The UK is building a dedicated regulatory network to speed up the approval and rollout of advanced gene and cell therapies that can cure diseases rather than just treat symptoms. These therapies—known as Advanced Therapy Medicinal Products (ATMPs)—are rapidly moving from rare disease treatments toward more common conditions, with a wave of new approvals expected within the next decade. But current regulatory frameworks, designed for conventional drugs, cannot handle their complexity. Guidance is still being written, and global rules remain fragmented. Without a fit-for-purpose regulatory system, companies may take their clinical trials and product launches elsewhere, leaving UK patients without access to transformative cures. This network focuses on five priority areas: accelerating regulatory decisions, using real-world evidence, integrating patient-reported outcomes, speeding up clinical development, and embedding patient and public involvement. Each area includes practical projects to streamline how these therapies are assessed, approved, and reimbursed. If successful, the network will keep the UK competitive as a destination for ATMP innovation—ensuring that patients here are among the first to benefit from therapies that could permanently resolve conditions that currently require lifelong management.
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Advanced Therapy Medicinal Products (ATMP) are innovative therapies with potential to cure, rather than ameliorate, diseases addressing significant unmet needs. The ATMP field is rapidly evolving and growing, with a strong global preclinical and clinical pipeline that's broadening from rare to higher prevalence indications and a wave of approvals anticipated in the next 10 years, as well as numerous already approved and reimbursed therapies across many countries. The anticipated growth is expected to address significant global healthcare gaps. However, the unique and innovative nature of ATMPs results in challenges in regulating within traditional regulatory frameworks, with regulatory guidance still being defined and lacking global harmonisation; same challenges also apply for reimbursement frameworks and their interconnectedness with regulation. Advances in UK regulatory infrastructure is therefore critical to ensure that the UK remains attractive to companies to innovate, run trials and launch products and hence ensuring patients access to these transformative therapies. This network identified, through Discovery Phase collaborator workshops, focal areas for optimisation to enable timely therapy adoption and maximise UK competitiveness. The following five focal areas, termed "Clusters", have been prioritised for delivery in this Implementation Phase, where each Cluster consists of several work packages (WPs) covering a combination of regulatory science and innovation projects that aim to streamline and enhance the ATMP regulatory processes. Cluster address the following topics: Regulatory Acceleration, Real World Evidence, Patient-Reported Outcomes, Clinical Development Acceleration, Patient and Public Involvement and Engagement, Reference Standards
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