Evidence regarding the effectiveness and safety of PD-1 inhibitor rechallenge in older patients with advanced non-small-cell lung cancer (NSCLC) previously exposed to immunotherapy remains limited. This study evaluated the effectiveness and safety of PD-1 inhibitor monotherapy rechallenge in patients aged ≥ 60 years with advanced NSCLC who had previously received immunotherapy. This single-center retrospective cohort study included 56 patients aged ≥ 60 years with advanced NSCLC who received PD-1 inhibitor monotherapy rechallenge at the Department of Oncology Radiotherapy at the Guilin People’s Hospital between January 2019 and March 2025. Objective response rate (ORR) and disease control rate (DCR) were assessed per RECIST 1.1 criteria. Progression-free survival (PFS) and overall survival (OS) were estimated by the Kaplan–Meier method. Treatment-related adverse events (TRAEs) were graded by Common Terminology Criteria for Adverse Events (CTCAE) v5.0. In the full analysis population of 56 patients with five non-evaluable patients counted as non-responders, the ORR and DCR were 21.4
In vancomycin treatment, follow-up therapeutic drug monitoring (TDM) within 1 week after the initial TDM is recommended in patients with dose change after the initial TDM or high risk of vancomycin-induced nephrotoxicity (VIN); however, the effectiveness of follow-up TDM implementation remains unclear. This study aimed to delineate outcomes of follow-up TDM after the initial assessment under real-world conditions in Japan and clarify its association with VIN. Retrospective data analysis was conducted using a Japanese health insurance claims database. Approval was granted by the Ethics Committee of Osaka Medical and Pharmaceutical University (protocol number: 2022–105). Adult patients who received intravenous vancomycin, excluding those without a dose change after the initial TDM and without risk of VIN between January 2020 and December 2021, were included. Follow-up TDM was defined as conducting TDM within 1 week after the initial assessment. The association between performing follow-up TDM and the occurrence of VIN was assessed in patients with dose change after the initial TDM or high risk of VIN. Among 492 patients who underwent the initial TDM, 403 (81.9
Minocycline, a second-generation tetracycline, is widely used for the management of several dermatologic and infectious conditions. Large-scale real-world studies demonstrating its tolerability, utilization patterns, and effectiveness in Indian clinical practice remain limited. The aim of this study was to evaluate real-world tolerability, utilization patterns, and effectiveness of oral minocycline formulations. This retrospective, real-world, observational, multicentric electronic medical records (EMR)-based study analyzed anonymized patient data retrieved from the HealthPlix EMR database between January 2017 and February 2023. Patients of either gender aged ≥12 years prescribed oral minocycline with at least one follow-up visit were included. The primary endpoint was tolerability, assessed as the proportion of patients with physician-documented complaints. Secondary outcomes included assessment of prescribing patterns and effectiveness. A total of 2161 patients from 189 centers across India met the eligibility criteria. The mean age ± standard deviation (SD) was 30.15 ± 12.50 years, and 62.56
Chronic non-cancer pain is a common reason for consultation in primary care. Although opioid therapy may be considered a treatment option for selected patients, guideline-recommended discontinuation can be challenging for both patients and general practitioners. This study investigated predictors of opioid discontinuation in primary care. We analyzed German claims data (2021) from patients aged ≥ 18 years with at least one oral or transdermal opioid prescription from general practitioners in the first three quarters of 2021. Exclusion criteria were malignant diseases or palliative care. Opioid discontinuation was operationalized as the absence of an opioid prescription in the fourth quarter. Multivariable logistic regression was used to examine associations between prespecified predictors and the absence of an opioid prescription in the fourth quarter. The cohort included 94,125 patients (mean age 65 years; 62
As the use of real-world evidence (RWE) expands, evaluating whether Japanese real-world data (RWD) can reproduce clinical trial findings is crucial. This study aimed to determine whether pseudonymized medical information collected under the Next-Generation Medical Infrastructure Act could reconstruct a patient cohort comparable to the Japanese subgroup of the OAK trial and to evaluate progression-free survival (PFS) and overall survival (OS) in patients with non-small cell lung cancer (NSCLC) treated with atezolizumab. We conducted a historical cohort study using the Millennium Medical Record database. Medical information collected between 1 October 2015, and 31 October 2022 was utilized, and the study itself was conducted from September 2023 to February 2024. Patients treated with atezolizumab for NSCLC were identified, and eligibility criteria from the OAK trial were applied. Effectiveness outcomes were extracted from unstructured electronic medical record text by trained abstractors through manual review of the medical records, ensuring robustness of the assessments. A total of 75 patients were analyzed. The median PFS was 3.5 months (95
Calcitonin gene-related peptide monoclonal antibodies (CGRP mAbs) are an effective preventive treatment for migraine. However, their population-level utilization patterns and treatment gaps are underreported in Japan. This analysis of the ObserVational survey of the Epidemiology, tReatment, and Care Of MigrainE (OVERCOME [Japan]) 2nd study, evaluated utilization patterns of CGRP mAbs and treatment gaps, and identified factors associated with CGRP mAb treatment experience among CGRP mAb-eligible individuals with migraine in Japan. This analysis of a nationwide, population-based, cross-sectional online survey included adults with migraine. Respondents were further classified according to CGRP mAb eligibility (based on the guidelines for the promotion of optimal use). Both CGRP mAb-eligible and ineligible individuals were stratified by experience with CGRP mAbs. CGRP mAb-experienced respondents were further stratified into “current CGRP mAb” and “discontinued CGRP mAb” subgroups. Respondents reported their CGRP mAb utilization pattern, headache perceptions, attitude, and barriers to treatment initiation. Multivariate logistic regression was performed to identify the factors associated with CGRP mAb treatment experience among CGRP mAb-eligible respondents. Among the 19,590 respondents with migraine, 1484 (7.6
Type 2 diabetes mellitus has multiple treatment options and high healthcare costs. In Japan, DPP-4 inhibitors have been widely used, but since 2022 the pharmacotherapy algorithm has shifted: DPP-4 inhibitors and biguanides for BMI < 25 kg/m ^2 , and biguanides and SGLT2 inhibitors for BMI ≥ 25 kg/m ^2 . However, real-world factors influencing prescribing decisions between biguanides and SGLT2 inhibitors remain unclear. This study investigates factors influencing the choice between biguanides and SGLT2 inhibitors in clinical practice. We also compare glycemic efficacy and medication persistence while accounting for baseline characteristics. This retrospective cohort study used the Millennium Medical Record Database, primarily capturing care from large hospitals. Adults aged ≥ 18 years who received a first prescription of a biguanide or a SGLT2 inhibitor between July 2019 and April 2023 were included. Patients were categorized as 1st-line or 2nd-line therapy. Confounding was addressed using coarsened exact matching with classification tree modeling. HbA1c changes were analyzed using mixed models for repeated measures, and persistence using Kaplan–Meier methods. A total of 1925 patients were included. In the 1st-line cohort (n = 1440), key selection factors were diuretic use, lipid-modifying agents, and baseline HbA1c. At 24 weeks, HbA1c decreased in both groups (biguanide: −1.37 percentage points, 95
Potential Drug–drug interactions (pDDIs) are a significant cause of adverse drug events and can compromise treatment outcomes, especially in hospitalized patients who are prescribed multiple medications. Pulmonary patients are especially vulnerable due to polypharmacy and the presence of multiple co-morbid conditions. This study aimed to determine the prevalence of pDDIs and identify associated risk factors among hospitalized pulmonary patients. A cross-sectional study was conducted among pulmonary patients admitted to a tertiary care hospital in Nepal between July 2024 and December 2025. Patients with a hospital stay of ≥ 24 h and receiving two or more medications were included. Data on socio-demographics, clinical conditions, and medications were collected using a structured form. pDDIs were identified using Lexicomp and IBM Micromedex. Descriptive statistics, bivariate analysis, and binary logistic regression were performed using SPSS V16, with p < 0.05 considered statistically significant. Out of 377 patients who met the inclusion criteria, Lexicomp and Micromedex identified 36.1
Up to half of cancer patients treated with immune checkpoint inhibitors (ICIs) develop immune-related adverse events (irAEs). However, the long-term consequences of irAEs remain poorly characterized. This study investigated the likelihood of chronic irAEs, challenges related to their corticosteroid treatment and clinical workload associated with their management. We retrospectively analyzed 230 patients who received ICIs at Kuopio University Hospital, Finland, between the years 2015 and 2024. IrAEs were chronic in 60.9
The global use of real-world data (RWD) for drug approval and post-marketing surveillance has increased significantly. During 2023 and 2024, the US Food and Drug Administration issued industry guidance, and the European Medicines Agency released real-world evidence framework reports to support regulatory decision making. In Japan, the Pharmaceuticals and Medical Devices Agency promotes the use of RWD, with collective efforts from the government and related industries. This report focuses on electronic health record (EHR) databases in Japan, aiming to (1) organize the characteristics of data generated from such databases, and (2) identify the challenges when using EHR databases for regulatory decision making. This report conducted an internet-based Google search using the keywords ‘electronic medical record database’ with the condition that the results include any of the keywords ‘Japan’ or ‘real-world data’. Online interviews were conducted with representatives from eight EHR database companies identified as being utilized in Japan, focusing on the characteristics of each EHR database, challenges in their utilization, and future perspectives. The characteristics and limitations of current EHR databases were summarized; they offer broad coverage in terms of patient backgrounds and easier access to clinical laboratory values but are unable to track patients who transfer between hospitals due to lack of data linkage, and there may be loss of information during data abstraction. Data collection methods were mostly retrospective. Critically, there is no overall standardization between the EHR databases. Currently, EHR studies are primarily observational to verify efficacy and safety in real clinical practice and are gradually being used for post-marketing surveillance to support regulatory decision making in Japan. Addressing operational, technical, and methodological challenges in using EHR databases for regulatory approval applications requires efforts from multiple stakeholders, including industry, academia, and government. Real-world data (RWD) are information collected from everyday medical care, not from clinical trials. Around the world, RWD are increasingly being used to verify the effectiveness and safety of medicines. In 2023 and 2024, the US and Europe published regulations to guide this process. In Japan, the government and industry are also working to utilize RWD for drug approval and safety monitoring. This topic matters to the general public because better use of RWD can lead to safer medicines, faster access to new treatments, and healthcare decisions that more accurately reflect everyday patient care, not just clinical trials. This report examines electronic health record (EHR) databases in Japan. These databases store patient information from hospitals. The report explains what these databases can do and what challenges exist. The study used online searches and interviews with database companies. EHR databases have some strengths. They include many types of patients and laboratory test results. But there are challenges. Patients cannot be tracked if they move to another hospital. Some information may be lost when data are processed. Most data are collected after care, not in real time. Also, there is no common standard across databases. Today, EHR data in Japan are mostly used to confirm drug safety and effectiveness after approval. To use these data for new drug approvals, many issues need to be resolved through collaboration between industry, universities, and government.
Atherosclerotic cardiovascular disease (ASCVD) is a leading cause of death in the USA. We evaluated lipid-lowering therapy (LLT) and the impact of adherence on healthcare costs among patients with ASCVD. This observational cohort study used payor claims and outpatient laboratory data. Adults with ASCVD diagnosed from 1 October 2015 to 31 December 2019 were observed 12 months before, and up to 24 months after, first LLT use (index; categorized as any LLT, statin monotherapy, ezetimibe monotherapy, or anti-proprotein convertase subtilisin/kexin type 9 monoclonal antibody [PCSK9 mAb] monotherapy). Treatment patterns, low-density lipoprotein cholesterol (LDL-C) measurements, LLT adherence (proportion of days covered ≥ 0.80), and healthcare resource utilization and costs were reported. Of patients with 12 months’ follow-up post-ASCVD diagnosis, 35
BACKGROUND:Antidepressants are widely prescribed, with increasing use among older adults. However, evidence on their association with dementia remains inconsistent. OBJECTIVE:This study examines the association between long-term antidepressant use and dementia risk in older adults using a propensity score-matched design, with a separate analysis of long-term tricyclic antidepressant (TCAs) use, given their anticholinergic properties. METHODS:A retrospective cohort study was conducted using Swiss healthcare claims data (2013-2023) from older adults aged 65 years and above. Long-term antidepressant users (> 2 years) and long-term TCAs users were separately matched to nonusers using variable ratio propensity score matching. The primary outcome was incident dementia, defined by a recorded dementia diagnosis or antidementia drug prescription. Covariates were derived from inpatient and/or outpatient records, depending on the respective variable. RESULTS:A final sample of 19,155 long-term antidepressant users were compared with 20,648 propensity score matched nonusers. After adjusting for covariates, long-term antidepressant use was significantly associated with dementia (odds ratio [OR] 1.92, 95% confidence interval [CI] 1.72-2.15, p < 0.001). Long-term TCAs users also had higher odds of developing dementia compared with nonusers (OR 1.73, 95% CI 1.33-2.25, p < 0.001). Sensitivity analyses largely confirmed these results: long-term antidepressant users had higher odds of developing dementia compared with short-term users (OR 1.23, 95% CI 1.11-1.35, p < 0.001), but for TCA, the difference between long-term and short-term users was not statistically significant (OR 1.25, 95% CI 0.99-1.57, p = 0.059). CONCLUSIONS:These findings suggest that long-term antidepressant and long-term TCAs use is associated with an increased risk of dementia in older adults, even after accounting for various comorbidities. This study adds to the ongoing debate on the link between antidepressants and dementia. Future research is needed to further clarify underlying mechanisms and guide clinical practice.
In China, the national medical insurance system has undergone rapid development over the past two decades. The National Healthcare Security Administration was established to manage the growing healthcare expenditure. Since then, this administration has maintained the drug formulary, known as the National Reimbursement Drug List. The list comprises Category A (essential and cost-effective drugs) and Category B (effective drugs with relatively higher cost profiles), with the Commercial Insurance Innovative Drug list (referred to as Category C) added in 2025. China has made progress in adopting real-world evidence into healthcare decision making. Real-world evidence helps address key uncertainties related to safety, effectiveness, and long-term outcomes to complement clinical trial findings using real-world data from routine clinical practice. Evolving international experiences provide practical insights into the use of real-world evidence to support drug reimbursement and reassessment decisions. This paper proposes practical strategies supporting the integration of real-world evidence into China’s drug value assessment to better inform national formulary management and reimbursement pathways.
Ofatumumab (OFA) is a fully human anti-CD20 monoclonal antibody approved for the treatment of relapsing multiple sclerosis (RMS). Real-world data on its effectiveness and safety remain limited. To assess the effectiveness and safety of OFA in a large Italian RMS cohort and to examine the impact of prior treatment exposure on clinical, radiological, and safety outcomes in routine clinical practice. This retrospective, multicenter study included adults with RMS who initiated OFA and had at least one follow-up visit. Patients were classified as treatment-naïve, switching from moderate-efficacy therapies (MET), or switching from high-efficacy therapies (HET). Outcomes included annualized relapse rate (ARR), magnetic resonance imaging (MRI) activity, disability measures (including confirmed disability worsening and improvement), no evidence of disease activity (NEDA-3), and adverse events (AEs). A total of 424 patients were analyzed, with a mean follow-up of 1.32 (± 0.61) years: 101 (23.8
In South Korea, belimumab is indicated for patients with systemic lupus erythematosus (SLE), including paediatric patients aged ≥ 5 years, and for adults with lupus nephritis, based on clinical trial evidence; however, real-world data in this population remain limited. To assess the real-world safety and effectiveness of intravenous (IV) belimumab administered in patients with SLE in South Korea. This observational study (GSK Study 216984) enrolled patients with active SLE and patients with lupus nephritis across 18 South Korean institutions (July 2021–February 2023). Patients received on-label IV belimumab plus standard therapy (antimalarials, glucocorticoids and immunosuppressants). Data were collected for a minimum of 48 weeks as part of routine clinical practice (July 2021–March 2024). Safety was assessed by monitoring the incidence of adverse events (AEs), adverse drug reactions (ADRs), serious AEs (SAEs)/ADRs and AEs of special interest (including psychiatric events) and summarised descriptively. Changes in SELENA-SLEDAI scores, laboratory biomarkers and glucocorticoid dosage from baseline to week 24 or 48 were tested using the Wilcoxon signed-rank test. Of 126 enrolled patients, 105 were eligible for the ≥ 48-week safety evaluation (completed ≥ 24 weeks of belimumab treatment). Patients were mostly female (91.4
In November 2020, olaparib became the first approved poly adenosine diphosphate (ADP)-ribose polymerase inhibitor (PARPi) for metastatic castration-resistant prostate cancer (mCRPC) with BRCA1/2 mutations (BRCAm) in the Netherlands. As randomized clinical trials include fitter patients, their findings may not fully reflect real-world outcomes. The aim was to evaluate genomic testing practice and subsequent use and outcomes of olaparib monotherapy in a real-world BRCAm mCRPC population. Data were derived from ten hospitals in the Dutch CAPRI-3 registry, including mCRPC patients diagnosed between 2016 and 2021. Those receiving olaparib in standard-of-care treatment after its national approval (from November 2020) were analyzed and grouped as taxane-naïve (TN) or post-taxane (PT). The primary endpoint was overall survival (OS). Among 1996 mCRPC patients, genomic analysis (somatic and/or germline) was performed in 23.4
Multiple sclerosis is an immune-mediated disorder characterized by demyelination and neurodegeneration. Disease-modifying therapies are available with a range of mechanisms of action, efficacies, safety profiles, places and routes of administration, and degrees of patient autonomy; however, comprehensive evaluations of satisfaction with high-efficacy therapies in patients with multiple sclerosis are lacking. We aimed to evaluate satisfaction of patients treated with high-efficacy therapies for relapsing-remitting multiple sclerosis and identify factors influencing preferences and acceptance, focusing on the perceived impact of administration routes on quality of life, daily activities, and treatment burden. We conducted a cross-sectional survey of 208 adults diagnosed with relapsing-remitting multiple sclerosis who were receiving high-efficacy therapies for at least 6 months between February and April 2025 at Italian MS centers. Data were collected using a structured questionnaire designed for this study. Domains included: (I) Temporal and Organizational Burden of Therapy, (II) Perceived Impact of Therapy, (III) Therapy Administration Experience, and (IV) Decision-Making Involvement Experience. A non-parametric analysis revealed that more favorable scores on the domains of (I) Temporal and Organizational Burden of Therapy, (II) Perceived Impact of Therapy, and (III) Therapy Administration Experience were associated with treatment with subcutaneous ofatumumab (n = 90), compared to the other high-efficacy therapies such as natalizumab (n = 55), ocrelizumab (n = 59), and alemtuzumab (n = 4) [all n = 118, p < 0.001]. Fewer than one in four patients recalled being offered the possibility of self-administering high-efficacy therapies at home. Subcutaneous ofatumumab treatment, a therapy self-administered at home, was associated with more favorable experiences compared to other high-efficacy therapies, highlighting the importance of shared decision making and organizational and logistical factors when selecting therapies for multiple sclerosis.
Lenvatinib is a multi-kinase inhibitor approved as a first-line therapy for advanced hepatocellular carcinoma. In India and other low- and middle-income countries, immune checkpoint inhibitor-based regimens are not universally accessible, and real-world data on lenvatinib are limited. We evaluated the effectiveness, safety, and predictors of outcomes of first-line lenvatinib in Indian patients with advanced hepatocellular carcinoma. This prospective observational cohort included consecutive patients with advanced hepatocellular carcinoma treated with first-line lenvatinib at the Regional Cancer Centre, Kerala, India, between March 2021 and May 2024. Lenvatinib was started at 8 or 12 mg once daily based on weight. Overall survival and progression-free survival were estimated using the Kaplan–Meier method. Cox regression analysis identified independent predictors of outcomes. Toxicities were graded using Common Terminology Criteria for Adverse Events Version 5.0. A total of 112 patients were enrolled; median age was 62 years, and 88
This study evaluated treatment patterns and oral corticosteroid (OCS) use in Japanese patients with myasthenia gravis, comparing late-onset (mean age 79.8 years) and early/adult-onset (mean age 61.3 years) cohorts. It also explored treatment frequency rates. We conducted a retrospective analysis of the DeSC Healthcare claims database (2014–21). Adults with myasthenia gravis starting immunosuppressant therapy and with ≥ 2 years of follow-up were included. Patients were stratified by age group (< 75 and ≥ 75 years) and by exposure to early fast-acting treatment (EFT). Outcomes included OCS dose trajectories, time to achieve a maintenance dose of ≤ 5 mg/day, and the frequency of myasthenia gravis cases treated with immunotherapy. Early fast-acting treatment use was lower in the late-onset cohort (31/148 patients; 20.9
Background Allergic rhinitis (AR) is highly prevalent in Asia, but limited data exist on the real-world effectiveness of MP-AzeFlu (azelastine/fluticasone combination nasal spray) in Asian populations. Objective To evaluate the real-world effectiveness of MP-AzeFlu in Asian patients with moderate-to-severe AR. Methods This prospective, multicenter, noninterventional study enrolled patients with moderate-to-severe AR in Thailand, Taiwan, Malaysia, and Hong Kong. The patients received MP-AzeFlu and completed visual analog scale (VAS) assessments for AR symptoms over 28 days. The primary outcome was the proportion of responders (VAS score < 50 mm at least once during follow-up). Results Of the 916 patients included in the safety population, 329 (35.92%) were males and 587 (64.08%) were females. Significant improvements were observed in the overall AR and nasal and ocular symptoms from day 1 through day 28 (p < 0.0001 for all). Asthma symptoms improved significantly in patients with comorbid asthma (11.7%), and 60% reduced use of asthma reliever medication was reported. Quality of life measures, including sleep quality and daily activities, also improved significantly. MP-AzeFlu was well-tolerated, with only six patients reporting mild adverse events. Conclusions In this real-world study, MP-AzeFlu provided rapid and sustained improvement in AR symptoms and quality of life in Asian patients with moderate-to-severe AR. MP-AzeFlu has also shown beneficial effects in patients with comorbid asthma. These results support the effectiveness of MP-AzeFlu in AR management in Asian populations.
