Indirect Treatment Comparisons in Healthcare Decision Making: A Targeted Review of Regulatory Approval, Reimbursement, and Pricing Recommendations Globally for Oncology Drugs in 2021-2023.

Introduction: Indirect treatment comparisons (ITCs) evaluate novel treatments compared to appropriate comparators when direct evidence is unavailable or infeasible. The objective of this study was to highlight the prevalence of different ITC methods in oncology drug submissions and to provide insights into how ITCs have been used in recent regulatory approval, reimbursement recommendations, or pricing decisions across various regions and diverse assessment frameworks.

Methods: A targeted literature review was conducted to identify assessment documents for oncology drug submissions that included ITCs.

Estimating cure proportion in cancer clinical trials using flexible parametric cure models.

Background: In the context of survival analysis in recent cancer clinical trials, there has been an increasing interest in the proportion of patients who are not susceptible to the event, known as the cure proportion. To estimate it, cure models implicitly specify the time point when patients are considered cured, although the impact of the cure point position on cure proportion estimates is unclear.

Methods: Sensitivity of cure proportion estimates to the knot number and placement in flexible parametric cure models was examined using data from a clinical trial of CheckMate 141, an immuno-oncology compound.

Predictors of discontinuation of osteoporosis treatment: sub-analysis of the Japanese osteoporosis intervention trial-05 (JOINT-05).

Introduction: To identify predictors of discontinuing treatment with teriparatide (TPTD) and alendronate (ALN), data from a randomized, controlled trial (JOINT-05) involving postmenopausal Japanese women at high risk of fracture were re-analyzed.

Materials And Methods: Participants received sequential therapy with once-weekly TPTD for 72 weeks followed by ALN for 48 weeks (TPTD-ALN group) or monotherapy with ALN for 120 weeks (ALN group). Background data including comorbidities, fracture prevalence, cognitive function, quality of life, activities of daily living, bone metabolism parameters, and nutrient intake were collected.

Clarifying Causal Effects of Interest and Underlying Assumptions in Randomized and Nonrandomized Clinical Trials in Oncology Using Directed Acyclic Graphs and Single-World Intervention Graphs.

Recent clinical trials in oncology have used increasingly complex methodologies, such as causal inference methods for intercurrent events, external control, and covariate adjustment, posing challenges in clarifying the estimand and underlying assumptions. This article proposes expressing causal structures using graphical tools-directed acyclic graphs (DAGs) and single-world intervention graphs (SWIGs)-in the planning phase of a clinical trial. It presents five rules for selecting a sufficient set of adjustment variables on the basis of a diagram representing the clinical trial, along with three case studies of randomized and single-arm trials and a brief tutorial on DAG and SWIG.

Rare Event Approximation Between Subdistribution Hazard Ratio and Cause-specific Hazard Ratio in Survival Analysis With Competing Risks.

Background: Despite the fact that competing risks are inevitable in epidemiological and clinical studies, distinctions between the hazard ratio estimated by handling competing risks as censoring and the subditribution hazard ratio are often overlooked.

Methods: We derived quantitative relationships between subdistribution hazard ratio and cause-specific hazard ratio and derive an approximate calculation method to transform the two into each other. Numerical examinations of hypothetical six scenarios and published information of a randomized clinical trial of cholesterol-lowering therapy and a registry of acute myeloid leukemia were provided.

A crossover comparison of patient satisfaction with two teriparatide regimens: primary results of the Japanese Osteoporosis Intervention Trial 06 (JOINT-06).

Introduction: This study aimed to compare treatment satisfaction with two dosing regimens (two teriparatide [TPTD] self-injection systems) in osteoporosis patients at high risk of fracture.

Materials And Methods: In this open-label crossover randomized trial comparing self-injected once-daily (1/D)-TPTD with self-injected twice-weekly (2/W)-TPTD, three satisfaction variables were evaluated by questionnaire for 2 years. The primary endpoint was overall satisfaction and secondary endpoints were satisfaction with treatment effectiveness and with utility of the self-injection device.

Achieving osteoporosis treat-to-target goals with teriparatide or alendronate: sub-analysis of Japanese Osteoporosis Intervention Trial-05 (JOINT-05).

Introduction: The purpose of this study was to evaluate whether bone mineral density (BMD) ≥ -2.5 SD could be used as the treat-to-target (T2T) goal when treating osteoporosis with teriparatide (TPTD) and alendronate (ALN), and to investigate the relationship with incident vertebral fracture by re-analyzing data from a randomized, controlled trial (JOINT-05) involving postmenopausal Japanese women at high fracture risk.

Materials And Methods: Participants received sequential therapy with once-weekly TPTD for 72 weeks, followed by ALN for 48 weeks (TPTD-ALN group) or ALN monotherapy for 120 weeks (ALN group).

Landscape of driver mutations and their clinical effects on Down syndrome-related myeloid neoplasms.

Transient abnormal myelopoiesis (TAM) is a common complication in newborns with Down syndrome (DS). It commonly progresses to myeloid leukemia (ML-DS) after spontaneous regression. In contrast to the favorable prognosis of primary ML-DS, patients with refractory/relapsed ML-DS have poor outcomes.

Decoupling power and type I error rate considerations when incorporating historical control data using a test-then-pool approach.

To accelerate a randomized controlled trial, historical control data may be used after ensuring little heterogeneity between the historical and current trials. The test-then-pool approach is a simple frequentist borrowing method that assesses the similarity between historical and current control data using a two-sided test. A limitation of the conventional test-then-pool method is the inability to control the type I error rate and power for the primary hypothesis separately and flexibly for heterogeneity between trials.

Correction: Electronic Health Record-Nested Reminders for Serum Lithium Level Monitoring in Patients With Mood Disorder: Randomized Controlled Trial.

[This corrects the article DOI: 10.2196/40595.].

The association between intraoperative anesthesia methods used during gastric cancer surgery and long-term mortality: A retrospective observational study using a Japanese claims database.

Purpose: Various basic and clinical studies have investigated the association between the types of anesthetic agents and prognosis. However, the results have varied among studies and remain controversial. In the present study, we aimed to investigate whether the risk of all-cause mortality differs between inhaled or intravenous anesthetics in patients with gastric cancer undergoing gastrectomy.

Reliability of early stage symptoms/clinical findings of osteonecrosis of the jaw: Japanese Osteoporosis Intervention Trial-05 (JOINT-05).

Introduction: To investigate the differences in the incidence rates of suspected stage 0/1 osteonecrosis of the jaw (ONJ) and incidence risk of relevant clinical findings of suspected stage 0 ONJ between patients treated with sequential therapy comprising weekly teriparatide for 72 weeks followed by alendronate for 48 weeks vs. those who received monotherapy with alendronate for 120 weeks.

Materials And Methods: Suspected stage 0/1 ONJ was defined by non-specific symptoms.

Employment Status of Patients With Colorectal Cancer After Surgery: A Multicenter Prospective Cohort Study in Japan.

Background: Because of improved survival rates, patients with colorectal cancer may try to return to work. Many countries, however, have limited knowledge of the employment status of these patients.

Objective: To explore the employment status of patients with colorectal cancer after surgery in Japan and the risk factors affecting the same.

Assessment of myopic rebound effect after discontinuation of treatment with 0.01% atropine eye drops in Japanese school-age children.

Purpose: Having previously demonstrated the efficacy of 0.01% atropine eye drops for inhibiting progression of childhood myopia, we conducted additional analyses to assess post-treatment changes in myopia progression.

Study Design: Analysis of follow-up data from a previously reported randomized controlled trial METHODS: A mixed-effects model was used to compare intergroup changes in spherical equivalent (SE) and axial length (AL) at 1 month and 12 months after discontinuation of 2-year treatment with atropine or placebo in 167 school-age children.

Relationship between the magnitude of body mass index reductions and remission in patients with type 2 diabetes in real world settings: Analysis of nationwide patient registry in Japan (JDDM74).

Aims: To determine the association between the magnitude of weight loss and incidence of remission according to baseline characteristics in patients with diabetes in clinical settings.

Methods: In total, 39 676 Japanese patients with type 2 diabetes aged ≥18 years with glycated haemoglobin (HbA1c) ≥6.5% and/or glucose-lowering drug prescription were identified from databases of specialists' clinics from 1989 and followed until September 2022.

G-estimation of structural nested mean models for interval-censored data using pseudo-observations.

Two large-scale randomized clinical trials compared fenofibrate and placebo in diabetic patients with pre-existing retinopathy (FIELD study) or risk factors (ACCORD trial) on an intention-to-treat basis and reported a significant reduction in the progression of diabetic retinopathy in the fenofibrate arms. However, their analyses involved complications due to intercurrent events, that is, treatment-switching and interval-censoring. This article addresses these problems involved in estimation of causal effects of long-term use of fibrates in a cohort study that followed patients with type 2 diabetes for 8 years.

Incidence and predictors of remission and relapse of type 2 diabetes mellitus in Japan: Analysis of a nationwide patient registry (JDDM73).

Aims: To determine the incidence of remission and 1-year relapse from remission and associated factors in patients with type 2 diabetes.

Materials And Methods: A total of 48 320 Japanese patients with type 2 diabetes aged ≥18 years, with glycated haemoglobin (HbA1c) levels ≥48 mmol/mol (6.5%) and/or glucose-lowering drug prescription, were identified from databases of specialist clinics from 1989 and followed until September 2022.

Electronic Health Record-Nested Reminders for Serum Lithium Level Monitoring in Patients With Mood Disorder: Randomized Controlled Trial.

Background: Clinical guidelines recommend regular serum lithium monitoring every 3 to 6 months. However, in the real world, only a minority of patients receive adequate monitoring.

Objective: This study aims to examine whether the use of the electronic health record (EHR)-nested reminder system for serum lithium monitoring can help achieve serum lithium concentrations within the therapeutic range for patients on lithium maintenance therapy.