Steps have also been taken to streamline the provision of relevant regulatory advice also for CGTs, with a single point of access for advice on regenerative medicine rules available from the Innovation Office within the Medicines and Healthcare Regulatory Authority (MHRA).
The MHRA has also published a draft tips on using real-world data (RWD) in clinical studies to support regulatory decisions. These tips apply to studies, regardless of their geographical location. The UK has also developed capabilities to carry out studies based on electronic health records (EHRs) for comparative effectiveness purposes.
A number of chords have already been achieved that provide patients with access to CGT in the UK. They include an accelerated agreement for Zolgensma, a unique gene therapy for spinal muscular atrophy and an access agreement managed through the Cancer Drugs Fund for the KTE-X19 CAR-T therapy for mantle cell lymphoma.
The British CGT ecosystem
CGTs are already widely recognized as the “next big therapeutic area” within the pharmaceutical industry. This is reflected in the wave of mergers and acquisitions over the past 12 months involving major pharmaceutical manufacturers such as Sanofi, Takeda and Novartis, to name a few. With this kind of attention, it is clear that CGT ventures will remain a major area of focus for biotech investors for some time to come, not least as the sector matures into its commercialization phase, the potential for generating income is extremely high.
The UK is already a thriving ecosystem for transformative treatments such as cell and gene therapy. According to a report by the Alliance for Regenerative Medicine and the BioIndustry Association there are over 70 companies developing Advanced Therapy Medicines (ATMPs) in the UK, and UK industry is expected to be worth £10 billion by 2035.
The UK is now a third country of the EU and as such for companies operating in the UK there have been regulatory changes which need to be taken into account in the development, manufacture and supply of CGT and ATMP more generally. The Catapult CGT published advice on the development and commercialization of CGTs and other ATMPs in the UK and EU after Brexit.
However, legislative and regulatory requirements are dynamic and evolving.
In the year ahead alone, we expect, and in some cases have already seen, major developments in areas of UK policy and regulation such as funding, taxation, data use, clinical trials, medical devices and the use of artificial intelligence tools.
It is incumbent on industry to take advantage of existing UK initiatives that support the development and commercialization of CGTs. They should also engage with ongoing reforms not only to understand their impact on their business, but also to seize opportunities, when they arise, to express their views on how policy, legislation , regulation and guidance could be shaped to provide the best possible ecosystem in which to operate.