We aimed to assess the role of real-world data (RWD)/real-world evidence (RWE) for regulatory decision-making of the marketing authorisation approvals of orphan medicinal products (OMPs) in the European Union (EU) between 2000 and 2025. All European public assessment reports (EPAR) of the OMPs approved in the EU between 1 January 2000 and 31 December 2025 were screened for RWD/RWE submitted by the applicant in the initial application (pre-authorisation phase) and classified as ‘main’, ‘supportive’ or ‘not known’. Post-authorisation measures foreseen, described in Annex II conditions or additional pharmacovigilance activities required to address specific safety concerns were checked and were included if RWD/RWE was present. RWD/RWE were present in about 80
Environmental contamination by pharmaceutical residues accumulation in water bodies, risk of ecological disturbance and development of antimicrobial resistance have become a serious global threat. Pharmaceutical active ingredients and metabolites of these substances expelled into the surrounding environment by excretion, industrial wastewater and improper disposal practices result in measurable levels in surface waters and wastewater globally. In response, regulatory authorities incorporated Environmental Risk Assessment (ERA) requirements into the drug licensing system. For human medicinal products, a product-specific, tiered Environmental Risk Assessment (ERA) has been applied by the European Medicines Agency (EMA). NEPA-based environmental review involving applicable categorical exclusions and Environmental Assessments (EAs) has been utilized by the US Food and Drug Administration (FDA), whereas there is no dedicated medicinal-product ERA guideline within the NDCTR framework currently for India’s Central Drugs Standard Control Organisation (CDSCO), but have a broader environmental legislation addressing environmental protection. This article mainly compares the scope, methodology, requirements for data, how the data should be reported, and challenges of implementation of the ERA regulatory infrastructure among the EMA, FDA, and CDSCO. To show similarities and differences, comparison tables and flow diagrams are added. Regulatory gaps, mainly in India, and possibilities for harmonised ERA frameworks that can safeguard global health and biodiversity by encouraging regulatory consolidation are discovered by this study.
Since 2011, the Taiwan Food and Drug Administration (TFDA) has established various facilitated regulatory pathways (FRPs, including expedited and accelerated review frameworks) for new drug applications. In 2017 and 2019, TFDA implemented the Refuse to File (RTF) mechanism and conducted a comprehensive revision of FRPs, respectively. This study aims to evaluate the outcomes of these reforms from 2015 to 2024, focusing on New Active Substance (NAS) approvals, pathway utilization, and international comparison. NAS approvals between 2015 and 2024 were analyzed using TFDA’s internal regulatory database, with U.S. Food and Drug Administration (US FDA), European Medicines Agency (EMA), and Japan’s Pharmaceuticals and Medical Devices Agency (Japan’s PMDA) data obtained from public sources. Analyses and visualizations were conducted with R. Following the 2017 and 2019 reforms, the on-time rate for the 360-day standard pathway surged from 19.0
In Peru, the regulatory framework governing clinical research has evolved substantially, particularly during the COVID-19 pandemic; however, recent patterns of authorized clinical trials (CTs) have not been systematically described. To characterize authorized CTs in Peru and to describe their evolution across the pre-pandemic (2017–2019), pandemic (2020–2022), and post-pandemic (2023–2025) periods. Retrospective descriptive study using secondary data from the Peruvian Clinical Trial Registry (REPEC). All CTs authorized between January 2017 and December 2025 were included. Trial, population, geographic, and regulatory characteristics were assessed. Categorical variables were compared using chi-square or Fisher’s exact tests, and continuous variables using the Kruskal–Wallis test. A total of 421 authorized CTs were analyzed. Most were foreign-sponsored (90.0
