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Review
. 2024 May 11;12(5):528.
doi: 10.3390/vaccines12050528.

The Platform Technology Approach to mRNA Product Development and Regulation

John H Skerritt  1 , Carolyn Tucek-Szabo  2 , Brett Sutton  3 , Terry Nolan  1   4
Affiliations
Review

The Platform Technology Approach to mRNA Product Development and Regulation

John H Skerritt et al. Vaccines (Basel). .

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 often utilised 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 that 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: drug development; mRNA; regulation; vaccine development.

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Conflict of interest statement

The working group and technical assistance for initial manuscript drafting was funded by Moderna Australia; TN has received personal payments for advisory roles with CSL Seqirus, GSK, Sanofi, Pfizer and Moderna and for data safety monitoring board (DSMB) membership on clinical trials sponsored by Arcturus, CSL Seqirus and Moderna.

Figures

Figure 1
Figure 1
Flowchart of the review—hierarchy of potential changes to mRNA products.
See this image and copyright information in PMC

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Grants and funding

Moderna Australia funded the working group secretariat and initial drafting of the paper by J.S., However, the work was undertaken as part of J.S.’s professorial responsibilities at the University of Melbourne. Participation of other working group members was funded by their employers.

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