Platform technologies can be defined as technologies with a fixed component that can be reused to develop multiple medicinal products, each containing an additional variable component targeting a specific disease or personalised to an individual or group.
The recent advances in vaccine, cell-based and gene editing technologies demonstrate that the current regulatory approach is outdated and a framework for regulation of platform technologies would be beneficial for faster access to innovative medicines and reduction of development costs through streamlined regulatory approval. This is especially important for vaccines, where a rapid adaptation to a changing target is often needed, as well as for ultra-rare diseases where meaningful clinical trials are not feasible due to low patient numbers.
Current regulations
Medicines are currently developed with the aim of obtaining a Marketing Authorisation for a specific medicinal product (i.e., one active substance). Current medicine regulations do not allow for authorisation of specific platform technologies, meaning that each Marketing Authorisation Application must contain a complete data set for each medicinal product.
‘Prior knowledge’ is accepted by regulators and Industry can include relevant supporting data from scientific publications or similar products and processes within Clinical Trial Applications or Marketing Authorisation Applications, thereby sharing evidence for safety, efficacy or quality across multiple medicinal products. Applicability of the prior knowledge to the medicinal product under development must be demonstrated within the application and bridging studies may be necessary. Scientific advice from regulators on the appropriate use of prior knowledge is therefore encouraged to reduce the risk of non-approval at the regulatory application stage.
Examples of prior knowledge include the annual update of the seasonal influenza vaccines as well as the approval of one of the Covid vaccines, where prior knowledge of the viral vectors used within the vaccine (such as specifications, control strategies and stability) was accepted by regulators. Approval was, therefore, accelerated and certain product specific data was deferred to the post-authorisation phase.
In addition, personalised medicines (n=1) are allowed, and clinical trials are currently possible for medicines produced according to a known process with patient specific targets, however, the regulatory landscape is currently very complex to navigate.
Future regulations
The new pharmaceutical legislation known as the ‘new pharma package’ proposes a new pharmaceutical Directive that provides for platform technologies. Following the first legislative proposal in 2023, near final draft text was published by the European Parliament 17 September 2026. Following adoption by the European Parliament, anticipated Autumn 2026, this new Directive will enter into force and full applicability is foreseen from 2028.
The new pharmaceutical Directive includes a provision for a Platform Marketing Authorisation, where regulatory approval of a defined component (or platform) of a medicinal product could be achieved. This Platform Marketing Authorisation must serve a clinical purpose and would be used as a basis for the approval of many different medicinal products, each comprising the same platform as well as different variable components that target different infectious agents or tailor the medicines to an individual or a group of patients. The use of a Platform Marketing Authorisation must be agreed with the regulator in advance of submitting the Marketing Authorisation Application.
Well-characterised and reproducible ‘prior knowledge’ supports the incorporation of a platform technology during the development, the manufacture and the quality control of medicinal products. As such, provision is also made in the new legislation for a Platform Technology Master File, which is a stand-alone, platform-specific dossier containing all of the quality, non-clinical and clinical data that is expected to remain unchanged across medicinal products employing the same technology. Annex II of the adopted new pharmaceutical Directive will detail the dossier requirements for these Master Files. The owner of the Master File may be a Marketing Authorisation Holder (MAH) or a third party, however, a MAH relying on a this type of Master File would be fully responsible for the Master File in their own Marketing Authorisation. The Marketing Authorisation Application would include both the entire Master File and the data showing the required customisation of the platform technology for a particular medicinal product. Therefore, the full data set would be available to regulators for the assessment of the risk benefit of the medicinal product. Regulators could rely on a previous regulatory assessment of the platform technology, thereby reducing assessment time. A simple lifecycle management process of the Master File will be required to ensure feasibility in regulatory applications and assures up to date information with protection of intellectual property.
Conclusion
The new pharmaceutical Directive allows for both a Platform Technology Marketing Authorisation and a Platform Technology Master File that should accelerate development and approval time lines and reduce development costs of associated medicinal products. It will still be possible to leverage prior knowledge once these frameworks are in place, as per current regulations, which is important for medicinal products not eligible for the platform technology regulation.
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