The Effectiveness and Value of Deflazacort and Exon-Skipping Therapies for the Management of Duchenne Muscular Dystrophy.

DISCLOSURES: Funding for this summary was contributed by Arnold Ventures, Commonwealth Fund, California Health Care Foundation, National Institute for Health Care Management (NIHCM), New England States Consortium Systems Organization, Blue Cross Blue Shield of Massachusetts, Harvard Pilgrim Health Care, Kaiser Foundation Health Plan, and Partners HealthCare to the Institute for Clinical and Economic Review (ICER), an independent organization that evaluates the evidence on the value of health care interventions. ICER's annual policy summit is supported by dues from Aetna, America's Health Insurance Plans, Anthem, Allergan, Alnylam, AstraZeneca, Biogen, Blue Shield of CA, Cambia Health Services, CVS, Editas, Express Scripts, Genentech/Roche, GlaxoSmithKline, Harvard Pilgrim, Health Care Service Corporation, Health Partners, Johnson & Johnson (Janssen), Kaiser Permanente, LEO Pharma, Mallinckrodt, Merck, Novartis, National Pharmaceutical Council, Premera, Prime Therapeutics, Regeneron, Sanofi, Spark Therapeutics, and United Healthcare. Agboola, Fluetsch, Rind, and Pearson are employed by ICER. Lin reports support from ICER during work on this economic model and grants from Mount Zion Health Fund, National Institutes of Health (National Cancer Institute and National Heart, Lung, and Blood Institute), and the Tobacco-Related Diseases Research Program, unrelated to this work. Walton reports support from ICER for work on this economic model and unrelated consulting fees from Baxter.

Evaluating a remote patient monitoring program for automated peritoneal dialysis.

BACKGROUND: The benefits of automated peritoneal dialysis (APD) have been established, but patient adherence to treatment remains a concern. Remote patient monitoring (RPM) programs are a potential solution; however, the cost implications are not well established. This study modeled, from the payer perspective, expected net costs and clinical consequences of a novel RPM program in Colombia. METHODS: Amarkov model was used to project costs and clinical outcomes for APD patients with and without RPM. Clinical inputs were directly estimated from Renal Care Services data or taken from the literature. Dialysis costs were estimated from national fees. Inpatient costs were obtained from a recent Colombian study. The model projected overall direct costs and several clinical outcomes. Deterministic and probabilistic sensitivity analyses (DSA and PSA) were also conducted to characterize uncertainty in the results. RESULTS: The model projected that the implementation of an RPM program costing US$35 per month in a cohort of 100 APD patients over 1 year would save US$121,233. The model also projected 31 additional months free of complications, 27 fewer hospitalizations, 518 fewer hospitalization days, and 6 fewer peritonitis episodes. In the DSA, results were most sensitive to hospitalization rates and days of hospitalization, but cost savings were robust. The PSA found there was a 91% chance for the RPM program to be cost saving. CONCLUSION: The results of the model suggest that RPM is cost-effective in APD patients which should be verified by a rigorous prospective cost analysis.

Comparison of 6-Month Costs Between Oral Antiplatelet Agents Following Acute Coronary Syndrome.

BACKGROUND: In patients with acute coronary syndrome (ACS) treated with percutaneous coronary intervention (PCI), newer antiplatelet agents prasugrel and ticagrelor have lower rates of cardiovascular events when compared with clopidogrel. However, it is unclear whether there are differences in economic outcomes when comparing these agents in ACS-PCI patients. OBJECTIVE: To assess aggregated costs and medical resource utilization among ACS-PCI patients prescribed prasugrel, ticagrelor, or generic clopidogrel, using a large commercial insurance claims database. METHODS: Costs attributable to any medical and pharmacy service and resource utilization including number of admissions, length of hospital stay, emergency room visits, and office visits over the 180-day postdischarge period were compared. All-cause and cardiovascular health care costs and resource utilization were separately analyzed for patients enrolled in the data over the continuous follow-up (CFU) period, and for patients continuously taking their initial treatment for 6 months (CTX). Potential confounders collected over a 6-month baseline assessment period were controlled for, using a generalized linear model. RESULTS: Over the 180-day follow-up, prasugrel and ticagrelor patients underwent fewer admissions (rate ratio [RR] = 0.87, 95% CI = 0.80-0.95) from CFU and RR = 0.81, 95% CI = 0.71-0.89 from CTX) compared with clopidogrel patients. The newer agent cohort incurred more overall health care costs than the generic clopidogrel group, with added costs of $957 (95% CI = $236-$1,725) in the CFU group and $1,122 (95% CI = $455-$1,865) in the CTX group, which were smaller than the increase in all-cause outpatient pharmacy costs associated with the newer agents versus clopidogrel (CFU: $1,175, 95% CI = $1,079-$1,278 and CTX: $1,360, 95% CI = $1,256-$1,487). Overall, there was no statistically significant difference in the economic outcomes associated with prasugrel and ticagrelor. There were, however, significant correlations between all-cause and cardiovascular-related outcomes. CONCLUSIONS: The higher price of prasugrel and ticagrelor was partially offset by a decrease in hospital admission compared with generic clopidogrel over a 6-month postdischarge period. Aggregated medical costs and resource utilization were not significantly different between prasugrel and ticagrelor patients. DISCLOSURES: No funding was received for this study. DiDomenico has received an honorarium from Amgen for preparation of a heart failure drug monograph for Pharmacy Practice News and serves as an advisory board member for a heart failure program at Otsuka America Pharmaceuticals and for Novartis Pharmaceuticals. Touchette has received unrestricted grant funding from Cardinal Health, Sunovion Pharmaceuticals, and Takeda and has served as a consultant to and director of the American College of Clinical Pharmacy Practice-Based Research Network on a study funded by Pfizer. Walton has served as a paid consultant for Bristol-Myers Squibb, Baxter, Merck, Genentech, Primus, Takeda, and Abbott. The other authors have nothing to disclose.

Comparative Effectiveness of Oral Antiplatelet Agents in Patients with Acute Coronary Syndrome.

OBJECTIVE: In randomized controlled trials, prasugrel and ticagrelor reduced cardiovascular complications in patients with acute coronary syndrome (ACS) compared with clopidogrel. However, limited head-to-head comparisons have been conducted across the three antiplatelet agents using real-world data. The aim of this study was to compare clinical outcomes of three strategies during a 1-year period after percutaneous coronary intervention (PCI). METHODS: Rates of all-cause and acute myocardial infarction (AMI) hospitalizations were compared retrospectively using an insurance claims database. Patients who filled a prescription for an oral antiplatelet agent between September 2011 and December 2013 for post ACS-PCI care were identified. Time to all-cause and AMI hospitalization for a 365-day postdischarge period was compared using Cox proportional hazard models controlling for potential confounders within a propensity score matched cohort. RESULTS: A matched cohort of 9504 clopidogrel, 7128 prasugrel, and 2376 ticagrelor patients was analyzed. The 1-year hazard ratio (HR) for the two newer agents versus clopidogrel was 0.84 (0.78-0.91). The HR for the newer agents versus clopidogrel of admission with AMI as the primary diagnosis was 0.78 (0.61-1.03), and for AMI as any diagnosis during a hospitalization was 0.88 (0.77-1.00). The HR of all-cause admission for ticagrelor versus prasugrel was 0.97 (0.84-1.13), and the HRs of AMI-related admission were not statistically significant between the two agents. Robustness checks across statistical methods to control for potential confounders did not influence the conclusion. CONCLUSION: This real-world study demonstrated that use of the newer agents following PCI was associated with a decrease in all-cause and AMI-related hospitalizations. However, no significant difference was found in the rate of admission between ticagrelor versus prasugrel. Due to concerns regarding statistical power, future studies should examine larger cohorts to obtain more precise estimates for AMI hospitalization for ticagrelor and prasugrel.

Letter--The Authors Respond.

DISCLOSURES: None of the authors of this study are involved in financial or personal relationships with agencies, institutions, or organizations that inappropriately influenced the statistical analysis plan or interpretation of the study results. DiDomenico received an honorarium from Amgen for the preparation of a heart failure drug monograph for Pharmacy Practice News and was a coinvestigator on funded research for the Patient-Centered Outcomes Research Institute. DiDomenico also serves as an advisory board member for a heart failure program at Otsuka America Pharmaceuticals and as an advisory board member at Novartis Pharmaceuticals. Touchette has received unrestricted grant funding from Cardinal Health and Sunovion Pharmaceuticals and has also served as a consultant to, and director of, the American College of Clinical Pharmacy Practice-Based Research Network on a study funded by Pfizer.

Contemporary Trends in Oral Antiplatelet Agent Use in Patients Treated with Percutaneous Coronary Intervention for Acute Coronary Syndrome.

BACKGROUND: Recent trials demonstrated the efficacy of prasugrel and ticagrelor compared with clopidogrel in the reduction of cardiovascular complications in patients with acute coronary syndrome (ACS). However, it is unclear how use of the 3 antiplatelet medications has changed in commercially insured patients since the advent of the new agents. OBJECTIVES: To (a) describe the adoption of prasugrel and ticagrelor in patients who received percutaneous coronary intervention (PCI) for the onset of ACS and (b) explore patient factors associated with the selection of the drug to provide insight into utilization patterns of these antiplatelet agents. METHODS: Patients who received a new dispensing of an antiplatelet agent following a hospitalization for a PCI administered for ACS were identified from insurance claims between 2009 and 2013. Demographics and comorbid conditions were determined based on a 6-month period before the ACS event. Longitudinal trends in antiplatelet agent selection were illustrated using descriptive statistics segmented by month and quarter. Using logistic regressions with stepwise model selection, factors associated with use of the newer medications, as well as with the selection between ticagrelor and prasugrel, were identified. RESULTS: The analysis included 66,335 subjects. The use of clopidogrel decreased from 100% to roughly 65% of total antiplatelet agent use by the end of 2011 and leveled off thereafter. The introduction of ticagrelor in 2011 coincided with a drop in prasugrel initiation from 35%-18% by December 2013. The use of new agents as opposed to use of clopidogrel was associated with younger age (< 65 years), male gender, and a diagnosis of ST-elevation myocardial infarction. In addition, conditions increasing mortality and risk of cardiovascular complication were associated with higher odds of using clopidogrel. The odds of using ticagrelor over prasugrel increased with older age and history of a cerebrovascular event. CONCLUSIONS: In 2013, clopidogrel remained the most prescribed agent. Meanwhile, ticagrelor had gradually replaced a substantial portion of prasugrel initiation. Further investigation into outcomes associated with the newer agents, as well as reasons behind the conservative use of the antiplatelet agents, is warranted. DISCLOSURES: No funding was received for the conduct of this study. DiDomenico received an honorarium from Amgen for the preparation of a heart failure drug monograph for Pharmacy Practice News and was a co-investigator on funded research for the Patient-Centered Outcomes Research Institute. DiDomenico also serves as an advisory board member for a heart failure program at Otsuka America Pharmaceuticals and as an advisory board member at Novartis Pharmaceuticals. Touchette has received unrestricted grant funding from Cardinal Health and Sunovion Pharmaceuticals and has also served as a consultant to and director of the American College of Clinical Pharmacy Practice-Based Research Network on a study funded by Pfizer. None of the authors of this study are involved in financial or personal relationships with agencies, institutions, or organizations that inappropriately influenced the statistical analysis plan or interpretation of the results. Study concept and design were contributed by Kim, Lee, Touchette, and Walton, with assistance from DiDomenico and Ardati. Kim and Lee collected the data, and data interpretation was performed by Lee, DiDomenico, and Ardati, along with Kim and Walton and assisted by Touchette. The manuscript was written by Kim and Walton, with assistance from the other authors, and revised by Kim, Walton, and Lee, with assistance from the other authors.