Individualised mRNA cancer immunotherapies do not meet the definition of vaccines, therefore the very sexy terminology of ‘cancer vaccines’ is inappropriate.
MHRA’s clarifies own definitions (https://assets.publishing.service.gov.uk/media/6799ef4d9a6dc0352ab34225/Individualised_mRNA_cancer_immunotherapies_0.6.5.pdf).
More appropriately, MHRA states that such products are individualised mRNA cancer immunotherapies that use lipid nanoparticle delivery systems, and the current scope is confined to the specific regulatory and scientific challenges of these technologies. These cancer immunotherapies contain mRNA as the active substance, encapsulated in lipid nanoparticles for drug delivery. The mRNA consists of a fixed component and a variable component. They are individualised because the design of the variable component of the mRNA molecule is tailored to each patient’s unique tumour neoantigen profile. Following administration, the mRNA molecule is delivered to host cells for expression of neoantigens, with the aim of generating an immune response against the tumour
MHRA has written and published draft guidance for comments by all stakeholders, informed by advice from the independent Highly Personalised Medicines Expert Working Group (EWG) and the Commission on Human Medicines (CHM). While this initial draft guidance document is primarily intended for developers of individualised mRNA cancer immunotherapies, the regulatory and scientific principles discussed herein could broadly be applicable to other disease indications or technologies that could benefit from personalisation or individualisation. Specific cases may
be clarified in subsequent updates to the guidance.
The MHRA correctly takes the opportunity to further clarify that some of the regulatory and scientific principles outlined in the draft guidance for consultation will also apply to individualised medicines that utilise other technologies or therapies that intend to treat other conditions including rare diseases. This is not akin to say that the same aetiology is involved between cancer and other, say, infectious diseases, but it seems to imply that some of the pathophysiological body response may be considered to behave similarly.
Anyway, the proposed draft guidance recently published by MHRA for comments from stakeholders envisages that in specific circumstances, an individualised medicine could be issued with a marketing authorisation (MA) under the HMRs, even where there is a variable component that is tailored to an individual patient’s characteristics. This means that a single MA could cover use across the target population defined in the indication. The production (including product design process, manufacturing site and process), strength, pharmaceutical form, and method of administration, would be otherwise identical between patient-specific batches.