Platform technologies for vaccines – why is there still no regulatory guidance available?

by Michael Pfleiderer

We have previously reported on the need and significance of platform technologies for the production of vaccines, specifically those against emerging and re-emerging infectious diseases (see our Newsletter from December 12, 2016.  

Meanwhile, almost three years later, the global outbreak situation reminds us even more drastically that rapid availability of vaccines against viral infectious agents causing regular or irregular regional and international outbreaks is of unprecedented importance. Particular urgency of this matter is exemplified by the recent widespread, long-lasting and recurrent outbreaks of Ebolavirus, Chikungunyavirus, Denguevirus, Zikavirus as well as other viral diseases [1].

Numerous funding organizations, venture capitalists and investment banks respond to this urgency by providing substantial financial support to vaccine developers. Investments made are, however, often linked to the expectation that a manufacturing process for a vaccine against one of the aforementioned diseases should also be applicable to one or more other vaccines, thus successively abbreviating development timelines and cutting down investments normally required for novel vaccines. Considering that development of emergency vaccines is mainly driven by academic institutions as well as by small and medium sized enterprises rather than by the very few global players in vaccine development and procurement, time and cost reducing technologies will play a fundamental role for future global public health. Furthermore, as those smaller enterprises are very often heavily dependent from financial support the risk for investors of losing their investments will be mitigated the more versatile a manufacturing process is.

As scientists and regulators unanimously vote for a change of paradigms governing development, testing and licensing of vaccines numerous novel regulatory principles, primarily priority programs, have been developed in the past by EMA and FDA that may be helpful to shorten timelines required to get from lab to license [2, 3].

However, platform technologies have so far not been recognized by regulatory systems as a top priority facilitating rapid access to urgently needed vaccines. Although it is clear that platforms per se cannot be licensed – as drug legislation is focused on product licensure and not on process licensure – regulatory guidance on important aspects pertinent to platform technologies would be welcome.

Accordingly, depending on the robustness of a manufacturing platform and the comparability of the starting materials, in particular viral vectors, large parts of the non-clinical development, process validation and clinical development could be adopted from growing experience with vaccines already derived from such a platform. In other words, with every licensed vaccine derived from a platform technology experience gathered could be used to abbreviate development programs for another vaccine produced under the same conditions. Over time, such an approach would allow significant reduction of development timelines, in the ideal case down to development periods not exceeding one year.

In that context, the Center for Health Security of Johns Hopkins’ Bloomberg School of Public Health has recently summarized the most important scientific and regulatory considerations regarding vaccine platform technologies in a scientific publication; “Vaccine Platforms: State of the Field and Looming Challenges” [4]. This publication provides an excellent overview on the crosslinking elements impacting vaccine development, licensure and procurement in the future within a functional framework regulating manufacturing process platforms.

Biopharma Excellence is actively contributing to establishing regulatory standards for vaccine manufacturing platforms. As such, we will be happy to assist you in taking advantage from the option of using manufacturing platforms for your vaccine development portfolio even in the absence of official guidance as we are able to anticipate future regulatory expectations ensuring continuous regulatory compliance of such development programs. Please contact us for more information.

[1]        https://www.who.int/emergencies/diseases/en/

[2]        FDA, 2017. Guidance on Expedited Programs for Serious Conditions  Drugs and Biologics, Retrieved from https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-serious-conditions-drugs-and-biologics

[3]        https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines

[4]        Amesh A. Adalja, Matthew Watson, Anita Cicero, Tom Inglesby. Vaccine Platforms: State of the Field and Looming Challenges, Retrieved from http://www.centerforhealthsecurity.org/our-work/publications/vaccine-platforms-state-of-the-field-and-looming-challenges