Innovation in the round – the HTA’s cells and tissues partner discussion

In our last blog, the HTA outlined how we and our stakeholders have a clear role to play in achieving the governments’ Life Sciences Vision, together delivering a progressive UK regulatory offer that unlocks innovation while safeguarding patients and ensuring public confidence.  To do this, we need to constructively challenge our regulatory model and tools, looking at how we can continue to improve outcomes for patients and the public as we contribute to a system-wide approach that promotes the UK’s regulatory environment as an incubator for life sciences innovation.  

We identified the critical starting point for this as a productive and open dialogue with our life science partners, a conversation that does not fight shy of the challenges we face together while also keeping opportunities in focus.  

Our first roundtable this autumn, with  Anthony Nolan and NHS Blood and Transplant (NHSBT), showed the importance of getting issues on the table in areas where innovation pushes regulation to keep pace. Below we outline the key points from the discussion and how we plan to progress recommendations ahead of a series of roundtables in 2022 focused on other critical areas.  

Developments in Advanced Therapy Medicinal Products (ATMP) present significant opportunities in regenerative medicine and treatment but also demand reconsideration of donor welfare and regulation that supports this. The complexities that come with multiple pieces of cross-cutting legislation, licensing and best practice guidelines have the potential to create ethical ‘grey zones’ leading to Anthony Nolan and NHSBT using their own internal review procedures to protect donors and assess applications for access to tissue and/or cells. These internal review procedures put extra burden on the organisations as well as potentially conflicting with the developer’s governance structures. For example, current regulations cover activities relating to organs donated in a donor hospital and transported directly to recipient centres for transplant but they do not cover organs going to an intermediary location where they might be perfused and samples taken. This raises questions not only about sample ownership and consent but licensing under The Quality and Safety of Organs Intended for Transplantation Regulations 2012 (as amended).  

The discussion produced two core suggestions. One was a principles-based approach to donor welfare, regardless of the end use of the product. This would entail defining ATMP activities from procurement to use, helping to clarify which regulation an activity sits under. The other core suggestion was the need for international collaboration with other regulators to facilitate innovation in the cell and gene therapy space.   

Other recommendations and discussion points from this first HTA roundtable included: encouraging innovation through removing regulatory ‘grey zones’ and collaborating internationally; determining as early as possible in the ATMP application which regulator is responsible for which phase and involving these regulators early in the ATMP design, expeditiously identifying regulatory gaps and boundaries; continued collaboration, including through a regulation discussion forum, creating opportunities for regulators to work together and think about how regulation of ATMPs might be more agile and integrated.   

 In 2022, the HTA will continue these roundtables with the aim of ensuring that the right regulation is in place to build public trust in, and enable, future innovation. We need the input of our stakeholders to have the most productive discussions that produce recommendations that help take innovation and regulation forward.  

If you would like to find out more or have a suggestion for a roundtable discussion contact policy@hta.gov.uk