Version 1
: Received: 16 April 2024 / Approved: 16 April 2024 / Online: 16 April 2024 (13:56:02 CEST)
How to cite:
SKERRITT, J.H.; Tucek-Szabo, C.; Sutton, B.; Nolan, T. The Platform Technology Approach to mRNA Product Development and Regulation. Preprints2024, 2024041060. https://doi.org/10.20944/preprints202404.1060.v1
SKERRITT, J.H.; Tucek-Szabo, C.; Sutton, B.; Nolan, T. The Platform Technology Approach to mRNA Product Development and Regulation. Preprints 2024, 2024041060. https://doi.org/10.20944/preprints202404.1060.v1
SKERRITT, J.H.; Tucek-Szabo, C.; Sutton, B.; Nolan, T. The Platform Technology Approach to mRNA Product Development and Regulation. Preprints2024, 2024041060. https://doi.org/10.20944/preprints202404.1060.v1
APA Style
SKERRITT, J.H., Tucek-Szabo, C., Sutton, B., & Nolan, T. (2024). The Platform Technology Approach to mRNA Product Development and Regulation. Preprints. https://doi.org/10.20944/preprints202404.1060.v1
Chicago/Turabian Style
SKERRITT, J.H., Brett Sutton and Terry Nolan. 2024 "The Platform Technology Approach to mRNA Product Development and Regulation" Preprints. https://doi.org/10.20944/preprints202404.1060.v1
Abstract
mRNA-lipid nanoparticle (LNP) medicinal products can be considered a platform technology because the development process is similar for different diseases and conditions, with similar noncoding mRNA sequences and lipid nanoparticles and essentially unchanged manufacturing and analytical methods utilised often for different products. It is critical not to lose the momentum built using the platform approach during the development, regulatory approval and rollout of vaccines for SARS-CoV-2 and its variants. This Review proposes a set of modifications to existing regulatory requirements for mRNA products, based on a platform perspective for quality, manufacturing, preclinical and clinical data. For the first time, we address development and potential regulatory requirements when the mRNA sequences and LNP composition vary in different products as well. In addition, we propose considerations for self-amplifying mRNA, individualised oncology mRNA products and mRNA therapeutics.
Providing a predictable development pathway for academic and commercial groups, so they can know in detail what product characterisation and data are required to develop a dossier for regulatory submission, has many potential benefits. These include reduced development and regulatory costs; faster consumer/patient access and more agile development of products in the face of pandemics; and for rare diseases where alternatives may not exist or to increase survival and the quality of life in cancer patients. Therefore, achieving consensus around platform approaches is both urgent and important. This approach with mRNA can be a template for similar platform frameworks for other therapeutics and vaccines to enable more efficient development and regulatory review.
Keywords
mRNA, vaccine development, drug development, regulation
Subject
Medicine and Pharmacology, Medicine and Pharmacology
Copyright:
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
