Navigating RNA therapeutics: a new regulatory roadmap for innovators

Sarah Brockbank, Strategy Leader – Complex Medicines at Medicines Discovery Catapult, shares insight into a new regulatory roadmap created by the UK’s Intracellular Drug Delivery Centre (IDDC). Developed with input from the MHRA, the Quick Start Guide aims to support innovators working on RNA therapeutics and vaccines by simplifying the complex journey from research to commercialisation.

The UK’s Intracellular Drug Delivery Centre (IDDC) has created a new regulatory roadmap for innovators developing RNA therapeutics and vaccines – a new class of medicines based on ribonucleic acid. With input from the Medicines and Healthcare products Regulatory Agency (MHRA), the Quick Start Guide aims to simplify the journey from research to commercialisation.

The IDDC has delivered the resource in response to engagement with the sector, making the pathway to product registration clearer.

The roadmap aims to help innovators developing new therapies and vaccines understand regulatory guidance, how to identify the most relevant information to them and how to access support for bespoke interpretation of the guidance.

Drawing on sector-specific expertise within the MHRA, a decision tree on how to navigate nanotechnology-based products for medical applications has been incorporated. The guide helps innovators define their products and points to the relevant ICH guidance documents.

RNA therapeutics are one example of ‘complex medicines’, which can be challenging to characterise as they often consist of different but closely related structures rather than a single well-defined chemical entity. It can also be difficult to identify specific characteristics of the active moiety (the part of the molecule responsible for the action of the medicine) or its delivery system that might impact therapeutic performance.

This presents challenges for innovators and regulatory authorities alike, with established regulatory frameworks having to evolve to accommodate ever-diversifying complex medicines.

Part of the IDDC’s mission is to provide drug discovery innovators with the support they need to enable the successful translation of intracellular drug delivery technologies, from research to commercialisation. It is hoped that innovators developing the next generation of complex medicines find this guide a useful resource to help accelerate their progress.