s of May 2024, there are 36 US-approved cellular and gene therapy products. These products offer unprecedented solutions for diseases where traditional approaches fall short. However, groundbreaking innovations like cell and gene therapies bring unique challenges in their development and commercialization. During a presentation at the DIA/Harvard-MIT Center for Regulatory Science (CRS) Executive Roundtable in January 2024, Director of the FDA Center for Biologics Evaluation and Research (CBER) Peter Marks emphasized that addressing challenges in manufacturing, clinical development timelines, and different global regulatory requirements for these products is one of the center’s main goals in the year ahead: “CBER aims to make 2024 a breakout year addressing key challenges to the development of cell and gene therapies, especially for rare disorders,” Marks said.

RNA-lipid nanoparticle (LNP) products form a platform technology because the development process is very similar for products for different diseases and conditions. Clarifying the data required for regulatory submissions is critical to provide a predictable development pathway. Benefits will include reduced development and regulatory costs, faster consumer access, and more nimble development of products for pandemics, rare diseases, and cancers where alternatives may not exist.

ingle institutional review board (sIRB) review for multisite or cooperative clinical trials in the United States has had a long, twisty history and is expected to take another turn by the end of the year. Industry sponsors and contract research organizations (CROs) may be aware of the anticipated FDA sIRB mandate, but many don’t fully appreciate its seismic impact.

wapping digital for paper has proven to be the springboard for operational transformation across dozens of industries from financial services to manufacturing. Yet protocol design—the building block for clinical research—remains rooted in paper, belaboring an already long, slow process.

Paper-based protocol design is not only inefficient but also vulnerable to error, leading to delays, protocol deviations, and potentially trial failure altogether. Considering, for example, that the median revenue of oncology drugs is about $1.6 billion (with the median cost to develop oncology drugs at $648 million), there’s a lot at stake. And not just money: poor protocol design can delay the delivery of life-altering medicines. Unfortunately, it has been difficult to digitize clinical trial protocols due to a lack of standardization, document variability, and significant increase in trial complexity.

he current iteration of Good Publication Practice (GPP) Guidelines for Company-Sponsored Research was published in Annals of Internal Medicine in late summer 2022. These guidelines included important new topics previously reviewed in Global Forum, such as working with patients and helping to establish scientific and medical publications as a key function not just within clinical research but across the scientific endeavor of biomedical research in company-sponsored settings.

egulatory oversight of medicinal products entails both challenges and opportunities for regulatory authorities, including the fact that today much information about medical products is in electronic form. Electronic information translates into vast quantities of medicinal product and regulatory data, not all of which are uniform in structure, format, and content across jurisdictions. The unambiguous global identification of medicinal products is needed to achieve common international public health goals in areas such as pharmacovigilance, medicinal product track and trace, and to support other international initiatives, such as pharmaceutical quality.

he IDMP standards developed by the International Organization for Standardization (ISO) provide an international framework to uniquely identify and describe medicinal products with consistent documentation and terminologies, as well as to facilitate the exchange of product information between global regulators, manufacturers, suppliers, and distributors. When fully implemented globally, the standards will facilitate the unique identification of medicinal products in the context of pharmacovigilance, drug shortages, and the safety of medications (and patients who take them) throughout the world.

dentification of Medicinal Products (IDMP) standards developed by the International Organization for Standardization (ISO) provides an international framework to uniquely identify and describe medicinal products with consistent documentation and terminologies, as well as to facilitate the exchange of product information between global regulators, manufacturers, suppliers, and distributors. When fully implemented globally, the standards will facilitate the unique identification of medicinal products in the context of pharmacovigilance, drug shortages, and the safety of medications (and patients who take them) throughout the world.

or the second consecutive year, DIA Europe hosted a Latin America Town Hall, a panel discussion to foster collaboration and share updates from regulatory activities in Latin America and Europe, and the intersections between them.

n April 2024, the Korea Regulatory Science Center signed a Memorandum of Understanding (MoU) with DIA. Through this MoU, the two organizations will jointly plan and conduct regulatory science network-based events, including meetings and workshops; jointly plan and conduct regulatory science educational programs as well as training courses; and exchange information plus collaborate on creating a forum for discussion of the latest regulatory and technology trends in the biopharmaceutical, device and diagnostic, healthcare, and life sciences industries.

n 1964, the Drug Information Association (DIA) was founded by a group of 30 pharmaceutical professionals, medical writers, and academicians who saw the need to facilitate communications and collaboration among professionals engaged in drug development, medical communications, and health information. (Read Six Decades of Impact: DIA’s Global Journey for a more detailed historical overview.) Tom Teal was the driving force behind DIA for many of the early years and beyond.

Our 2024 EMEA Regional Inspire Award Winners