Real-World Treatment Patterns and Outcomes Following First-Line Pertuzumab and Trastuzumab Among Patients with HER2+ Metastatic Breast Cancer.

Introduction: Many patients with human epidermal growth factor receptor-2-positive metastatic breast cancer (HER2+ mBC) require subsequent lines of therapy (LOTs) after being treated with pertuzumab and trastuzumab-based regimens in the first line (1L). Although the efficacy of the second-line (2L) therapies has been demonstrated in clinical trials, the real-world effectiveness of these treatments is understudied. This retrospective cohort study assessed the real-world treatment patterns and outcomes for patients with HER2+ mBC following 1L therapy with pertuzumab and trastuzumab-based regimens in the United States (US) during 2015-2019.

Treatment Patterns and Adverse Event-Related Hospitalization Among Patients with Epidermal Growth Factor Receptor (EGFR)-Mutated Metastatic Non-small Cell Lung Cancer After Treatment with EGFR Tyrosine Kinase Inhibitor and Platinum-Based Chemotherapy Regimens.

Background: Epidermal growth factor receptor-tyrosine kinase inhibitors (EGFR TKIs) are established first-line treatments among patients with metastatic non-small cell lung cancer harboring EGFR-sensitizing mutations. Upon EGFR TKI resistance, there are scant data supporting a standard of care in subsequent lines of therapy.

Objective: We aimed to characterize real-world treatment patterns and adverse events associated with hospitalization in later lines of therapy.

Frequency and clinicoeconomic impact of delays to definitive diagnosis of obstructive hypertrophic cardiomyopathy in the United States.

Aims: The diagnostic history in the years leading up to the definitive diagnosis of patients with obstructive hypertrophic cardiomyopathy (HCM) has not been studied.

Methods: Patients with a delay in the definitive diagnosis of obstructive HCM from January 2009 to March 2019 were identified in the US IBM MarketScan Commercial and Medicare Supplemental Databases if they had an alternative diagnosis indicating a misdiagnosis during the 24 months before the definitive obstructive HCM diagnosis. Resource use and costs associated with the delay were estimated during the same period.

Clinical Outcomes, Resource Utilization, and Treatment Over the Disease Course of Symptomatic Obstructive Hypertrophic Cardiomyopathy in the United States.

We sought to describe the clinical outcomes, resource utilization, and treatment options for patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) over the course of their disease. Adults with obstructive HCM who were symptomatic were identified from the IBM MarketScan Commercial and Medicare supplemental database (January 2009 to March 2019). The index date was the initial obstructive HCM diagnosis date.

Event-Free Survival as a Surrogate for Overall Survival in Gastric and Gastroesophageal Junction Adenocarcinoma: A Meta-analysis in the Neoadjuvant ± Adjuvant Setting.

Purpose: This study assessed the trial-level association between event-free survival (EFS) and overall survival (OS) in gastric or gastroesophageal junction (GEJ) adenocarcinoma in the neoadjuvant ± adjuvant settings.

Experimental Design: A systematic literature review was conducted to identify randomized controlled trials (RCT) that evaluated neoadjuvant therapies with or without adjuvant therapies for gastric or GEJ adenocarcinoma. A meta-analysis was performed using weighted linear regressions of the treatment effect of OS on the treatment effect of EFS.

Advancing the development of real-world data for healthcare research in China: challenges and opportunities.

Various real-world data (RWD) sources have emerged in China with the intention of generating real-world evidence (RWE) that can be used in clinical and regulatory decision-making. Despite these efforts, significant barriers remain that hinder high-quality healthcare research. A workshop with 30 representatives from healthcare research agencies, technology companies focused on healthcare big data and pharmaceutical companies was held in December 2020 to identify strategies to overcome the barriers associated with the usability and quality of RWD in China.

The challenges and opportunities in using real-world data to drive advances in healthcare in East Asia: expert panel recommendations.

Objective: To provide recommendations for overcoming the challenges associated with the generation and use of real-world evidence (RWE) in regulatory approvals, health technology assessments (HTAs), and reimbursement decision-making in East Asia.

Methods: A panel of experts convened at the International Society for Pharmacoeconomics and Outcomes Research Asia Pacific 2020 congress to discuss the challenges limiting the use of RWE in healthcare decision-making and to provide insights into the perspectives of regulators, HTA agencies, the pharmaceutical industry, and physicians in China, Japan, and Taiwan. A nonsystematic literature review was conducted to expand on the themes addressed.

Cost-Effectiveness Analysis of Venetoclax in Combination with Azacitidine Versus Azacitidine Monotherapy in Patients with Acute Myeloid Leukemia Who are Ineligible for Intensive Chemotherapy: From a US Third Party Payer Perspective.

Objectives: Using individual patient-level data from the phase 3 VIALE-A trial, this study assessed the cost-effectiveness of venetoclax in combination with azacitidine compared with azacitidine monotherapy for patients newly diagnosed with acute myeloid leukemia (AML) who are ineligible for intensive chemotherapy, from a United States (US) third-party payer perspective.

Methods: A partitioned survival model with a 28-day cycle and three health states (event-free survival (EFS), progressive/relapsed disease, and death) was developed to estimate costs and effectiveness of venetoclax + azacitidine versus azacitidine over a lifetime (25-year) horizon. Efficacy inputs (overall survival (OS), EFS, and complete remission (CR)/CR with incomplete marrow recovery (CRi) rate) were estimated using VIALE-A data.

Treatment Changes, Healthcare Resource Utilization, and Costs Among Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy: A Claims Database Study.

Introduction: There is limited evidence on therapies for obstructive hypertrophic cardiomyopathy (HCM), and data regarding treatment patterns and cost are scarce. This study assessed treatment patterns and economic outcomes in patients with symptomatic obstructive HCM.

Methods: Adults with symptomatic obstructive HCM as per study design and treated with pharmacotherapies [beta blockers (BBs), calcium channel blockers (CCBs), BB + CCB, or disopyramide] or procedures (septal reduction therapy, heart transplantation, implantable cardioverter-defibrillator, and pacemaker implantation) were identified from the IBM® MarketScan® Commercial and Medicare Supplemental database (January 2009-March 2019).

Projecting the Long-term Clinical Value of Mavacamten for the Treatment of Obstructive Hypertrophic Cardiomyopathy in the United States: An Assessment of Net Health Benefit.

Purpose: The aim of the study was to project the long-term net health benefits of mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States.

Methods: A Markov model with 4 mutually exclusive health states (New York Heart Association [NYHA] functional classes I, II, and III/IV and death) was developed to project the life-years (LYs) and quality-adjusted life-years (QALYs) over a lifetime horizon for patients with symptomatic obstructive HCM receiving mavacamten with or without β-blocker (BB) or calcium channel blocker (CCB) monotherapy or placebo with or without BB or CCB monotherapy. The model simulated a patient cohort with a starting age of 59 years and 41% women.

Assessing health-related quality-of-life in patients with symptomatic obstructive hypertrophic cardiomyopathy: EQ-5D-based utilities in the EXPLORER-HCM trial.

Aims: To assess the effects of mavacamten on health-related quality-of-life (HRQoL) in symptomatic obstructive hypertrophic cardiomyopathy (HCM) and estimate health utilities by New York Heart Association (NYHA) functional class.

Materials And Methods: Patients with symptomatic obstructive HCM were randomized to 30 weeks of mavacamten or to placebo treatment, with or without beta-blocker or calcium channel blocker monotherapy, in EXPLORER-HCM (ClinicalTrials.gov identifier: NCT03470545).

Real-World Data for Healthcare Research in China: Call for Actions.

Objectives: This study aimed to provide an overview of major data sources in China that can be potentially used for epidemiology, health economics, and outcomes research; compare them with similar data sources in other countries; and discuss future directions of healthcare data development in China.

Methods: The study was conducted in 2 phases. First, various data sources were identified through a targeted literature review and recommendations by experts.

Conceptual Framework and Methodological Challenges for Modeling Effectiveness in Oncology Treatment Sequence Models.

In this review, we summarize the challenges faced by existing oncology treatment sequence decision models and introduce a general framework to conceptualize such models. In the proposed framework, patients with cancer receive at least two lines of therapy (LOTs) followed by palliative care throughout their lifetime. Patients cycle through progression-free and progressive disease health states in each LOT before death.

Reasons for canakinumab initiation among patients with periodic fever syndromes: a retrospective medical chart review from the United States.

Background: Although canakinumab has demonstrated efficacy in multiple trials in patients with periodic fever syndromes (PFS), the evidence on initiation of canakinumab among PFS patients in real world setting is not well understood. We aimed to characterize the reasons for canakinumab initiation among patients with PFS, specifically, cryopyrin-associated periodic syndrome (CAPS), hyperimmunoglobulin D syndrome/mevalonate kinase deficiency (HIDS/MKD), TNF receptor-associated periodic syndrome (TRAPS) and familial Mediterranean fever (FMF).

Methods: Physicians retrospectively reviewed the medical charts of PFS patients prescribed canakinumab between 2016 and 2018.

Clinical and economic burden of obstructive hypertrophic cardiomyopathy in the United States.

Aims: Obstructive hypertrophic cardiomyopathy (oHCM) is a disease of the cardiomyocyte in which dynamic left ventricular outflow track obstruction may lead to heart failure, valvular disease, and sudden death. Little is known about healthcare resource utilization (HRU) and costs associated with oHCM. This study investigated the clinical and economic burden of oHCM in patients with or without symptoms associated with oHCM.

Characteristics and treatment patterns of relapsed/refractory diffuse large B-cell lymphoma in patients receiving ≥3 therapy lines in post-CAR-T era.

Objective: Several novel treatments have been approved for relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) since chimeric antigen receptor T-cell (CAR-T) therapy became available. The objective of this study was to describe characteristics and treatment patterns in patients with R/R DLBCL post-CAR-T approval.

Methods: Adult patients with R/R DLBCL who initiated third-line treatment or later (3 L+) since 18 October 2017 were identified using administrative claims from IQVIA PharMetrics Plus (1 January 2014-31 March 2020).

Predictors and timing of response to teduglutide in patients with short bowel syndrome dependent on parenteral support.

Background And Aims: This study aimed to identify predictors and estimate time to teduglutide response among adult patients with short bowel syndrome with intestinal failure (SBS-IF) dependent on parenteral support (PS).

Methods: Post-hoc analysis was performed on individual patient data from teduglutide-treated patients in the phase III teduglutide trial STEPS and the STEPS-2 extension. Response was defined as ≥20% PS volume reduction from baseline for two consecutive visits.

Cost-effectiveness analysis of ceritinib vs. crizotinib in previously untreated anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) in Hong Kong.

Introduction: Lower-dose ceritinib (450 mg) once-daily with food was approved in 2018 in Hong Kong (HK) for first-line treatment of patients with anaplastic lymphoma kinase-positive (ALK +) advanced non-small cell lung cancer (NSCLC). This study examined the cost-effectiveness of ceritinib vs. crizotinib in the first-line treatment of ALK + NSCLC from a HK healthcare service provider's or government's perspective.

An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies.

Introduction: Haemophilia A (HA) is a rare X chromosome-linked bleeding disorder resulting in missing or defective clotting factor VIII (FVIII) and causes large disease burden.

Aim: As a member of World Federation of Hemophilia, China seeks to understand the current epidemiology, disease profile and treatment landscape of patients with HA through the Hemophilia Treatment Center Collaboration Network of China (HTCCNC).

Methods: The HTCCNC enabled data collection on patients with HA from 166 member hospitals (2007-2019) across China.

The cost-effectiveness of low-dose budesonide as a Step 2 treatment for pediatric asthma in China.

To compare the cost-effectiveness of low-dose budesonide versus montelukast among patients aged 1-5 years from a Chinese patient and healthcare payer perspective. A Markov model based on exacerbation states was developed. Exacerbation was defined as the need for rescue therapy (mild exacerbation) or hoscopitalization (moderate-to-severe exacerbation).

Clinical and Economic Outcomes of Erythropoiesis-Stimulating Agent Hyporesponsiveness in the Post-Bundling Era.

Rationale & Objective: Since the change in erythropoiesis-stimulating agent (ESA) labeling and bundling of dialysis services in the United States, few studies have addressed the clinical importance of ESA hyporesponsiveness and none have considered health care resource use in this population. We aimed to further explore ESA hyporesponsiveness and its consequences.

Study Design: Retrospective observational cohort study.

Cost-effectiveness analysis of double low-dose budesonide and low-dose budesonide plus montelukast among pediatric patients with persistent asthma receiving Step 3 treatment in China.

Aims: For children aged 1-5 years with persistent asthma, double low-dose inhaled corticosteroids (ICS) are recommended as the preferred Step 3 treatment and low-dose ICS plus leukotriene receptor antagonists (LTRA) as an alternative. Budesonide inhalation suspension (0.5 mg daily) and montelukast (4.

Association of Pathologic Complete Response with Long-Term Survival Outcomes in Triple-Negative Breast Cancer: A Meta-Analysis.

Pathologic complete response (pCR) following neoadjuvant therapy has been associated with improved event-free survival (EFS) and overall survival (OS) in early-stage breast cancer. The magnitude of this association varies by breast cancer subtype, yet further research focusing on subtype-specific populations is limited. Here we provide an updated and comprehensive evaluation of the association between pCR and survival outcomes in triple-negative breast cancer (TNBC).