PMDA Perspective on Use of Real-World Data and Real-World Evidence as an External Control: Recent Examples and Considerations.

Recent discussions about the utilization of real-world data (RWD) and real-world evidence (RWE) have been more focused on drug development for regulatory approval rather than during the post-marketing stage. In Japan, RWD/RWE have been practically utilized as an external control for drug approval. Most cases were related to orphan diseases where the feasibility of conducting randomized controlled clinical trials was generally low.

Neurotoxicity and Rare Adverse Events in BCMA-Directed CAR T Cell Therapy: A Comprehensive Analysis of Real-World FAERS Data.

Chimeric antigen receptor T (CAR T) cell therapies have emerged as a valuable treatment modality for patients with plasma cell disorders. As the population of patients receiving CAR T therapies grows, the identification and management of associated rare toxicities become increasingly crucial. This study aims to identify safety signals associated with commercial anti-B-cell maturation antigen (BCMA) CAR T therapies using the Food and Drug Administration Adverse Event Reporting System (FAERS).

Risk of artery dissection during systemic exposure to vascular endothelial growth factor pathway inhibitors.

Whether or not vascular endothelial growth factor pathway inhibitors (VPIs) increase the risk of artery dissection is still unknown. This study aimed to quantitatively evaluate the possibility of artery dissection as a class effect of VPIs using nationwide real-world data. This cohort study was conducted based on the National Database of Health Insurance Claims and Specific Health Checkups of Japan (NDB), which spans nearly the entire Japanese population of over 100 million individuals.

Metastatic Patterns: Insights from Japanese Pathological Autopsy Registry Analysis.

Introduction: Understanding the metastatic patterns is crucial for the treatment of malignancies. This study aimed to identify the characteristic organ metastases of primary malignancies, including rare malignancies, and classify them according to their metastatic patterns.

Methods: We extracted data on primary malignancies and organ metastases from the Annual of Pathological Autopsy Cases in Japan recorded in 1993-2021.

Health Care Provider E-Cigarette-Related Advice and E-Cigarette Harm Perceptions Among Youth.

Purpose: Health care providers (HCP) are uniquely positioned to advise against electronic cigarette (e-cigarette) use, potentially influencing youth perceptions of e-cigarette harms. However, research examining these associations is scant. We examined whether HCP e-cigarette-related advice is associated with youth e-cigarette harm perceptions.

An almost infinite sites model.

Motivation: A main challenge in molecular evolution is to find computationally efficient mutation models with flexible assumptions that properly reflect genetic variation. The infinite sites model assumes that each mutation event occurs at a site never previously mutant, i.e.

Initial experimentation with tobacco is associated with subsequent tobacco use patterns among youth in the United States.

Understanding the association between initial experimentation with a tobacco product and subsequent patterns of tobacco use among youth is important to informing prevention activities for youth in the US. We conducted an online survey from August to October 2017 among youth aged 13-18 years. The current analysis focused on respondents reporting initial experimentation with any tobacco product (n = 2,022).

Anti-Tumor Necrosis Factor Therapy and the Risk of Gestational Diabetes in Pregnant Women With Inflammatory Bowel Disease.

Introduction: Anti-tumor necrosis factor (anti-TNF) therapy may improve insulin sensitivity, and its impact during pregnancy remains unclear. We aimed to assess the risk of gestational diabetes mellitus (GDM) associated with anti-TNF treatment among pregnant women with inflammatory bowel disease (IBD).

Methods: This nationwide cohort study included patients with IBD in Korea from 2010 to 2021.

The challenges of regulatory pluralism.

Countries with small and/or less-resourced regulatory authorities that operate outside of a larger medical product regulatory system face a regulatory strategy dilemma. These countries may rely on foreign well-resourced regulators by recognising the regulatory decisions of large systems and following suit (regulatory reliance); alternatively, such countries may extend formal decision recognition to regulators in multiple other jurisdictions with similar oversight and public health goals, following a system which we call regulatory pluralism. In this policy comment, we discuss three potential limitations to regulatory pluralism: (i) regulatory escape, in which manufacturers exploit regulatory variation and choose the lowest regulatory threshold for their product; (ii) increased fragmentation and complexity for countries adopting this approach, which may, in turn, lead to inconsistent processes; and (iii) loss of international bargaining power in developing regulatory policies.

Biosimilar approval pathways: comparing the roles of five medicines regulators.

Biologics are playing an increasingly important role in health care globally but are placing a substantial burden on payers. The development of biosimilars-drugs that are highly similar to and have no clinically meaningful differences from originator biologics-is critical to improving the affordability and accessibility of these medications. Medicines regulators, however, have had varied success with biosimilars to date.

Extensive social media use and frequency of current e-cigarette use among US youth.

Introduction: Both electronic cigarette (e-cigarette) and social media use among youth are public health concerns. While the health impacts of extensive social media use and frequent use of e-cigarettes have been discussed independently in the literature, little is known about the relationship between the two. This study aims to examine the potential association between extensive social media use and the frequency of current, e-cigarette use among United States (US) youth.

Mapping the regulatory landscape for artificial intelligence in health within the European Union.

Regulatory frameworks for artificial intelligence (AI) are needed to mitigate risks while ensuring the ethical, secure, and effective implementation of AI technology in healthcare and population health. In this article, we present a synthesis of 141 binding policies applicable to AI in healthcare and population health in the EU and 10 European countries. The EU AI Act sets the overall regulatory framework for AI, while other legislations set social, health, and human rights standards, address the safety of technologies and the implementation of innovation, and ensure the protection and safe use of data.

Factors associated with disposable electronic cigarette use among US youth: A national repeated cross-sectional study, 2021-22.

Background And Aims: Disposable electronic cigarette (e-cigarette) use is becoming more popular among US youth, given the shifting landscape in product marketing, availability and regulation. This study aimed to estimate the prevalence of and test factors associated with the most used e-cigarette product (disposable versus other) among US youth aged 9-18 years who currently use e-cigarettes.

Design, Setting And Participants: This was an observational study using cross-sectional data from the US-based 2021 and 2022 National Youth Tobacco Surveys (n = 48 704).

Prevalence of and trends in current cannabis use among US youth and adults, 2013-2022.

Introduction: Cannabis use is increasing due to several factors including the adoption of laws legalizing its use across the United States (US). We examined changes in current cannabis use among US youth and adults and by key sociodemographic groups.

Methods: Using data from the 2013-2022 National Survey on Drug Use and Health (n=543,195), we estimated the prevalence of (2013-2019, 2020, 2021-2022) and trends in (2013-2019, 2021-2022) current (i.

Cardiovascular Safety of COVID-19 Vaccination in Patients With Cancer: A Self-Controlled Case Series Study in Korea.

Background: Cancer patients have an increased risk of cardiovascular outcomes and are susceptible to coronavirus disease 2019 (COVID-19) infection. We aimed to assess the cardiovascular safety of COVID-19 vaccination for cancer patients in South Korea.

Methods: We conducted a self-controlled case series study using the K-COV-N cohort (2018-2021).

DifferentialRegulation: a Bayesian hierarchical approach to identify differentially regulated genes.

Although transcriptomics data is typically used to analyze mature spliced mRNA, recent attention has focused on jointly investigating spliced and unspliced (or precursor-) mRNA, which can be used to study gene regulation and changes in gene expression production. Nonetheless, most methods for spliced/unspliced inference (such as RNA velocity tools) focus on individual samples, and rarely allow comparisons between groups of samples (e.g.