Criteria for the application of the platform approach for mRNA vaccines and gene therapy products in regulatory practice
TL;DR
It was found that the regulatory documentation of the Russian Federation and the EAEU lacks criteria for applying the platform approach to mRNA vaccines and gene therapy products, which hinders the accelerated introduction of innovative personalized medicinal products into clinical practice.
Abstract
INTRODUCTION. The platform approach, which involves the use of an unchanged manufacturing process and standardized quality control methods, is recognized by leading regulatory authorities, such as the Food and Drug Administration (FDA) and the European Medicines Agency (EMA), as an effective tool for accelerating market entry of innovative products, namely mRNA vaccines and gene therapy products. In this context, the implementation of this concept is relevant for the Russian Federation and the countries of the Eurasian Economic Union (EAEU), as it would accelerate pharmaceutical development, optimize quality control and process validation, and improve regulatory review during the registration of such products.
Aim
This study aimed to analyze international regulatory approaches to the application of platform technologies in the manufacturing and quality control of mRNA vaccines and gene therapy products based on viral vectors and genome editing systems, in order to provide a rationale for their implementation in the national legislation and regulatory documentation of the Russian Federation and the EAEU.
Discussion
An analysis of publications from 2015 to 2025 in the PubMed, Google Scholar, and eLIBRARY.ru databases, as well as official documents from regulatory authorities, including the FDA, the EMA, the World Health Organization (WHO), the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), the Medicines and Healthcare products Regulatory Agency, and the Pharmaceuticals and Medical Devices Agency (PMDA), addressing the application of platform technologies in the manufacturing and quality control of medicinal products, was performed. It was found that the regulatory documentation of the Russian Federation and the EAEU lacks criteria for applying the platform approach to mRNA vaccines and gene therapy products, which hinders the accelerated introduction of innovative personalized medicinal products into clinical practice. International regulatory approaches (FDA, EMA, PMDA) were shown to accelerate development and process validation, and reduce the scope of preclinical studies and clinical trials through the reuse of data on previously manufactured and approved products (including data on the manufacturing process and safety profiles for mRNA vaccines and gene therapy products). The main unresolved issues remain the amount of supporting data and the need for a first approved product before platform technology status can be granted. The need to develop unified regulatory requirements for relevant experience and process validation was substantiated, which would allow the benefits of the platform approach to be realized while maintaining a high level of safety and efficacy of medicinal products during regulatory review.
Conclusions
The platform approach, based on an unchanged manufacturing process and standardized quality control methods, can accelerate pharmaceutical development, reduce the extent of preclinical studies and clinical trials, and optimize the regulatory review of mRNA vaccines and gene therapy products. To implement this approach in the Russian Federation and the EAEU, it is necessary to incorporate provisions into the regulatory documentation allowing the use of standardized modules within a single technological platform, which would accelerate the development and market launch of gene therapy products and personalized products, and increase their affordability.
