Regulatory Rapporteur
December 2024 | Volume 21 | No.11
This month’s Regulatory Rapporteur focuses on North America, although it must be kept in mind how intertwined all regulatory processes are in the current environment.
We’ve acknowledged previously how the management of regulations and guidance has exploded in recent years, due to both technology and cooperation between regulatory agencies. This pace shows no sign of slowing, especially as the industry continues to leap forward in terms of innovation and the volume of available raw data.
Our first article, by Leah R. Kleylein of Accenture, centres around recent notable US Food and Drug Administration (FDA) pilot programmes. Four pilots are highlighted: the FDA Dataset-JSON Pilot, the Guidance Snapshot Pilot, the Total Product Life Cycle Advisory Program (TAP) Pilot, and the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. At any time, the FDA can have dozens of pilots running in and across various Centers, and it can be difficult to locate what’s been completed and what is still ongoing. This article reviews the status of these four pilots, chosen as examples across different topic areas, including study data technology, general guidance comprehension, medical devices and rare diseases.
Our second article, by John Watkins and Morgane Lejeune of Thermo Fisher Scientific, provides a comprehensive summary of what to consider when preparing quality and new substance notification documentation for investigational medicinal products in Canada. It offers key awareness and guidance on quality overall summary (QOS) requirements for biopharmaceuticals, advanced therapy medicinal products, genetically modified organisms and vaccines, and summarises new substance notification requirements with respect to chemicals and biochemicals to be used in a clinical trial. In addition to following Health Canada guidelines, it also suggests following the recommendations in the European Medicines Agency quality guidelines and FDA guidelines for facilities information.
In our third article, Susan Neadle of Combination Products Consulting Services provides a thorough analysis of the June 2024 FDA draft guidance for industry on ‘Essential Drug Delivery Outputs for Devices Intended to Deliver Drugs and Biological Products’ and the international standards that are also key to combination product development. The FDA guidance, which attempts to clarify earlier communications on the performance requirements for drug-led (or biological product-led) drug-device combination products, eliminates the term ‘essential performance requirements’ and replaces it with ‘essential drug delivery outputs’. This guidance is much needed by industry as it addresses device design-dependent system level functions whose performance must be assured to guarantee the quality of the drug delivery function throughout the combination product lifecycle. Outstanding comments and issues on the draft guidance still remain, many of which are identified in this comprehensive review.
All clinical trials authorised under the Clinical Trials Directive with at least one active site in the European Economic Area on 30 January 2025 must complete the transition to the Clinical Trials Regulation (CTR) within the transition period of three years ending on 31 January 2025. In our fourth article, Valentina Geisseler-Homann, Natalia Lugli, Jo Molloy and Marco Rafael of F. Hoffman-La Roche recount the process involved in the transition of a clinical studies portfolio to the CTR (EU) 536/2014 by the deadline. Roche developed a proactive, enterprise-wide strategy for managing the transition and the article shares excellent lessons learned from their experience executing the process, including the challenges encountered. Revisions and updates to the CTR guidance and the Clinical Trial Information System were ongoing during the transition process which required real-time adaptation in changing processes that were already underway.
This issue of Regulatory Rapporteur provides us with the latest medical device standards update, compiled by Charlotte Holleran of Endomagnetics. This includes newly published European and ISO standards on requirements for terminally sterilised medical devices and aseptic processing of healthcare products, as well as several new ISO standards which are under review on topics such as sterilisation, degradation and manufacturer-supplied information. A European standard for culture media is also under review. After being added to the FDA consensus list and the EU, standards on sterilisation processes, software testing and clinical performance studies have been introduced into the list. Finally, of note, the entire series EN ISO 18113-1 to EN ISO 18113-5 (information supplied by the manufacturer for IVDs) from 2011 has been withdrawn and superseded by a 2024 version, none of which are either on the EU harmonised or the FDA consensus list.
Our final article this month is a post-pandemic review on the early development of vaccines and prophylactics in infectious diseases, authored by Bruno Speder of AstriVax and Robin Rogiers of Leyden Labs. The unprecedented agility that the industry showed during the COVID-19 pandemic in accelerating the development and approval of vaccines has shown us that this can be done not only quickly, but safely. However, a lack of clear guidance and clarity on what regulatory authorities consider acceptable in the evolution of a trusted clinical trial (such as limited size, time constraints and a lack of resources forcing a trial to be more agile than planned) are hindrances, but hopefully only temporarily.
We hope you enjoy this issue.
