Bayesian statistics plays a pivotal role in advancing medical science by enabling healthcare companies, regulators, and stakeholders to assess the safety and efficacy of new treatments, interventions, and medical procedures. The Bayesian framework offers a unique advantage over the classical framework, especially when incorporating prior information into a new trial with quality external data, such as historical data or another source of co-data. In recent years, there has been a significant increase in regulatory submissions using Bayesian statistics due to its flexibility and ability to provide valuable insights for decision-making, addressing the modern complexity of clinical trials where frequentist trials are inadequate. For regulatory submissions, companies often need to consider the frequentist operating characteristics of the Bayesian analysis strategy, regardless of the design complexity. In particular, the focus is on the frequentist type I error rate and power for all realistic alternatives. This tutorial review aims to provide a comprehensive overview of the use of Bayesian statistics in sample size determination, control of type I error rate, multiplicity adjustments, external data borrowing, etc., in the regulatory environment of clinical trials. Fundamental concepts of Bayesian sample size determination and illustrative examples are provided to serve as a valuable resource for researchers, clinicians, and statisticians seeking to develop more complex and innovative designs.

This study aimed to demonstrate the differences in the way cell and gene therapy (CGT) products have been developed and reviewed for approval in Japan, the USA, and the EU by comparing regulations and successfully launched products in each region, and to examine the background to such differences.
Information on relevant regulations and approved CGT products were collected from the public source and compared by region.
While regulations on CGT products are largely consistent among these regions, some differences could have a substantial impact on the practices defining CGT products, the timing of responses required to comply with the regulations for handling gene-modified organisms, and the acceptable validation processes under good manufacturing practice regulations. Although CGT products are given some preferential status in all regions, the preferential treatment given to CGT products varies across regions. The CGT products launched in each region also differ significantly in type, indications, the nature of the developers, and the clinical evidence submitted. While all the cellular products launched in Japan were approved based on small uncontrolled trials, most cellular products in the USA and EU were approved based on controlled studies. A trend was observed for companies to enter their home markets.
Our study showed differences of regulations on CGT products and of features in approved products as well as the trend of their home market entries, which may have been driven by a different context than that of traditional pharmaceuticals.

Previous analyses of the effect of e-cigarettes on real world smoking cessation success have mainly been based on surveys undertaken in the United States and United Kingdom, where nicotine e-cigarettes can be readily obtained. In Australia, regulations have made obtaining e-cigarettes containing nicotine difficult. The effectiveness of e-cigarette use as a smoking cessation aid in Australia might therefore be lower than survey-based estimates published to date. This study aimed to estimate the effect of using e-cigarettes for a smoking cessation attempt on past-year smoking cessation success in Australia.
Multivariable logistic regression models for past-year smoking cessation success.
Respondents to the 2019 wave of Australia\'s National Drug Strategy Household Survey who made a smoking cessation attempt in the 12 months leading up to the survey.
Past-year smoking cessation success was assumed if a smoking cessation attempt resulted in abstinence of more than a month at the time of the survey.
In 2019, Australians who attempted to quit smoking using e-cigarettes achieved greater success than smokers attempting to quit without e-cigarettes [adjusted odds ratio (aOR) = 1.68; 95% confidence interval (CI) = 1.09-2.60]. If people who only tried e-cigarettes once or twice are considered not to have used e-cigarettes, the estimated effect was slightly stronger (aOR = 1.98; 95% CI = 1.27-3.10). Also, the estimated odds ratio was higher among vapers who acquired their e-cigarettes from overseas websites (aOR = 2.24; 95% CI = 1.02-4.93).
Use of e-cigarettes for a smoking cessation attempt appears to be associated with greater success among Australians who attempted to quit tobacco in 2019 compared with Australians attempting to quit without e-cigarettes, after adjusting for confounding effects.

Sub-Saharan Africa\'s regulatory environment ranks amongst the least business friendly in the world. The difficulty of starting and operating businesses and the high tax burden are amongst the major conditions that make the regulatory environment hostile. This study examines how these business regulatory conditions explain the growing challenges in mitigating CO2 emissions in the sub-region. For this purpose, data from 1997 to 2018 are used to analyse an extended environmental Kuznets curve (EKC) equation for thirty (30) Sub-Saharan African countries. The results of the Method of Moments Quantile Regression analysis show that the inverted U-shaped curve of the EKC hypothesis is statistically not valid across the entire quantile distributions. The impact of increasing tax burden on CO2 emissions is positive and increases across the entire quantile distributions. Business regulatory efficiency has a negative (i.e. decreasing) impact on CO2 emissions across the entire quantile distributions and shows a stronger impact in countries at the upper quantiles, such as in South Africa, Botswana, Gabon, and Nigeria. Conclusively, policy choices that seek to reduce tax burden on households and firms and foster greater economic freedom for businesses are needed to break the growing trend in Sub-Saharan Africa\'s CO2 emissions.

Stability of samples for flow cytometry is a critical parameter since storage period of samples is restricted to only a limited period after collection. For most studies, clinical samples have to be shipped to a testing laboratory, in contrast to preclinical samples, which can be analyzed on-site or off-site. Therefore, evaluating stability is critical to provide flexibility on testing of samples to obtain reliable data. A wide variety of factors contributes to establishing stability from sample collection through acquisition. We provided suggestions for experimental and stability parameters to be taken into consideration when designing a flow cytometry method. The case studies presented represent how certain stability issues were overcome to perform flow cytometry assays in a regulated bioanalytical environment.

In vitro diagnostic devices (IVDs) help clinicians determine specific conditions, monitor therapeutic efficacy, and prevent drug resistance development. While stringent regulatory authorities (SRAs) regulate IVDs in most high-income countries, regulatory authorities in many low- and middle-income countries (LMICs) are nonexistent or do not enforce rigorous standards. In 2010, the World Health Organization established its Prequalification of In Vitro Diagnostics (PQDx) program to ensure \"access to safe, appropriate and affordable\" IVDs, especially in LMICs with little or no domestic regulatory frameworks, thereby reaching underserved populations. However, challenges in PQDx policies and procedures include an overloaded pipeline, timelines not publicly available, confusion about which products PQDx focuses on, perceived burden for documenting changes to prequalified products, overlap with SRA approvals, and uncertainty around long-term financing. PQDx can maximize its impact by considering the perspective of IVD manufacturers; similarly, IVD manufacturers should exercise adequate quality control over their submissions and associated processes.

In the past few years, there have been numerous updates to policy and guidelines governing the conduct of clinical research in India. These measures were taken by regulators considering safety of Indian patients as the topmost priority although the overall regulatory environment became challenging. However, in the recent past, Indian regulations have evolved positively to favorably support clinical research in India while appropriately balancing patient safety as well. These regulatory changes are expected to bring newer innovative medicines to Indian patients at an earliest.

Informal employment represents more than half of nonagricultural employment in most developing regions, contributes to the overall economy, and provides pathways to reduction of poverty and inequality. Support to the informal economy should include the expansion of occupational health and safety to include informal workers, based on an analysis of their work places and work risks. The paper presents main schools of thought and argues for a holistic understanding of the different segments of the informal work force and for policies and interventions tailored to the needs and constraints of these different segments. The paper recommends a policy approach which seeks to extend social protection, including occupational health and safety services, to informal workers, and to increase the productivity of informal enterprises and informal workers through an enabling environment and support services. The paper calls for a new paradigm of a hybrid economy which would value and integrate the informal economy alongside the formal economies.

BACKGROUND: The incidence of type 1 diabetes (T1D) is increasing worldwide and there is a very large need for effective therapies. Essentially no therapies other than insulin are currently approved for the treatment of T1D. Drugs already in use for type 2 diabetes and many new drugs are under clinical development for T1D, including compounds with both established and new mechanisms of action. Content of the Review: Most of the new compounds in clinical development are currently in Phase 1 and 2. Drug classes discussed in this review include new insulins, SGLT inhibitors, GLP-1 agonists, immunomodulatory drugs including autoantigens and anti-cytokines, agents that regenerate β-cells and others. Regulatory Considerations: In addition, considerations are provided with regard to the regulatory environment for the clinical development of drugs for T1D, with a focus on the United States Food and Drug Administration and the European Medicines Agency. Future opportunities, such as combination treatments of immunomodulatory and beta-cell regenerating therapies, are also discussed.