoonchian, Ellia and Makris, Michael}, year={2023}, month={Jun} }
 @article{jamie m. m. o'sullivan_tootoonchian_ziemele_makris_federici_djambas_el ekiaby_rotellini_sidonio_iorio_et al._2023, title={Von Willebrand disease: Gaining a global perspective}, volume={5}, ISSN={["1365-2516"]}, DOI={10.1111/hae.14804}, abstractNote={Recent guidelines for von Willebrand Disease (VWD) highlighted the challenges in diagnosis and management. Identifying the number of persons with VWD (PwVWD) internationally will help target support to aid diagnosis of PwVWD.}, journal={HAEMOPHILIA}, author={Jamie M. M. O'Sullivan and Tootoonchian, Ellia and Ziemele, Baiba and Makris, Michael and Federici, Augusto B. B. and Djambas, Claudia Khayat and El Ekiaby, Magdy and Rotellini, Dawn and Sidonio, Robert F. F. and Iorio, Alfonso and et al.}, year={2023}, month={May} }
 @article{stonebraker_o'mahony_noone_iorio_2021, title={Converting factor and nonfactor usage into a single metric to facilitate benchmarking the resources consumed for haemophilia care across jurisdictions and over time}, volume={6}, ISSN={["1365-2516"]}, url={https://doi.org/10.1111/hae.14364}, DOI={10.1111/hae.14364}, abstractNote={The World Federation of Hemophilia started measuring factor utilization at the country level as IU/capita (International Units of factor concentrates used per country population) in 2001 for its Annual Global Survey. IU/capita have been used to benchmark a country's usage over time and for advocacy. The introduction of a common metric usage spanning across standard half‐life (SHL), and extended half‐life (EHL) clotting factor concentrates (CFCs) and emicizumab would be a valuable simplification for national healthcare policymaking and industrial production planning.}, journal={HAEMOPHILIA}, author={Stonebraker, Jeffrey S. and O'Mahony, Brian and Noone, Declan and Iorio, Alfonso}, year={2021}, month={Jun} }
 @article{germini_chai-adisaksopha_pete_curtis_frick_nichol_noone_o'mahony_page_stonebraker_et al._2021, title={Evaluation of the sexual health in people living with hemophilia}, volume={9}, ISSN={["1365-2516"]}, DOI={10.1111/hae.14410}, abstractNote={Knowledge about sexual health, difficulty with sexual activity and intimacy (sexual difficulty), in people with hemophilia is little understood.}, journal={HAEMOPHILIA}, author={Germini, Federico and Chai-Adisaksopha, Chatree and Pete, Drashti and Curtis, Randall and Frick, Neil and Nichol, Michael B. and Noone, Declan and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey S. and et al.}, year={2021}, month={Sep} }
 @article{stonebraker_ducore_2021, title={Modelling future usage and cost of factor and emicizumab to treat haemophilia A for the US Western States Region IX haemophilia treatment centres}, volume={27}, url={https://doi.org/10.1111/hae.14159}, DOI={10.1111/hae.14159}, abstractNote={Increased usage of emicizumab in the United States will affect standard half‐life (SHL) and extended half‐life (EHL) products usage and cost.}, number={1}, journal={Haemophilia}, publisher={Wiley}, author={Stonebraker, Jeffrey S. and Ducore, Jonathan M.}, year={2021}, month={Jan} }
 @article{chai‐adisaksopha_noone_curtis_frick_nichol_germini_o'mahony_page_stonebraker_skinner_et al._2021, title={Non‐severe haemophilia: Is it benign? – Insights from the PROBE study}, volume={9}, url={https://doi.org/10.1111/hae.14105}, DOI={10.1111/hae.14105}, abstractNote={There are limited data on the impact of haemophilia on health status and health‐related quality of life (HRQL) in people with non‐severe (mild and moderate) haemophilia.}, journal={Haemophilia}, publisher={Wiley}, author={Chai‐Adisaksopha, Chatree and Noone, Declan and Curtis, Randall and Frick, Neil and Nichol, Michael B. and Germini, Federico and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey S. and Skinner, Mark W. and et al.}, year={2021}, month={Jan} }
 @article{assis_suarez_prestemon_stonebraker_carrillo_dasmohapatra_jameel_gonzalez_2021, title={Risk Analysis, Practice, and Considerations in Capital Budgeting: Evidence from the Field for the Bio-based Industry}, volume={16}, ISSN={["1930-2126"]}, url={http://dx.doi.org/10.15376/biores.16.1.19-45}, DOI={10.15376/biores.16.1.19-45}, abstractNote={This study aims to examine how organizations in the bio-based industry perceive risks and perform risk analysis within the capital investment decision-making process. More specifically, this study aims to assess sources of uncertainty commonly considered, identify tools and methods used for risk assessment, and understand how risk analysis is considered in capital budgeting. Eighty-six respondents were electronically surveyed on practices for capital investment risk analysis, including C-suite and upper management from different organization sizes and segments in the bio-based industry. It was found that some forms of risk analysis are utilized either in project assessment and/or for decision making by most respondents; however, qualitative and deterministic assessment practices dominate over probabilistic methods. In addition, risk assessment is most commonly performed in the later stages of a project, with less than 50% of adoption at the earlier stages. Overall, the main sources of uncertainties considered when performing risk assessment are financial, market and sales, and technology, with competition being considered mostly by upper management levels. Additionally, consistent with previous studies in other industry sectors, Internal Rate of Return, Return on Investment, and Net Present Value are the preferred financial indicators used to evaluate capital investments.}, number={1}, journal={BIORESOURCES}, publisher={BioResources}, author={Assis, Camilla Abbati and Suarez, Antonio and Prestemon, Jeffrey P. and Stonebraker, Jeffrey and Carrillo, Carlos and Dasmohapatra, Sudipta and Jameel, Hasan and Gonzalez, Ronalds}, year={2021}, month={Feb}, pages={19–45} }
 @article{stonebraker_bolton‐maggs_brooker_evatt_iorio_makris_o'mahony_skinner_coffin_pierce_et al._2020, title={The World Federation of Hemophilia Annual Global Survey 1999‐2018}, url={https://doi.org/10.1111/hae.14012}, DOI={10.1111/hae.14012}, abstractNote={The World Federation of Hemophilia (WFH) strives to achieve care for all patients with inherited bleeding disorders through research, advocacy, capacity building and education. The WFH developed and implemented the Annual Global Survey (AGS), through which comprehensive demographic and treatment data on bleeding disorders are collected each year from its constituent non‐governmental national organizations.}, journal={Haemophilia}, author={Stonebraker, Jeffrey S. and Bolton‐Maggs, Paula H. B. and Brooker, Mark and Evatt, Bruce and Iorio, Alfonso and Makris, Michael and O'Mahony, Brian and Skinner, Mark W. and Coffin, Donna and Pierce, Glenn F. and et al.}, year={2020}, month={Jul} }
 @article{iorio_stonebraker_chambost_makris_coffin_herr_germini_stonebraker_iorio_byams_et al._2019, title={Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males A Meta-analytic Approach Using National Registries}, volume={171}, ISSN={["1539-3704"]}, DOI={10.7326/M19-1208}, abstractNote={Hemophilia is caused by a mutation in either the F8 (hemophilia A) or F9 (hemophilia B) gene and results in substantive morbidity and mortality if not treated. The severity of clinical manifestations in hemophilia is proportional to the amount of residual factor activity in the blood. In severe cases (clotting factor level <0.01 IU/mL), patients bleed spontaneously, and in moderate (0.01 to <0.05 IU/mL) and mild (0.05 to <0.4 IU/mL) cases, patients mostly bleed after minor trauma and surgical challenges. Uncontrolled bleeding into critical organs may lead to death, and bleeding into joints and muscles results in irreversible and progressive crippling (1). In the developed world, factor replacement therapy and newer therapeutic approaches are available to control, and in many cases prevent, bleeding (2). In the developing world, these therapies are minimally and variably available, and even identifying all hemophilia cases is largely beyond the capacity of many national health care systems (3, 4). Furthermore, treatment is associated with a range of problemsnamely the development of inhibitory antibodies against the administered factor and bloodborne infectionsthat complicate the use of blood products in the absence of appropriate procedures for virus inactivation (5, 6). The burden of a disease is its cumulative effect, including health outcomes, social aspects, and costs to society. Burden of disease is commonly calculated as the gap between an ideal situation, where everyone lives free of disease and disability, and the cumulated current health status in a community (7). It is often measured in disability-adjusted life-years, which incorporate years lived with the disability and years of life lost. Estimation of burden of disease requires knowledge of the prevalence (or the incident cases over time where applicable) of the disease in the population, stratified by age and sex (8). Therefore, establishing an accurate estimate of the number of persons with hemophilia in each setting of interest and worldwide is a necessary first step toward measuring the associated burden of disease, which in turn is important in comparing and contrasting the adequacy of health care systems across countries and levels of wealth. The first estimate of the global prevalence of hemophilia was 400000 patients (9); it was proposed in the early 2000s and was based on the prevalence in the United States (10) and a global population of 6 billion. This estimate did not distinguish severe from moderate and mild disease, nor did it distinguish between the 2 major types of hemophilia, A and B. Three main findings over the past few years have prompted a thorough reanalysis of the global prevalence and prevalence at birth of hemophilia: 1) The large variability in hemophilia prevalence across countries was measured, and the negative effect of lower socioeconomic status was highlighted (11, 12); 2) a large, countrywide, rigorous inception cohort study in France provided new data on the prevalence of hemophilia at birth (13); and 3) total number of patients with hemophilia, proportion of patients with severe hemophilia, and the gap between prevalence and prevalence at birth were proposed as indexes of the maturity of national health care systems (14), which moves us toward estimation of the global burden of hemophilia. The main aim of this study was to accurately estimate the prevalence and prevalence at birth of hemophilia, separately for severe and all hemophilia and for patients with hemophilia A and hemophilia B, using robust epidemiologic data from established national patient registries. Methods Data Sources Data from national patient registries in 6 high-income countries (Australia [15], Canada [16], France [17], Italy [18], New Zealand, and the United Kingdom [19]) were used to estimate prevalence, whereas prevalence at birth was estimated using data from the 3 most established registries (Canada, France, and the United Kingdom). Data from registries were gathered from the literature, from online data reports, and by contacting registry administrations directly for additional data. The registries of Canada, France, and the United Kingdom provided summary reports of the numbers of patients with hemophilia in their databases by year of birth over many years; these numbers were used to estimate the prevalence at birth of hemophilia. The total number of patients with hemophilia worldwide was taken from the World Federation of Hemophilia Annual Global Survey (20). Numbers of males and live male births by year and by country were taken from relevant national statistical databases (2126). Statistical Analysis All statistical analyses were done using Stata, version 15 (StataCorp). Prevalence Prevalence is the total number of identified cases of hemophilia divided by the total number of males in a population at a given time. Prevalence was segmented by type of hemophilia (A or B) and severity (severe only or all severities [that is, mild, moderate, and severe]). To estimate the prevalence of hemophilia A, severe hemophilia A, hemophilia B, and severe hemophilia B for each country, we used the most recent available data from its national registry on the number of patients with hemophilia and the corresponding male population data (23). The most recent data were from 2016 for Australia and Italy and 2017 for Canada, France, New Zealand, and the United Kingdom. To measure the completeness and accuracy of the registry data collection, we calculated prevalence for every year of registry data we had available (Supplement). Supplement. Supplementary Material Prevalence at Birth A congenital, nontransmissible disease like hemophilia has no incidence because no new cases emerge in the population after birth. Prevalence at birth, which represents the proportion of cases of disease in the population of live male births over a period of time, is a measure of newly identified cases over that period. We estimated prevalence at birth for hemophilia A, severe hemophilia A, hemophilia B, and severe hemophilia B for individual countries using data on the number of patients with hemophilia by year of birth from the 3 most established patient registries (France, Canada, and the United Kingdom) and live male births for the same years from relevant national statistical databases. To adjust for random variation in prevalence at birth by year, we averaged the prevalence at birth from all years between 1991 and 2015 within each registry. To correct for the delay in diagnosis and reporting to the registries (13), we excluded the last 5 years of data (the Supplement provides corrected estimates and details). We did sensitivity analyses by excluding the last 3 or 7 years of data (Supplement). Pooling Estimates of Prevalence and Prevalence at Birth Across Countries The country-specific estimates of prevalence and prevalence at birth were pooled using a random-effects meta-analysis method for proportions (27, 28) with the procedure metaprop for Stata. Life Expectancy Disadvantage For a monogenic disorder like hemophilia, when replacement therapy is available, treatment prevents all major consequences of the defect, and there is no expected differential mortality with respect to the general population. This assumption is supported by observations in patients with mild and moderate hemophilia after correction for hepatitis C virus (HCV) and HIV; in patients who have mild or moderate hemophilia but not HCV or HIV, the life expectancy tends to approximate that of the healthy population (29). Under this assumption, the 1 crude ratio of prevalence to prevalence at birth of hemophilia can be used as a raw measure of the survival disadvantage associated with the disease. We have calculated this ratio for hemophilia A, severe hemophilia A, hemophilia B, and severe hemophilia B for high-income countries. Estimate of the Global Number of Patients With Hemophilia Regardless of the existence of a life expectancy disadvantage, many patients with hemophilia worldwide are never diagnosed or reported to a registry and will therefore face disability and survival disadvantage. As a raw measure of the gap in care provision for hemophilia, we have compared the actual number of patients reported by the World Federation of Hemophilia (20), the number of patients expected assuming that the prevalence is the same in developed and developing countries (thus accounting for the survival disadvantage observed where care is available), and the number of patients expected based on prevalence at birth (that is, assuming no survival disadvantage associated with hemophilia). Role of the Funding Source This study was not supported by external funders. Results Table 1 shows the estimated prevalence (per 100000 males) obtained by pooling across the included registries for the last year of reporting for hemophilia A, severe hemophilia A, hemophilia B, and severe hemophilia B. No significant heterogeneity was detected for severe hemophilia A or B, whereas estimates including mild and moderate disease were heterogeneous. The Supplement provides prevalence data by country and by reporting year. Table 1. Estimated Prevalence (per 100000 Males) of Hemophilia A and Hemophilia B, for all Severities and Severe Only Table 2 shows the pooled estimates of prevalence at birth (per 100000 males) for Canada, France, and the United Kingdom for birth years 1991 to 2015 for hemophilia A, severe hemophilia A, hemophilia B, and severe hemophilia B. No heterogeneity was detected. Table 3 shows the pooled estimates of prevalence at birth (per 100000 males) after correction for diagnostic delay (that is, exclusion of birth years 2011 to 2015). No heterogeneity was detected. The Supplement provides the prevalence at birth for each year and each country and plots of the cumulative prevalence at birth for each of the 3 countries over time. Table 2. Estimated Prevalence at Birth (per 100000 Male Births) of Hemophilia A and Hemophilia B}, number={8}, journal={ANNALS OF INTERNAL MEDICINE}, author={Iorio, Alfonso and Stonebraker, Jeffrey S. and Chambost, Herve and Makris, Michael and Coffin, Donna and Herr, Christine and Germini, Federico and Stonebraker, Jeffrey S. and Iorio, Alfonso and Byams, Vanessa and et al.}, year={2019}, month={Oct}, pages={540-+} }
 @article{chai-adisaksopha_skinner_curtis_frick_nichol_noone_o'mahony_page_stonebraker_thabane_et al._2019, title={Exploring regional variations in the cross-cultural, international implementation of the Patient Reported Outcomes Burdens and Experience (PROBE) study}, volume={25}, ISSN={["1365-2516"]}, DOI={10.1111/hae.13703}, abstractNote={The Patient Reported Outcomes Burdens and Experience (PROBE) study has developed and validated the PROBE questionnaire for assessing patient‐reported outcomes in people with haemophilia and participants without bleeding disorders.}, number={3}, journal={HAEMOPHILIA}, author={Chai-Adisaksopha, Chatree and Skinner, Mark W. and Curtis, Randall and Frick, Neil and Nichol, Michael B. and Noone, Declan and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey and Thabane, Lehana and et al.}, year={2019}, month={May}, pages={365–372} }
 @article{chai-adisaksopha_skinner_curtis_frick_nichol_noone_o'mahony_page_stonebraker_thabane_et al._2019, title={Test-retest properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire and its constituent domains}, volume={25}, ISSN={["1365-2516"]}, DOI={10.1111/hae.13649}, abstractNote={The Patient Reported Outcomes, Burdens and Experiences (PROBE) study aims to develop and validate questionnaire for assessing health status in patients with haemophilia and participants without bleeding disorders.}, number={1}, journal={HAEMOPHILIA}, author={Chai-Adisaksopha, Chatree and Skinner, Mark W. and Curtis, Randall and Frick, Neil and Nichol, Michael B. and Noone, Declan and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey and Thabane, Lehana and et al.}, year={2019}, month={Jan}, pages={75–83} }
 @article{stonebraker_hajjar_orange_2018, title={Latent therapeutic demand model for the immunoglobulin replacement therapy of primary immune deficiency disorders in the USA}, url={https://doi.org/10.1111/vox.12651}, DOI={10.1111/vox.12651}, abstractNote={Our research aim is to model latent therapeutic demand (LTD) for the immunoglobulin replacement therapy (IgGRT) of primary immune deficiency disorders (PIDDs) in the USA. Given the high level of variability of IgGRT use and major differences among American and European practices in the management of patients with PIDDs, we develop a USA‐specific LTD model for common variable immune deficiency (CVID), hyper IGM syndrome, severe combined immune deficiency, Wiskott–Aldrich syndrome and X‐linked agammaglobulinemia (XLA).}, journal={Vox Sanguinis}, author={Stonebraker, J. S. and Hajjar, J. and Orange, J. S.}, year={2018}, month={Jul} }
 @article{noone_chai-adisaksopha_curtis_frick_nichol_o'mahony_page_stonebraker_iorio_skinner_2018, title={Occurrence and impact of pain among patients living with hemophilia: An analysis from the patient reported outcomes, burdens and experiences (PROBE) study}, volume={24}, number={S1}, journal={Haemophilia}, author={Noone, D. and Chai-Adisaksopha, C. and Curtis, R. and Frick, N. and Nichol, M. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A. and Skinner, M.W.}, year={2018}, pages={26–27} }
 @article{chai-adisaksopha_skinner_curtis_frick_nichol_noone_o'mahony_page_stonebraker_thabane_et al._2018, title={Psychometric properties of the Patient Reported Outcomes, Burdens and Experiences (PROBE) questionnaire}, volume={8}, ISSN={["2044-6055"]}, DOI={10.1136/bmjopen-2018-021900}, abstractNote={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.}, number={8}, journal={BMJ OPEN}, author={Chai-Adisaksopha, Chatree and Skinner, Mark W. and Curtis, Randall and Frick, Neil and Nichol, Michael B. and Noone, Declan and O'Mahony, Brian and Page, David and Stonebraker, Jeffrey and Thabane, Lehana and et al.}, year={2018}, month={Aug} }
 @article{noone_chai-adisaksopha_curtis_frick_nichol_o'mahony_page_stonebraker_iorio_skinner_2018, title={The Effect of Inhibitors on Quality of Life and Pain in Adult Patients with Severe Haemophilia in the PROBE Cohort}, volume={2}, number={S1}, journal={Research and Practice in Thrombosis Haemostasis}, author={Noone, D. and Chai-Adisaksopha, C. and Curtis, R. and Frick, N. and Nichol, M. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A. and Skinner, M.W.}, year={2018}, pages={76–77} }
 @article{pastarnak_curtis_frick_nichol_noone_o'mahony_page_stonebraker_iorio_skinner_2018, title={The PROBE Data Dashboard - A new tool for real-time data visualization and analysis of patient reported outcomes}, volume={24}, journal={Haemophilia}, author={Pastarnak, A. and Curtis, R. and Frick, N. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J. and Iorio, A. and Skinner, M.}, year={2018}, pages={181–182} }
 @article{dougall_chandra pani_daly_chai-adisaksopha_curtis_frick_iorio_noone_stonebraker_skinner_2018, title={The PROBE Study –Global burden of bleeding from the mouth in Haemophilia – a call to action}, volume={24}, number={S5}, journal={Haemophilia}, author={Dougall, A. and Chandra Pani, S. and Daly, B. and Chai-Adisaksopha, C. and Curtis, R. and Frick, N. and Iorio, A. and Noone, D. and Stonebraker, J. and Skinner, M.W.}, year={2018}, pages={66} }
 @article{skinner_chai-adisaksopha_curtis_frick_nichol_noone_o’mahony_page_stonebraker_iorio_2018, title={The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project: development and evaluation of a questionnaire assessing patient reported outcomes in people with haemophilia}, volume={4}, ISSN={2055-5784}, url={http://dx.doi.org/10.1186/s40814-018-0253-0}, DOI={10.1186/s40814-018-0253-0}, abstractNote={The interest of health care agencies, private payers and policy makers for patient-reported outcomes (PRO) is continuously increasing. There is a substantial need to improve capacity to collect and interpret relevant PRO data to support implementation of patient-centered research and optimal care in haemophilia. The Patient Reported Outcomes, Burdens and Experiences (PROBE) Project aims to develop a patient-led research network, to develop a standardized questionnaire to gather patient-reported outcomes and to perform a feasibility study of implementing the PROBE questionnaire.A pilot questionnaire was developed using focus group methodology. Content and face validity were assessed by a pool of persons living with haemophilia (PWH) and content experts through interactive workshops. The PROBE questionnaire was translated with the forward-backward approach. PROBE recruited national haemophilia patient non-governmental organizations (NGOs) to administer the questionnaire to people with and without haemophilia. PROBE measured the time to complete the questionnaire and gathered feedback on its content and clarity; staff time and cost required to implement the questionnaire were also collected.The PROBE questionnaire is comprised of four major sections (demographic data, general health problems, haemophilia-related health problems and health-related quality of life using EQ-5D-5L and EQ-VAS). Seventeen NGOs participated in the pilot study of the PROBE Project, recruiting 656 participants. Of these, 71% completed the questionnaire within 15 min, and all participants completed within 30 min. The median total staff and volunteer time required for the NGOs to carry out the study within their country was 9 h (range 2 to 40 h). NGO costs ranged from $22.00 to $543.00 USD per country, with printing and postage being the most commonly reported expenditures.The PROBE questionnaire assesses patient-important reported outcomes in PWH and control participants, with a demonstrated short completion time. PROBE proved the feasibility to engage diverse patient communities in the structured generation of real-world outcome research at all stages.Trial registration: NCT02439710.}, number={1}, journal={Pilot and Feasibility Studies}, publisher={Springer Nature}, author={Skinner, MW. and Chai-Adisaksopha, C. and Curtis, R. and Frick, N. and Nichol, M. and Noone, D. and O’Mahony, B. and Page, D. and Stonebraker, J. S. and Iorio, A.}, year={2018}, month={Feb}, pages={118–119} }
 @article{skinner_chai-adisaksopha_curtis_frick_noone_o'mahony_page_stonebraker_iorio_2017, title={Burden of Comorbid Diseases in Patients with Hemophilia: The Cross-Sectional Analysis of the Patient Reported Outcomes, Burden and Experiences (PROBE) Study}, volume={130}, number={S1}, journal={Blood}, author={Skinner, M.W. and Chai-Adisaksopha, C. and Curtis, R. and Frick, N. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A.}, year={2017}, pages={3411} }
 @article{skinner_chai adisaksopha_curtis_frick_nichol_noone_o'mahony_page_stonebraker_iorio_2017, title={Burden of comorbidities in persons living with haemophilia: insight from the PROBE phase 2b study database}, volume={1}, number={S1}, journal={Research and Practice in Thrombosis Haemostasis}, author={Skinner, M.W. and Chai Adisaksopha, C. and Curtis, R. and Frick, N. and Nichol, M.B. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A.}, year={2017}, pages={840–841} }
 @article{thompson_srivastava_skinner_stonebraker_epstein_kauf_valentino_2017, title={Estimating the global annual bleed rate in haemophilia}, volume={23}, journal={Haemophilia}, author={Thompson, C. and Srivastava, A. and Skinner, M. and Stonebraker, J. and Epstein, J. and Kauf, T. and Valentino, L.}, year={2017}, pages={86–86} }
 @article{chai-adisaksopha_skinner_curtis_frick_noone_o'mahony_page_stonebraker_iorio_2017, title={Psychometric Properties of the Patient Reported Outcomes Burdens and Experiences (PROBE) Questionnaire}, volume={130}, number={S1}, journal={Blood}, author={Chai-Adisaksopha, C. and Skinner, M.W. and Curtis, R. and Frick, N. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A.}, year={2017}, pages={5645} }
 @article{chai-adisaksopha_skinner_curtis_frick_nichol_noone_o'mahony_page_stonebraker_iorio_2017, title={Sexual Health in Patients with Hemophilia; The Insights from the Patient Reported Outcomes, Burdens and Experiences (PROBE) Study}, volume={130}, number={S1}, journal={Blood}, author={Chai-Adisaksopha, C. and Skinner, M.W. and Curtis, R. and Frick, N. and Nichol, M.B. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Iorio, A.}, year={2017}, pages={2141} }
 @article{chai-adisaksopha_iorio_curtis_frick_nichol_noone_o'mahony_page_stonebraker_skinner_2017, title={Test-retest reliability analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE) study}, volume={23}, number={S2}, journal={Haemophilia}, author={Chai-Adisaksopha, C. and Iorio, A. and Curtis, R. and Frick, N. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J. and Skinner, M. W.}, year={2017}, pages={50–51} }
 @article{iorio_stonebraker_brooker_soucie_2016, title={Measuring the quality of haemophilia care across different settings: a set of performance indicators derived from demographics data}, volume={23}, ISSN={1351-8216}, url={http://dx.doi.org/10.1111/hae.13127}, DOI={10.1111/hae.13127}, abstractNote={BackgroundHaemophilia 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.}, number={1}, journal={Haemophilia}, publisher={Wiley}, author={Iorio, A. and Stonebraker, J. S. and Brooker, M. and Soucie, J. M.}, year={2016}, month={Dec}, pages={e1–e7} }
 @article{chai-adisaksopha_iorio_curtis_frick_nichol_noone_o'mahony_page_stonebraker_skinner_2016, title={Psychometric Test-Retest Reliability Analysis of the Patient Reported Outcomes Burdens and Experiences (PROBE)}, volume={128}, number={22}, journal={Blood}, author={Chai-Adisaksopha, C. and Iorio, A. and Curtis, R. and Frick, N. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S. and Skinner, M.W.}, year={2016}, pages={5964} }
 @inproceedings{skinner_curtis_frick_iorio_nichol_noone_o'mahony_page_stonebraker_2016, title={The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Phase 1}, booktitle={HTAi 2016 Annual Meeting Abstract Book}, author={Skinner, M.W. and Curtis, R. and Frick, N. and Iorio, A. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S.}, year={2016}, pages={63–64} }
 @article{skinner_curtis_frick_iorio_nichol_noone_o'mahony_page_stonebraker_2016, title={The Patient Reported Outcomes, Burdens, and Experiences (PROBE) Phase 1 Study Methodology and Feasibility}, volume={22}, number={S2}, journal={Haemophilia}, author={Skinner, M.W. and Curtis, R. and Frick, N. and Iorio, A. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S.}, year={2016}, pages={43–44} }
 @article{skinner_curtis_frick_iorio_nichol_noone_o'mahony_page_stonebraker_2016, title={The patient reported outcome burdens and experiences (PROBE) study - phase 1 results show PROBE study methodology feasible}, volume={14}, number={S1}, journal={Journal of Thrombosis and Haemostasis}, author={Skinner, M.W. and Curtis, R. and Frick, N. and Iorio, A. and Nichol, M. and Noone, D. and O'Mahony, B. and Page, D. and Stonebraker, J.S.}, year={2016}, pages={47} }
 @inproceedings{iorio_stonebraker_brooker_soucie_2015, title={How to measure progress in the provision of care for hemophilia}, booktitle={The Proceedings of the World Federation of Hemophilia’s Ninth Global Forum on Research and Treatment Products for Bleeding Disorders}, author={Iorio, A. and Stonebraker, J.S. and Brooker, M. and Soucie, J.M.}, year={2015}, pages={16–17} }
 @article{stonebraker_farrugia_gathmann_orange_2014, title={Modeling Primary Immunodeficiency Disease Epidemiology and Its Treatment to Estimate Latent Therapeutic Demand for Immunoglobulin}, volume={34}, ISSN={["1573-2592"]}, DOI={10.1007/s10875-013-9975-1}, abstractNote={[[{:Label=>"PURPOSE", :NlmCategory=>"OBJECTIVE"}, "Estimating the underlying demand for immunoglobulin (Ig) is important to ensure that adequate provision is made for patients with primary immune deficiency (PID) in the context of the competing demands for Ig and to ensure optimal therapeutic regimens. The concept of latent therapeutic demand (LTD) was used to estimate evidence-based requirements and compared to the actual Ig consumption in different countries. The estimates were performed for common variable immunodeficiency (CVID) and X-linked Agammaglobulinaemia (XLA), the two most commonly studied PIDs using Ig."], [{:Label=>"METHODS", :NlmCategory=>"METHODS"}, "The LTD model for CVID and XLA was derived using decision analysis methodology. Data for the epidemiology and treatment variables were obtained from peer-reviewed publications, clinical registries and publicly-available patient surveys. Incomplete data records from registries were excluded from analysis. The variables impacting LTD were ranked in order of sensitivity through a tornado diagram. The uncertainty surrounding the variables was modeled using probabilistic distributions and evaluated using Monte Carlo simulation."], [{:Label=>"RESULTS", :NlmCategory=>"RESULTS"}, "Treatment dosage and prevalence were determined to be the most sensitive variables driving demand. The average potential usage of Ig for the treatment of CVID and XLA was estimated at 72 g per 1,000 population, which is higher than the estimated Ig usage in CVID and XLA of 27-41 g per 1,000 population in the US."], [{:Label=>"CONCLUSION", :NlmCategory=>"CONCLUSIONS"}, "The potential demand for treating CVID and XLA exceeds the currently observed usage of Ig in these disorders. Variable usage in different countries is due to varying prevalence and dosage practices. Under-reporting in patient registries represents a major obstacle to calculating the true prevalence of CVID and XLA. Modeling demand relies heavily upon accurate prevalence and practice estimates which reemphasize the importance of accurate registries and improved registry methods. As better data becomes available, revision of model variables provides opportunities to anticipate and prepare for evolving patient needs."]]}, number={2}, journal={JOURNAL OF CLINICAL IMMUNOLOGY}, author={Stonebraker, Jeffrey S. and Farrugia, Albert and Gathmann, Benjamin and Orange, Jordan S.}, year={2014}, month={Feb}, pages={233–244} }
 @inproceedings{stonebraker_farrugia_gathman_2013, title={A decision analysis model to estimate latent therapeutic demand for immunoglobulin therapy in primary immunodeficiencies}, booktitle={Proceedings of the 15th Meeting of the European Society Immunodeficiencies (ESID)}, author={Stonebraker, J. and Farrugia, A. and Gathman, B.}, year={2013}, pages={29–34} }
 @article{stonebraker_2013, title={Product-Generation Transition Decision Making for Bayer's Hemophilia Drugs: Global Capacity Expansion Under Uncertainty with Supply-Demand Imbalances}, volume={61}, ISSN={["0030-364X"]}, DOI={10.1287/opre.2013.1204}, abstractNote={ We present an application of decision analysis to global production capacity expansion under uncertainty for Bayer Group's proposed new biotechnology drug to treat hemophilia A. This decision analysis developed an improved approach to Bayer's 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, Bayer's 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 modeling in a large-scale applied decision analysis. The approach is applicable to other product-generation transition decision making for expanding global production capacity under uncertainty in a supply-constrained environment, especially for new product introductions and new product development decisions when supply is limited. }, number={5}, journal={OPERATIONS RESEARCH}, author={Stonebraker, Jeffrey S.}, year={2013}, pages={1119–1133} }
 @article{stonebraker_bolton-maggs_soucie_walker_brooker_2012, title={A study of variations in the reported haemophilia B prevalence around the world}, volume={18}, ISSN={["1351-8216"]}, DOI={10.1111/j.1365-2516.2011.02588.x}, abstractNote={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.}, number={3}, journal={HAEMOPHILIA}, author={Stonebraker, J. S. and Bolton-Maggs, P. H. B. and Soucie, J. Michael and Walker, I. and Brooker, M.}, year={2012}, month={May}, pages={e91–e94} }
 @article{farrugia_stonebraker_gathmann_2012, title={Decision analysis model for estimating latent therapeutic demand for immunoglobulin therapy in primary immune deficiencies}, volume={32}, number={S1}, journal={Journal of Clinical Immunology}, author={Farrugia, A. and Stonebraker, J.S. and Gathmann, B.}, year={2012}, pages={18} }
 @article{stonebraker_gil_kirkwood_handfield_2012, title={Impact factor as a metric to assess journals where OM research is published}, volume={30}, ISSN={["1873-1317"]}, url={http://www.scopus.com/inward/record.url?eid=2-s2.0-84857369472&partnerID=MN8TOARS}, DOI={10.1016/j.jom.2011.05.002}, abstractNote={Highlights► We conducted an exhaustive comparison of OM journal rankings based on impact factors versus other ranking methods (113 characters) ► Impact factors are useful metrics to rank OM journals (53 characters) ► Impact factor rankings alone are not a replacement for survey‐based, citation‐based, or author‐based methods (108 characters) ► Impact factors evaluate OM journal quality from another perspective and can be used with other methods to rank OM journals (122 characters) ► Impact factors are likely to shape and influence future perception of OM journal quality (88 characters)}, number={1-2}, journal={JOURNAL OF OPERATIONS MANAGEMENT}, author={Stonebraker, Jeffrey S. and Gil, Esther and Kirkwood, Craig W. and Handfield, Robert B.}, year={2012}, month={Jan}, pages={24–43} }
 @article{stonebraker_bolton-maggs_brooker_farrugia_srivastava_2011, title={A study of reported factor IX use around the world}, volume={17}, ISSN={["1365-2516"]}, DOI={10.1111/j.1365-2516.2010.02461.x}, abstractNote={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.}, number={3}, journal={HAEMOPHILIA}, author={Stonebraker, J. S. and Bolton-Maggs, P. H. B. and Brooker, M. and Farrugia, A. and Srivastava, A.}, year={2011}, month={May}, pages={446–455} }
 @inbook{stonebraker_keisler_2011, title={Empirically Investigating the Portfolio Management Process: Findings from a Large Pharmaceutical Company}, ISBN={9781441999429 9781441999436}, ISSN={0884-8289}, url={http://dx.doi.org/10.1007/978-1-4419-9943-6_6}, DOI={10.1007/978-1-4419-9943-6_6}, abstractNote={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.}, booktitle={Portfolio Decision Analysis}, publisher={Springer New York}, author={Stonebraker, Jeffrey S. and Keisler, Jeffrey}, year={2011}, pages={131–148} }
 @article{handfield_edwards_stonebraker_2011, title={NC State's Supply Chain Resource Cooperative Educates in the Real World}, volume={41}, ISSN={["1526-551X"]}, url={http://www.scopus.com/inward/record.url?eid=2-s2.0-83455262975&partnerID=MN8TOARS}, DOI={10.1287/inte.1110.0584}, abstractNote={ North Carolina State University's Supply Chain Resource Cooperative (SCRC) is a unique university-industry initiative that provides MBA and undergraduate students with opportunities to apply supply chain (SC) theory and concepts to field-based projects over a semester. We integrate field-based student course projects with real problems that companies are facing. The projects are led by SCRC directors, faculty at the Poole College of Management, and SC managers that financially support the SCRC. The SCRC focuses on improving the caliber of students entering the SC management profession. It accomplishes this by bringing the classroom into industry and involving students in finding viable solutions to real business problems, thus facilitating interactions between students and companies. Since 2000, 1,251 students have completed 331 projects with 39 companies. The SCRC is a self-funded small business that is supported by donor companies. Its organizational structure enables us to develop and maintain long-term relationships with donor companies, which are critical for a successful student-based outreach initiative with industry. In this paper, we describe the SCRC's evolution and benefits, and provide insights that may help other institutions interested in establishing similar organizations. }, number={6}, journal={INTERFACES}, author={Handfield, Robert B. and Edwards, Steven A. and Stonebraker, Jeffrey S.}, year={2011}, pages={548–563} }
 @article{bailey_snapp_yetur_stonebraker_edwards_davis_cox_2011, title={Practice Summaries: American Airlines Uses Should-Cost Modeling to Assess the Uncertainty of Bids for Its Full-Truckload Shipment Routes}, volume={41}, ISSN={["0092-2102"]}, DOI={10.1287/inte.1100.0515}, abstractNote={ 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 airline's risk of overpaying an FTL supplier. }, number={2}, journal={INTERFACES}, author={Bailey, Michael J. and Snapp, John and Yetur, Subramani and Stonebraker, Jeffrey S. and Edwards, Steven A. and Davis, Aaron and Cox, Robert}, year={2011}, pages={194–196} }
 @article{stonebraker_brooker_amand_farrugia_srivastava_2010, title={A study of reported factor VIII use around the world}, volume={16}, ISSN={["1365-2516"]}, DOI={10.1111/j.1365-2516.2009.02131.x}, abstractNote={Summary.  The effect of replacement therapy has significantly improved the morbidity and mortality of people with haemophilia A in high income countries, a recent socio‐economic development as the availability of safe concentrates has been matched by a willingness for their provision through reimbursement. In the developing world, however, this state has not been achieved, primarily because of the low visibility of haemophilia coupled with its expense, leading to inadequate treatment with its sequelae of severe pain, joint deformities, arthropathy, disabilities, and even death in childhood or early adult life. The objective of this paper was to study the reported factor VIII (FVIII) use on a country‐by‐country basis. Data on the reported FVIII use for 104 countries were obtained from the Marketing Research Bureau, Inc. and the World Federation of Hemophilia. The results show that FVIII use varies considerably among countries, even among the wealthiest of countries. The use of FVIII concentrate increases as economic capacity increases; in addition, consumption of FVIII has been increasing at a greater rate in high income countries. Given these trends, there probably will be a global increase in FVIII concentrates usage. Such information is critical for national healthcare agencies to determine realistic budget priorities in planning for an increased allocation of resources required to improve the treatment of patients with haemophilia A. This information is also important for pharmaceutical manufacturers to adequately plan for increased production of FVIII concentrates.}, number={1}, journal={HAEMOPHILIA}, author={Stonebraker, J. S. and Brooker, M. and Amand, R. E. and Farrugia, A. and Srivastava, A.}, year={2010}, month={Jan}, pages={33–46} }
 @article{stonebraker_bolton-maggs_soucie_walker_brooker_2010, title={A study of variations in the reported haemophilia A prevalence around the world}, volume={16}, ISSN={["1365-2516"]}, DOI={10.1111/j.1365-2516.2009.02127.x}, abstractNote={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.}, number={1}, journal={HAEMOPHILIA}, author={Stonebraker, J. S. and Bolton-Maggs, P. H. B. and Soucie, J. Michael and Walker, I. and Brooker, M.}, year={2010}, month={Jan}, pages={20–32} }
 @article{stonebraker_2009, title={Factor Usage - Global Data.}, journal={The Proceedings of the World Federation of Hemophilia’s Sixth Global Forum on the Safety and Supply of Hemophilia Treatment Products for Bleeding Disorders – Making the Case for Clotting Factor Concentrates}, author={Stonebraker, J.S.}, year={2009}, pages={9–11} }
 @article{stonebraker_keefer_2009, title={OR Practice—Modeling Potential Demand for Supply-Constrained Drugs: A New Hemophilia Drug at Bayer Biological Products}, volume={57}, ISSN={0030-364X 1526-5463}, url={http://dx.doi.org/10.1287/opre.1070.0506}, DOI={10.1287/opre.1070.0506}, abstractNote={This paper describes the evolution and application of a novel approach for forecasting drug demand in markets where supply limitations have significantly curtailed sales volumes and thus reduced the usefulness of conventional sales-based forecasting methods. This occurs frequently with biological (biotech) drugs. We use methods from decision analysis to explicitly model the variability in epidemiological data together with the variability in treatment modalities to estimate latent therapeutic demand (LTD)---the underlying demand that captures how physicians would prescribe treatment and how patients would comply if ample supplies of drugs were available and affordable. Our approach evolved from efforts to help Bayer Biological Products with strategic decisions regarding its drug for treating hemophilia A, the future of which had been clouded for several years, primarily due to a lack of confidence in demand estimates. Use of the LTD model resulted in a better understanding of the therapeutic needs of the global hemophilia community and helped Bayer make good decisions. We believe this approach is widely applicable to forecasting potential demand for supply-constrained as well as brand-new drugs, and thus can be very useful in helping both drug manufacturers and health-care agencies worldwide to ensure adequate supplies of critical drugs.}, number={1}, journal={Operations Research}, publisher={Institute for Operations Research and the Management Sciences (INFORMS)}, author={Stonebraker, Jeffrey S. and Keefer, Donald L.}, year={2009}, month={Feb}, pages={19–31} }
 @inbook{beccue_stonebraker_2007, place={Cambridge UK}, title={Balancing Environmental and Operational Objectives in Nuclear Refueling Strategies}, ISBN={9780511611308}, url={http://dx.doi.org/10.1017/cbo9780511611308.028}, DOI={10.1017/cbo9780511611308.028}, abstractNote={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 $1 billion. IP3 withdraws 840,000 gallons of water per minute from the Hudson River for cooling steam and then returns it to the river. When water is withdrawn from the river between February and September so are fish eggs and small fish. Some of the fish do not survive as they pass through the cooling water system of IP3 (entrainment) because of temperature increases, pressure changes, and sheer forces. The effect of entrainment on fish populations in the Hudson River has been the subject of both extensive litigation and scientific research (Barnthouse et al, 1988). The NYPA can reduce the effect of entrainment by scheduling plant shutdowns to refuel IP3 when fish eggs and small fish are most abundant in the Hudson River and taking those outages as scheduled. In the past, the NYPA prepared its nuclear refueling outage schedules for IP3 using a 10-year planning horizon, assuming the operation and refueling of IP3 went exactly as planned. However, unforeseen events often altered operation and refueling, causing refueling outage schedules to deviate from what was planned. In the future, there is uncertainty about when New York State will deregulate the electric utility industry and the effect on the cost of replacement power that the NYPA will have to buy during refueling outages. NYPA must systematically consider these uncertainties to ensure that it can continue to provide low cost power and can reduce the environmental effects of operating IP3. For these reasons, the NYPA decided to use Advances: Nuclear Refueling Page 4 of 32 Ch 27 060520 V06 decision analysis rather than other approaches, such as mixed-integer programming (Fourcade et al., 1997), to help it to schedule nuclear refueling outages at IP3. In this chapter, we describe the decision analysis tools and processes that we brought to the environmental and operational dilemma faced by the NYPA. We will describe the model structure and highlight the key inputs and how we collected those inputs from experts within the NYPA. We conclude with insights and results and comment on our own challenges as well as the reactions from the stakeholders. Problem Structuring – Designing the Project for Success The decision analysis project started on October 23, 1997, and the NYPA required that it be finished by December 4, 1997. Our decision analysis approach consisted of four phases: framing, modeling, data collection, and evaluation. During the framing phase, we determined who would be involved in the project, the scope of the analysis, and the important factors (alternatives, uncertainties, and objectives) of the refueling outage plan. The project team consisted of a core group (NYPA senior managers and decision analysts—the authors), subject-matter experts, and decision-makers. Our role was to conduct a decision analysis that was responsive to the needs of decision-makers. We used various decision analysis tools to organize and record the results of the framing phase. These included a decision-quality spider diagram, a decision pyramid, an objectives hierarchy, a strategy table, a decision tree, and an influence diagram. Advances: Nuclear Refueling Page 5 of 32 Ch 27 060520 V06 Mission Statement We determined the scope of the project by (1) developing a mission statement and (2) clearly defining the decisions and their boundaries. We constructed the mission statement by considering four questions: what are we going to do, why are we doing this, how will we know we are successful, and how can we fail? The resulting mission statement was as follows: 1) Identify strategies for scheduling refueling outages at IP3 over the period 1999 through 2008 that range from unrestricted operation, in which outages can begin at any time and protection of fish eggs and small fish is not a concern, to restricted operation, in which outages must begin during a specified week and the primary concern is maximizing protection of fish eggs and small fish, 2) Develop a decision analysis model for comparing the cost and amount of fish protection associated with the refueling strategies based on information provided by the experts at the NYPA and the level of confidence they have in that information, 3) Compare the refueling strategies in terms of cost and fish protection, and 4) Provide, by December 4, 1997, the results of the decision analysis model to the NYPA decision-makers. Decision Pyramid During the first week of the project, we listed decisions that could affect the mission. We then categorized each decision as policy, strategic, or tactical to help bound the NYPA’s problem and focus our attention at the right level. A decision pyramid (Matheson and Matheson, 1998) helped us to organize the three categories of decisions (Figure 27.1). This decision pyramid defines the Advances: Nuclear Refueling Page 6 of 32 Ch 27 060520 V06 scope of the analysis for the NYPA by focusing attention on the key strategic choices. We assume that policy decisions are given. Tactical decisions are to be made in the future.}, booktitle={Advances in Decision Analysis}, publisher={Cambridge University Press}, author={Beccue, Phillip C. and Stonebraker, Jeffrey S.}, editor={Edwards, Ward and Miles, Ralph F., Jr and von Winterfeldt, DetlofEditors}, year={2007}, month={Dec}, pages={564–581} }
 @inproceedings{stonebraker_2007, title={Understanding the supply-demand-price conundrum in the FVIII market}, booktitle={The Proceedings of the World Federation of Hemophilia’s Fifth Global Forum on the Safety and Supply of Hemophilia Treatment Products}, author={Stonebraker, J.S.}, year={2007}, pages={20–21} }
 @article{stonebraker_amand_bauman_nagle_larson_2004, title={Modelling haemophilia epidemiology and treatment modalities to estimate the unconstrained factor VIII demand}, volume={10}, ISSN={1351-8216 1365-2516}, url={http://dx.doi.org/10.1046/j.1365-2516.2003.00841.x}, DOI={10.1046/j.1365-2516.2003.00841.x}, abstractNote={Summary.  The article presents a new method for estimating the unconstrained factor VIII (FVIII) demand based on the principles of decision analysis. Epidemiology and treatment modalities were integrated into a model for unconstrained FVIII demand. Assumptions for each variable with impact on the unconstrained FVIII demand were defined and probability estimates for these variables were obtained from the literature and medical experts. The sensitivity of the unconstrained FVIII demand to each of the variables was determined, and the variables with the greatest impact were modelled probabilistically. The probability‐weighted average for the unconstrained FVIII demand model was 6.9 units per capita with a 90% uncertainty interval of 2.7–13.6 units per capita. When compared with FVIII usage in countries, only Luxembourg's use of FVIII (7.7 units per capita) exceeded the probability‐weighted average for the modelled unconstrained FVIII demand. As better information becomes available, revision of model variables is easily accomplished allowing for a more accurate and dynamic forecast of demand over time. More accurate modelling of the ‘true’ demand longitudinally should help prevent shortages of FVIII concentrates such as those that have occurred in the past. In addition, a more accurate forecast of FVIII demand will allow national health care policy makers to better allocate financial and other resources. Sufficient and consistent supply of FVIII concentrates and appropriate financing of haemophilia care will allow the clinical benefits of more aggressive treatment regimens such as prophylaxis to be realized.}, number={1}, journal={Haemophilia}, publisher={Wiley}, author={Stonebraker, J. S. and Amand, R. E. and Bauman, M. V. and Nagle, A. J. and Larson, P. J.}, year={2004}, month={Jan}, pages={18–26} }
 @article{stonebraker_amand_nagle_2003, title={A country-by-country comparison of FVIII concentrate consumption and economic capacity for the global haemophilia community}, volume={9}, ISSN={1351-8216 1365-2516}, url={http://dx.doi.org/10.1046/j.1365-2516.2003.00758.x}, DOI={10.1046/j.1365-2516.2003.00758.x}, abstractNote={Summary.  The article presents a method to estimate the level of factor VIII (FVIII) concentrate consumption and economic capacity for each of the 110 countries considered in the study. A number of health‐economic indicators were examined and purchasing power parity (PPP) gross national income (GNI) per capita had the strongest correlation with amount of FVIII concentrates sold in a country. Available data on FVIII concentrate consumption and health‐economic indicators were used to calculate index values for FVIII use and economic capacity so each country can assess and compare its level of FVIII concentrate consumption with the economic resources of other countries in the global haemophilia A community. This article is of importance to policy makers, care givers and patient organizations in planning health‐care resources for the treatment of haemophilia.}, number={3}, journal={Haemophilia}, publisher={Wiley}, author={Stonebraker, J. S. and Amand, R. E. and Nagle, A. J.}, year={2003}, month={May}, pages={245–250} }
 @inproceedings{larson_stonebraker_amand_bauman_nagle_2003, title={Probabilistic Modelling of population-based epidemiology and treatment modalities to determine unconstrained factor VIII demand}, volume={1}, number={S1}, booktitle={Journal of Thrombosis and Haemostasis}, author={Larson, P.J. and Stonebraker, J.S. and Amand, R.E. and Bauman, M.V. and Nagle, A.J.}, year={2003}, pages={P1645} }
 @article{stonebraker_2002, title={How Bayer Makes Decisions to Develop New Drugs}, volume={32}, ISSN={0092-2102 1526-551X}, url={http://dx.doi.org/10.1287/inte.32.6.77.6475}, DOI={10.1287/inte.32.6.77.6475}, abstractNote={Drug development is time consuming, resource intensive, risky, and heavily regulated. To ensure that it makes the best drug-development decisions, Bayer Pharmaceuticals (Pharma) uses a structured process based on the principles of decision analysis to evaluate the technical feasibility and market potential of its new drugs. In July 1999, the biological products leadership committee composed of the senior managers within Bayer Biological Products (BP), a business unit of Pharma, made its newly formed strategic-planning department responsible for the commercial evaluation of a new blood-clot-busting drug. Even though Pharma's use of decision analysis began in the late 1980s, this commercial evaluation was BP's first decision analysis project. Previously, BP had analyzed a few business cases for review by Pharma. Pharma senior managers considered our recommendations relevant to their decision making. The project also institutionalized decision analysis at the business-unit level.}, number={6}, journal={Interfaces}, publisher={Institute for Operations Research and the Management Sciences (INFORMS)}, author={Stonebraker, Jeffrey S.}, year={2002}, month={Dec}, pages={77–90} }
 @article{dunning_lockfort_ross_beccue_stonebraker_2001, title={New York Power Authority Uses Decision Analysis to Schedule Refueling of Its Indian Point 3 Nuclear Power Plant}, volume={31}, ISSN={0092-2102 1526-551X}, url={http://dx.doi.org/10.1287/inte.31.5.121.9658}, DOI={10.1287/inte.31.5.121.9658}, abstractNote={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 shut down for 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 third week in May, rather than allowing the starting date to float throughout the period from May through August. However, the decision makers were surprised that the more restrictive strategies also resulted in lower costs.}, number={5}, journal={Interfaces}, publisher={Institute for Operations Research and the Management Sciences (INFORMS)}, author={Dunning, Dennis J. and Lockfort, Steve and Ross, Quentin E. and Beccue, Phillip C. and Stonebraker, Jeffrey S.}, year={2001}, month={Oct}, pages={121–135} }
 @article{stonebraker_kirkwood_1997, title={Formulating and solving sequential decision analysis models with continuous variables}, volume={44}, ISSN={0018-9391}, url={http://dx.doi.org/10.1109/17.552807}, DOI={10.1109/17.552807}, abstractNote={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.}, number={1}, journal={IEEE Transactions on Engineering Management}, publisher={Institute of Electrical and Electronics Engineers (IEEE)}, author={Stonebraker, J.S. and Kirkwood, C.W.}, year={1997}, pages={43–53} }
 @article{stonebraker_sage_leak_1997, title={Practice Abstract: Decision Analysis Provides Insight to Ford Microelectronics Incorporated}, volume={27}, number={2}, journal={Interfaces}, author={Stonebraker, J.S. and Sage, J.J. and Leak, B.L.}, year={1997}, month={Jan}, pages={107–111} }
 @inproceedings{stonebraker_1996, place={Maxwell Air Force Base, AL}, title={It’s Time to Think About Values First}, booktitle={Quality Air Force in the 21st Century: Technology & Innovation 1996 Quality Air Force Symposium Proceedings}, publisher={Air Force Quality Institute}, author={Stonebraker, J.S.}, year={1996}, pages={78–88} }
 @inproceedings{francis_stonebraker_1991, title={Can source selection benefit from decision analysis?}, booktitle={Acquisition Research Symposium Proceedings}, author={Francis, V.E. and Stonebraker, J.S.}, year={1991}, pages={287–298} }
 @article{francis_stonebraker_antons_1991, title={Simon says: Rational decision-making for source selection}, volume={20}, number={5}, journal={Program Manager: Journal of the Defense Systems Management College}, author={Francis, V.E. and Stonebraker, J.S. and Antons, C.M.}, year={1991}, month={Sep}, pages={34–40} }