Cost-effectiveness analysis of rFVIIIFc versus contemporary rFVIII treatments for patients with severe hemophilia A without inhibitors in the United States

Background A range of treatments for patients with severe hemophilia A (HA) have been developed over the last decade, allowing for reduced frequency of administration and improved outcomes (joint health and breakthrough bleeding rates). While clinically effective, the cost effectiveness of these treatments has not been established. Objective This study presents a cost-effectiveness analysis of contemporary rFVIII treatments for severe HA patients without inhibitors. Methods A published semi-Markov model was used to compare three different prophylaxis regimens: (1) extended half-life (EHL) recombinant Factor VIII (rFVIII) Fc-fusion protein (rFVIIIFc, Eloctate®, Sanofi), (2) EHL PEGylated rFVIII (PEG-rFVIII, Adynovate®, Takeda), and (3) standard half-life (SHL) rFVIII (antihemophilic factor [recombinant], Advate®, Takeda), used as a proxy for all SHL rFVIII treatments. Acquisition costs were included based on published dosing and weight data. Benefits were incorporated through published annualized bleeding rates, rates of target joint development/resolution, and improvements in the modified hemophilia joint health score. Results were presented as total, discounted costs, and quality-adjusted life-years (QALYs). Results rFVIIIFc was shown to provide the most QALYs (27.922) compared with both PEG-rFVIII (27.454) and SHL rFVIII (27.071), at lower costs. Discounted lifetime costs were estimated at US$18.235m (rFVIIIFc), US$20.198m (PEG-rFVIII), and US$18.285m (SHL rFVIII), and were predominantly affected by model settings related to acquisition costs, patient weight, and dosing. Conclusions rFVIIIFc may offer a cost-effective option for severe HA patients. Uncertainties owing to the limited evidence base is the main limitation of the study

Insights Into Patients' Experience With Type 1 Diabetes: Exit Interviews From Phase III Studies of Sotagliflozin

Purpose: The purpose of this study was to conduct qualitative participant interviews to provide context to the meaningfulness of improvements in end points seen in 2 large-scale Phase III sotagliflozin trials in participants with type 1 diabetes. Methods: Participants were eligible for an interview if they had exited one of the clinical trials within the previous 12 months. Participants were recruited by investigators at the clinical trial sites, and interviews were conducted by independent interviewers by telephone in accordance with a semistructured interview guide. Both interviewers and participants were blinded to treatment assignment. Qualitative analysis was conducted using ATLAS-ti version 7.5, and descriptive statistics were computed and summarized. Findings: Across 3 countries, 41 participants were interviewed. Difficulty maintaining blood glucose within a desired range, described by participants as lack of blood glucose “stability,” was the most concerning symptom that they reported, wanting to see it improved during the clinical trial because it negatively impacted their physical, mental, and emotional lives. Participants who reported symptom improvement also reported a positive psychosocial impact while taking the clinical trial medication. All participants who monitored ketones described themselves as being “pretty confident” to “very confident” that they could avoid diabetic ketoacidosis by monitoring both ketone levels and understanding the physical signs and symptoms of hyperglycemia. Implications: Improvements in glucose stability and control were important to participants with type 1 diabetes, as these improvements were correlated with improvements in the participants' lives. ClinicalTrials.gov identifiers: NCT02384941; NCT02421510

Review of the cost of venous thromboembolism

Maria M Fernandez,1 Susan Hogue,1 Ronald Preblick,2 Winghan Jacqueline Kwong2 1RTI-Health Solutions, Market Access and Outcomes Strategy, Research Triangle Park, NC, 2Daiichi Sankyo, Inc., Health Economics & Outcomes Research, Parsippany, NJ, USA Background: Venous thromboembolism (VTE) is the second most common medical complication and a cause of excess length of hospital stay. Its incidence and economic burden are expected to increase as the population ages. We reviewed the recent literature to provide updated cost estimates on VTE management. Methods: Literature search strategies were performed in PubMed, Embase, Cochrane Collaboration, Health Economic Evaluations Database, EconLit, and International Pharmaceutical Abstracts from 2003–2014. Additional studies were identified through searching bibliographies of related publications. Results: Eighteen studies were identified and are summarized in this review; of these, 13 reported data from the USA, four from Europe, and one from Canada. Three main cost estimations were identified: cost per VTE hospitalization or per VTE readmission; cost for VTE management, usually reported annually or during a specific period; and annual all-cause costs in patients with VTE, which included the treatment of complications and comorbidities. Cost estimates per VTE hospitalization were generally similar across the US studies, with a trend toward an increase over time. Cost per pulmonary embolism hospitalization increased from 5,198–6,928 in 2000 to $8,764 in 2010. Readmission for recurrent VTE was generally more costly than the initial index event admission. Annual health plan payments for services related to VTE also increased from$10,804–16,644 during the 1998–2004 period to an estimated average of 15,123 for a VTE event from 2008 to 2011. Lower costs for VTE hospitalizations and annualized all-cause costs were estimated in European countries and Canada. Conclusion: Costs for VTE treatment are considerable and increasing faster than general inflation for medical care services, with hospitalization costs being the primary cost driver. Readmissions for VTE are generally more costly than the initial VTE admission. Further studies evaluating the economic impact of new treatment options such as the non-vitamin K antagonist oral anticoagulants on VTE treatment are warranted. Keywords: deep vein thrombosis, pulmonary embolism, costs, pharmacoeconomics, resource utilizatio