In the human application sector, we license and inspect establishments that procure, process, store, distribute, import and export tissue for human use, or carry out donor testing. These include a range of organisations such as hospitals, stem cell laboratories and tissue banks that can use a variety of tissues and cells (such as bone, skin, heart valves and stem cells). It is a diverse sector where there has been considerable innovation and growth.
Avoiding delays in patient treatment
Under human tissue legislation, establishments are required to notify us before they start new licensable activities or vary methodologies. This is to ensure appropriate procedures and practices are safe for patients.
In 2023/24, we received 332 licence variation requests relating to patient treatment within the human application sector. While the majority were routine, seven requests were considered time-critical and prioritised as they had the potential to cause unnecessary delays to patient treatment. By collaborating at pace with establishments, we were able to rapidly authorise licence variations and enable patients to access the urgent care they needed. This included a hospital that sought to conduct urgent paediatric treatment within two weeks. To approve this time-sensitive request and expedite our assessment of their application, we met the proposed licensee to understand their needs and the planned procedure. After gaining necessary assurances, we approved the application within seven working days enabling the procedure to go ahead as scheduled.
In emergencies, we can directly authorise any person to distribute, import or export tissue for human use outside of the usual licensing framework. These unforeseen events can be brought about by factors such as a change in a patient’s circumstances or the availability of highly matched human tissue.
During 2023/24, we used our powers to provide direct authorisation on four occasions. To do this, we worked closely with establishments to gain assurances about the planned treatment, ensure any risks to patient safety were appropriately considered and managed, and ensure patients were appropriately informed and provided proper consent.
By prioritising urgent requests to amend licenses or providing emergency authorisations, we continue to ensure that establishments meet legislative requirements. This proactive approach also enables the use of innovative procedures without compromising patient treatment and care.
Facilitating innovation
We use our position and expertise to support licensed establishments use the latest technologies in accordance with human tissue legislation.
In the human application sector, we do this by adapting our licensing approach to keep pace with innovation and changing clinical practices. There were previously scenarios where a licensed establishment would use a remote site to carry out licensable activity, such as exporting human tissue. Customarily, we licensed both the establishment and remote site in line with our approach in other sectors. However, this model was not practical for manufacturing Advanced Therapy Medicinal Products (ATMPs; medicines for human use that contain genes, tissues and cells). Our typical licensing approach could also lead to additional costs for the sector and undue regulatory burden.
In response to this scenario, we worked with relevant establishments in 2023/24 to find appropriate solutions. This included the introduction of ‘hub and spoke’ licences where a central establishment oversees activities across remote sites and removes the need to license and inspect each remote site. This approach also ensures we continue to have robust operational and regulatory oversight – for example, by inspecting the central establishment to assure ourselves that remote sites are adhering to our standards through formal agreements. This model has most recently been used to transport human cells across the UK to develop CAR T-cell therapies (a type of cancer immunotherapy treatment that uses genetically altered immune cells to locate and destroy cancer cells).
By adapting our licensing model, we have supported the sector to continue innovating life and health sciences, without compromising on our need to maintain regulatory oversight.
Working with other regulators to support innovation
The Regulatory Advice Service for Regenerative Medicines (RASRM) was set up in 2014 to provide a ‘one stop shop’ to access free, clear, expert advice about the regulation of regenerative medicines.
To provide this advice, we work alongside organisations such as the Medicines and Healthcare products Regulatory Agency, Human Fertilisation and Embryology Authority and Health Research Authority. Our aim is to help researchers and professionals navigate the regulatory landscape and comply with associated standards in this complex area.
In line with our commitment to be an open, supportive and accessible regulator that supports innovation, we will continue to support the field of regenerative medicine and ensure safe access to innovative therapies. We will also continue to signpost establishments within the human application sector to expert groups and others working in the field, to help facilitate best practice and support across the sector.