Live biotherapeutic products are an emerging class of biological medicines that rely on viable microorganisms to treat or prevent disease. Unlike probiotics marketed as foods or dietary supplements, live biotherapeutic products are regulated as drugs and are therefore subject to rigorous requirements for quality, safety, and clinical efficacy. Recent approvals of microbiota-based treatments for recurrent Clostridioides difficile (C difficile) infection, a bacterial illness that causes severe diarrhea, highlight their therapeutic potential and the regulatory challenges posed by using living organisms. This article examines regulatory expectations for live biotherapeutic products in the US and EU, with an emphasis on safety evaluation, clinical development strategies, and manufacturing controls.

As medical device innovation accelerates and regulatory requirements grow more complex, medtech companies face operational challenges in scaling their regulatory affairs (RA) functions. RA teams, which have historically been underfunded, must handle more activity with the same number of employees. This article examines the ensuing challenges RA teams face in bringing new products to markets and how using technology to manage regulatory information can help centralize information, structure documentation and data, and standardize and streamline workflows. The article emphasizes the importance of training and upskilling programs and shifting from reactive, manual processes to integrated, proactive systems.

Pediatric plans are regulatory frameworks ensuring that the well-being of the pediatric population is adequately considered during the development of new medicinal products. With global harmonization, regulatory writing becomes critical in drafting compliant, ethical, and scientifically robust pediatric study strategies. This article provides an overview of the differences in pediatric plan requirements across the European Medicines Agency (EMA), Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), and the US Food and Drug Administration (FDA), highlighting the regulatory submission process, writing considerations, and common challenges encountered during plan development.

This article examines the Eurasian Economic Union (EAEU) regulatory pathway following the completion of the transition period in December 2025, after which all medicinal products on the union's market must comply with unified registration rules. It provides a practical guide to the key regulatory institutions, the hierarchy of normative documents, registration procedures, and electronic common technical document (eCTD) dossier requirements. Particular attention is paid to document preparation, common applicant mistakes, and strategies to optimize the regulatory process. The recommendations are based on an analysis of the EAEU regulatory framework and the author’s extensive hands-on experience.

This article discusses how comparability protocols provide structured, pre-approved plans for managing postapproval chemistry, manufacturing, and controls (CMC) changes while ensuring product quality. These protocols explain the scientific rationale for the proposed change, outline the analytical strategy to demonstrate comparability, define the acceptance criteria, and specify the recommended reporting category. The use of comparability protocols aligns with the principles outlined in International Council for Harmonisation (ICH) guidelines, which stress risk-based decision making and strong lifecycle quality management. This article also discusses the advantages and limitations, differences between the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approaches, and practical implementation considerations for comparability protocols.

This article presents a case study outlining the development and submission of a commercial investigational new drug (IND) application for a new chemical entity (NCE) developed at The Ohio State University (OSU). Drawing on their experience as academic drug developers directly involved in the project, the authors describe the regulatory pathway from academic discovery to IND submission. They explore the unique challenges and opportunities for academic drug development and provide valuable insights for other academic institutions embarking on similar paths to an IND submission. Key lessons include the importance of thorough preparation, regulatory strategy, interdisciplinary collaboration, regulatory expertise, and the ability to swiftly respond to feedback.

This article is the first of two on strategic regulatory intelligence for cell and gene therapies (CGTs). It will examine how gaps between product approvals and patient access remain despite significant evolutions in the CGT landscape. Longer approval timelines and increasing product withdrawals underscore the need for robust regulatory intelligence frameworks that integrate geopolitical considerations, real-world evidence (RWE), artificial intelligence/machine learning (AI/ML) tools, and multistakeholder collaboration to build an effective regulatory strategy. The article examines the role of regulatory intelligence in advancing effective regulatory strategy across the full CGT and advanced therapy medicinal product (ATMP) lifecycles, including innovative clinical trial designs, chemistry, manufacturing, and controls (CMC) optimizations, and postauthorization surveillance across major global jurisdictions. The second article explores global CGT pricing and reimbursement models.

This is the second of two articles on cell and gene therapies (CGTs). CGTs are among the most transformative and commercially complex therapeutics with their curative potential and one-time administration model. However, their extremely high research, development, and manufacturing costs create a unique set of regulatory and market access challenges not adequately addressed by the existing frameworks. The article synthesizes comparative regulatory intelligence across five major jurisdictions and discusses the corresponding health technology assessments (HTA), payer expectations, and reimbursement landscapes governing commercial access for approved CGT products. The first article focuses on the role of strategic regulatory intelligence in shaping CGT strategy across the development lifecycle.