BACKGROUNDCarbidopa/levodopa enteral suspension (CLES) is indicated for the treatment of advanced Parkinson's disease (aPD) with severe motor fluctuations.OBJECTIVETo determine the cost, quality-adjusted life years (QALY), and cost-effectiveness of CLES compared to the standard-of-care (SoC) for aPD patients in the United States (US), using real-world data.METHODSA published Markov model, comprising of 25 health states and a death state, (defined by a combination of the Hoehn and Yahr scale and waking time spent in OFF-time) was adapted to estimate the benefits for CLES versus oral SoC over a patient's lifetime in the US healthcare setting. Clinical inputs were based on a clinical trial and a registry study; utility inputs were sourced from the Adelphi-Disease Specific Programmes.RESULTSCLES compared to SoC was associated with incremental costs ($1,031,791 vs. $1,025,180) and QALY gain (4.61 vs. 3.76), resulting in an incremental cost-effectiveness ratio of $7711/QALY.CONCLUSIONCLES is a cost-effective treatment for aPD patients with medication resistant motor fluctuations. © 2023 AbbVie, Inc and The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Glecaprevir/pibrentasvir (G/P) was approved on 26 September 2019 by the US Food and Drug Administration for 8-week duration in treatment-naïve (TN) hepatitis C virus (HCV)-infected patients with compensated cirrhosis (CC). Evidence from the EXPEDITION-8 study demonstrated that 8 weeks of G/P achieved a 98% intent-to-treat (ITT) sustained virologic response rate 12 weeks post treatment (SVR12) in 343 TN/CC patients. The aim of this study is to demonstrate the first US real-world effectiveness of G/P 8-week treatment in genotype 1–6 TN/CC HCV patients. Data from 73 TN/CC patients who initiated 8 weeks of G/P treatment between August 2017 and November 2018 were collected electronically from providers and specialty pharmacies of the Trio Health network and analyzed. Cirrhosis was determined by FIB-4 > 5.2 or was physician reported. The primary outcome was Per Protocol (PP) SVR12. The majority (60%) of patients were male, with (mean values): age 59 years, body mass index (BMI) of 30, aspartate aminotransferase (AST) 105, and alanine aminotransferase (ALT) 101 IU/ml. HCV genotypes (GT) were: GT1 81% (59/73), GT2 10% (7/73), GT3 5% (4/73), GT4 3% (2/73), and GT6 1% (1/73). Eight percent (6/73) of patients had concurrent proton pump inhibitor (PPI) use, and 15% (11/72) had a baseline viral load > 6 MM IU/ml. Zero patients discontinued, two patients were reported as lost to follow-up, and there was one virologic failure. PP sustained virologic response at 12 weeks (SVR12) rate was 99% (70/71), and the intent-to-treat (ITT) SVR12 rate was 96% (70/73). Early real-world experience indicates high effectiveness of the 8-week G/P regimen in a diverse treatment-naïve, compensated cirrhotic US population.

Glecaprevir/Pibrentasvir (G/P) is the only regimen approved by the United States Food and Drug Administriation with 8-week treatment duration for chronic HCV genotype (GT) 1-6 infection in treatment naïve (TN), noncirrhotic (NC) patients. Real world effectiveness data from the US are lacking in these populations and the aim of this study was to address this gap. Data from 560 treatment naïve, noncirrhotic patients who initiated 8 weeks of G/P treatment between August 2017 and April 2018 were collected electronically from providers and specialty pharmacies of the Trio Health network. The primary outcome assessed was Per Protocol (PP) sustained virologic response at 12 weeks post-treatment (SVR12). Seventy one percent (397/560) of patients had HCV genotype 1 infection, and 89% (496/560) had a fibrosis score of F0-2. The majority of patients (85%) received care in community practices. The per protocol (PP) SVR12 rate was 99% (537/540) and intent-to-treat (ITT) SVR12 rate was 96% (537/560). Reasons for not achieving SVR12 were virological failure (0.5%, 3/560), lost-to-follow-up (2%, 13/560) and discontinuation (1%, 7/560). Bivariate analyses including gender, practice type, HCV genotype, eGFR, FIB-4, baseline HCV viral load and insurance type did not reveal significant association with SVR12. Reasons for discontinuations were medical care unrelated to treatment, patient choice, physician choice, insurance denial, positive drug/alcohol screening and side effects. 8-week G/P treatment is highly effective in 560 treatment naïve, noncirrhotic patients from the TRIO network with SVR12 rates similar to those as in registration trials.

Although hepatitis C virus (HCV) infection remains a major clinical, economic, and societal burden, the development of curative antiviral therapy may accelerate the path toward elimination. This analysis assessed the progress of United States (US) states towards achieving the World Health Organization’s (WHO) 2030 HCV elimination targets for incidence, mortality, diagnosis, and treatment. A previously published Markov model was used to simulate HCV progression over time to estimate the path to HCV elimination in each state based on prevalence, annual treatment, and diagnosis inputs from two large US laboratory datasets from January 2013 to December 2017. State-specific fibrosis stage restrictions on treatment in 2017 were included. The model estimated the year individual states would meet the WHO targets for diagnosing 90% of the HCV-infected population, treating 80% of the eligible population, reducing new HCV infections by 80%, and reducing HCV-related deaths by 65%. The minimum number of annual treatments needed between 2020 and 2030 to achieve the WHO treatment target was also calculated. Overall, the USA is projected to achieve HCV elimination by 2037, with individual targets related to mortality, diagnosis, treatment, and incidence being achieved by 2020, 2027, 2033, and 2037, respectively. Three states (Connecticut, South Carolina, and Washington) are on track to meet all four elimination targets by 2030, and 18 states are not expected to meet these targets before 2040. The estimated annual number of treatments required during 2020–2030 nationally to reach the WHO treatment target is 173,514. With the exception of three states, the USA is not on target to meet the WHO 2030 elimination targets and 35% are off track by 10 years or more. Strategies must be implemented to reduce overall prevalence by preventing new infections, increasing rates of screening, improving linkage to care, and implementing unfettered access to curative therapy.

Chronic infection with hepatitis C virus (HCV) is a leading cause of liver disease and infectious disease deaths. While recent and emerging treatment options for HCV patients have enabled higher rates of sustained virologic response (SVR), the demographic, clinical, geographic, and payer characteristics of the estimated 3.4 million chronic HCV patients in the USA are poorly understood. The goal of this study was to create a dataset describing the current HCV patient landscape in the USA. Data from two large national laboratory companies representing the majority of US patients screened for HCV antibody and/or tested for HCV RNA from 2013 through 2016 were organized into the present study dataset. Age, gender, payer channel, 3-digit ZIP code and ordering physician specialty, and 3-digit ZIP code information were available for all patients. Among RNA-positive patients, additional clinical characteristics included HCV genotype, fibrosis stage, renal function, and HIV status. Initiating treatment and attaining cure were imputed using data-driven algorithms based on successive RNA viral load measurements. The number of RNA-positive HCV patients increased from 200,066 patients in 2013 to 469,550 in 2016. The availability of clinical data measurements and rates of treatment initiation increased over the study period, indicating improved care engagement for HCV patients. Treatment and cure rates varied by age, disease severity, geographic location, and payer channel. Sensitivity and specificity of the cure prediction algorithms were consistently above 0.90, validating the robustness of the data imputation approach. This is the largest, most comprehensive dataset available to describe the current US HCV patient landscape. Our results highlight that the epidemiology of HCV is evolving with an increasing number of patients who are younger and have milder disease than described in previous years. Results of this study should help guide efforts toward the elimination of HCV in this country. Future work will focus on factors associated with varying treatment and cure patterns and describing recent changes in the HCV patient landscape. AbbVie. Plain language summary available for this article.

Purpose: To determine associations between patient characteristics and adjudicated PPI use to determine predictors of twice daily (BID) PPI dosing. This study investigated the unique patient characteristics associated with Medicare and Medicaid patients receiving BID proton-pump inhibitors (PPIs). Methods: This was a cross-sectional, retrospective database analysis of an adult Medicare and/or Medicaid population (2 California Managed Care Plans) on BID PPIs vs. once daily (QD) PPIs as of June 30, 2010 (index date). BID users were those having an average daily consumption (DACON) >1.5 doses/day, while QD users had a PPI DACON of ≤1.5 doses/day. The period reviewed was 30 months (through January 1, 2008). Inclusion criteria required being a current PPI user and at least 20 years of age on June 30, 2010. Main exclusion criteria were those receiving Helicobacter pylori eradication therapy. Logistic regression analysis was used to identify characteristics of PPI BID dosing among PPI users. Results: Of the 6,568 patients on a PPI, 262 (4.0%) were prescribed BID therapy (prescription or adjudicated OTC included). The mean age at the index date was significantly higher in the BID group than the QD group (58.7 years vs. 56.0 years, respectively; P=0.005). The majority of the population was female, (69.5% vs. 64.8% in the BID and QD groups, respectively). The mean duration of PPI therapy was 418 days for the BID group versus 301 days for the QD group (P<0.001). The average cost per 30 days was significantly higher if patients used multiple unique PPIs vs. one PPI ($74 vs. $32 for QD group and $115 vs. $58 for BID group, respectively). In addition, 19.5% of patients on BID therapy who had previously been on QD therapy (n=133) had changed PPIs prior to switching to a BID regimen. Based on the adjusted odds ratios, the characteristics associated with BID therapy were: Medicare vs. Medicaid recipients, patients on a PPI than ≥12 months vs. <6 months, use of lansoprazole vs. omeprazole, prior BID dosing regimen (prior to their most recent continuous PPI episode), and using more than 20 unique medications vs. 1-10 medications during the last 12 months of the study. Conclusion: The results of this small study suggest that patients on BID therapy were older, female and had more co-morbidities than daily PPI users. BID dosing was associated with longer PPI duration of therapy, higher costs, and an increased prevalence in Medicare patients compared to Medicaid. Disclosure: Dr. Olson is am employee of Lumetra; Lumetra received study funding from Takeda Pharmaceuticals USA, Inc. Dr. Chan is am employee of Touro University and has no financial disclosures. Dr. Trask - Employee: Takeda Pharmaceuticals USA, Inc. Dr Strezewski was an employee of Takeda Pharmaceuticals USA, Inc. at the time the study was conducted. This research was supported by an industry grant from Takeda Pharmaceuticals USA, Inc.

genes, and other genes which have not yet been previously described to be induced by these drugs.CONCLUSION Genotyping of patients and the ethnic population the patients belong to, is important to reach efficacious treatment and to reduce toxic effects.This study provides helpful information for the prediction of appropriate usage of 5-ASA and AZA in IBD patients.