Lawrence Tallon
CEO, Medicines and Healthcare products Regulatory Agency (MHRA)
If penicillin’s discovery marked a turning point in medicine in the twentieth century, then the development of cell and gene therapies, turbo-charged by AI, could be the defining moment of the early twenty-first.
The evolution of these new therapies represents a paradigm shift in how we treat a wide range of genetic disorders and diseases, including cancers and rare diseases. While welcome, it also presents scientific questions about how we support development, evaluation and approval of these therapies to ensure their quality, safety and efficacy.
How can regulation support pioneering new treatments?
As the UK’s regulator for medicines, medical devices and blood products, we need to keep pace with innovations that can best meet patients’ needs. That’s why we’re evolving new regulatory frameworks and guidance to facilitate the development of these new technologies as soon as we can be confident of their safety and efficacy.
This new approach will cut years from the traditional development
timescale without compromising safety
For example, we recently conducted a consultation on a proposed new framework to streamline the development, licensing and post-market oversight for therapies targeting rare conditions.
The new Rare Disease Pathway will allow more flexibility for the approval of rare disease therapies where conventional approaches aren’t feasible. This new approach will cut years from the traditional development timescale without compromising safety.
How does regulation benefit patients?
Our guiding principle is always patient safety first, while facilitating access to new medicines and technologies that can transform lives, as quickly and safely as possible. We recognise that regulation needs to change and evolve to enable innovation that benefits patients, rather than be a barrier to progress.
The UK has an opportunity to lead the world in gene therapies — thanks to our single national genomics provider, the NHS as a single-payer system with rich and diverse datasets, diverse population and strong academic base.
Our new regulatory approaches will make gene therapy development more commercially viable and help to make the UK a global leader in life sciences. The MHRA is breaking new ground in regulatory science globally, playing a key role in harnessing cutting-edge science and technology, helping to lead innovation for the benefit of patients and protecting public health.
