A new paper has recommended a path forward so that mRNA products can be considered a 'platform technology' to enable a more predictable development and regulatory pathway.
The paper's authors are former TGA head Professor John Skerrit, Moderna's Dr Carolyn Tucek-Szabo, Dr Brett Sutton AO, and Professor Terry Nolan.
The paper says that mRNA-lipid nanoparticle medicinal products can be considered a platform technology because their development process is similar for different diseases and conditions. They have similar noncoding mRNA sequences, lipid nanoparticles, and essentially unchanged manufacturing and analytical methods often utilised for different products.
The authors argue that adopting a platform approach during the development, regulatory approval, and rollout of the vaccines against COVID-19 and its variants generated momentum that should not be lost.
They have proposed modifications to existing regulatory requirements for mRNA products based on a platform perspective for quality, manufacturing, preclinical, and clinical data.
"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," say the authors.
They say the platform approach's benefits include reduced development and regulatory costs, faster patient access, and more agile product development in the face of pandemics. It could also benefit patients living with rare diseases who lack treatments and increase survival and 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," say the authors.
