Cost-consequence model comparing eltrombopag versus romiplostim for adult patients with chronic immune thrombocytopenia.

BACKGROUND: Thrombopoietin-receptor agonists eltrombopag (EPAG) and romiplostim (ROMI) are treatment options for adults with chronic immune thrombocytopenia (cITP) who have had an insufficient response to corticosteroids or immunoglobulins. METHODS: A cost-consequence model was developed to evaluate the costs relative to treatment success of EPAG, ROMI, and watch and rescue (W&R) in previously treated patients. The primary endpoint assessed was severe bleeding, derived from all identified phase III registered clinical trials. Health outcomes were compared via indirect treatment comparison. Costs incorporated in the model included drug and administration, routine care, rescue medications, bleeding-related adverse events, other adverse events, and mortality costs. A trial (26-week) time horizon was used, as certain endpoints used in the model were bound to within-trial results. RESULTS: In the intent-to-treat (ITT) population, the overall estimated cost per patient for EPAG was US$66,560 compared to US$91,039 for ROMI and US30,099 for W&R. Compared to the ITT population, the difference in cost between EPAG and ROMI was slightly greater in splenectomized patients (US65,998 for EPAG compared to US$91,485 for ROMI) and slightly less in non-splenectomized patients (US$67,151 for EPAG compared to US$91,455 for ROMI), though the overall trend remained the same. When assessing cost per severe bleeding event avoided in the ITT population, EPAG dominated (less expensive, more effective) ROMI. Sensitivity analyses confirmed these results. CONCLUSION: EPAG was preferred over ROMI in the treatment of cITP, largely driven by the reduction in severe bleeding events associated with its use

The Long-Baseline Neutrino Experiment: Exploring Fundamental Symmetries of the Universe

The preponderance of matter over antimatter in the early Universe, the dynamics of the supernova bursts that produced the heavy elements necessary for life and whether protons eventually decay --- these mysteries at the forefront of particle physics and astrophysics are key to understanding the early evolution of our Universe, its current state and its eventual fate. The Long-Baseline Neutrino Experiment (LBNE) represents an extensively developed plan for a world-class experiment dedicated to addressing these questions. LBNE is conceived around three central components: (1) a new, high-intensity neutrino source generated from a megawatt-class proton accelerator at Fermi National Accelerator Laboratory, (2) a near neutrino detector just downstream of the source, and (3) a massive liquid argon time-projection chamber deployed as a far detector deep underground at the Sanford Underground Research Facility. This facility, located at the site of the former Homestake Mine in Lead, South Dakota, is approximately 1,300 km from the neutrino source at Fermilab -- a distance (baseline) that delivers optimal sensitivity to neutrino charge-parity symmetry violation and mass ordering effects. This ambitious yet cost-effective design incorporates scalability and flexibility and can accommodate a variety of upgrades and contributions. With its exceptional combination of experimental configuration, technical capabilities, and potential for transformative discoveries, LBNE promises to be a vital facility for the field of particle physics worldwide, providing physicists from around the globe with opportunities to collaborate in a twenty to thirty year program of exciting science. In this document we provide a comprehensive overview of LBNE's scientific objectives, its place in the landscape of neutrino physics worldwide, the technologies it will incorporate and the capabilities it will possess.Comment: Major update of previous version. This is the reference document for LBNE science program and current status. Chapters 1, 3, and 9 provide a comprehensive overview of LBNE's scientific objectives, its place in the landscape of neutrino physics worldwide, the technologies it will incorporate and the capabilities it will possess. 288 pages, 116 figure

Cost-effectiveness analysis for midostaurin versus standard of care in acute myeloid leukemia in the United Kingdom

Abstract Aims Midostaurin (MIDO) has been proposed for the treatment of newly-diagnosed adult patients with FMS-like tyrosine kinase 3 mutation-positive (FLT3+) acute myeloid leukemia (AML) in combination with standard chemotherapy. The cost-effectiveness of MIDO and standard of care (SOC) followed by MIDO monotherapy was compared to SOC alone for newly-diagnosed FLT3+ AML in the UK. Methods A partitioned survival model was developed from a UK public healthcare system perspective to compare the cost-effectiveness of MIDO plus SOC and SOC over a lifetime horizon. The model included the following health states/partitions: induction, consolidation, monotherapy, complete remission (CR), relapse, stem cell transplantation (SCT), SCT recovery, and post-SCT recovery. Data on CR, overall survival, and adverse events were obtained from a Phase III clinical trial. Overall survival was extrapolated beyond the trial horizon using a ‘cure model’ approach and data from the Office for National Statistics. Utilities were identified via a systematic review. Routine care utilization was obtained from the National Institute for Health and Care Excellence single technology appraisal for azacitidine in AML (TA399). The costs of drugs and administration, adverse events, hospitalizations, physician visits, and end-of-life care were incorporated. Results Incremental life years (LYs) and quality-adjusted life years (QALYs) gained by patients on MIDO and SOC versus SOC were 1.67 and 1.47, respectively. At an incremental cost of £54,072 over a lifetime horizon, the ICER was £32,465 per LY and £36,826 per QALY. Sensitivity analyses were generally consistent with the base case findings. Conclusions With limited treatments in FLT3+ AML, MIDO represents a clinically significant advance in the management of newly-diagnosed AML. Using a threshold of £50,000 per QALY for end-of-life treatment, MIDO was shown to be a cost-effective option for newly-diagnosed FLT3+ AML

A decision framework for treating chronic immune thrombocytopenia with thrombopoietin receptor agonists

Aim: Eltrombopag and romiplostim are comparable second-line therapies in chronic immune thrombocytopenia. Treatment decisions are made in different contexts. A framework was created to outline decision pathways for physicians and payers. Materials & methods: The costs of drugs, administration, routine care, bleeding, other adverse events and mortality were included in the year-long calculation of total costs from a US private payer perspective. Treatment parameters and outcome data were obtained from relevant clinical trials. Results: The total cost per year, per patient of eltrombopag was US$51,000 versus US$76,000 for romiplostim. Drug costs and costs associated with bleeding-related events were the main drivers of cost difference. Conclusion: This framework facilitates decision-making in the management of chronic immune thrombocytopenia with eltrombopag and romiplostim