Technology governance and regulatory fragmentation in advanced therapies: the UK’s clinical network for CAR-T cell therapies targeting resistant cancers

The field of advanced therapies, which include cutting-edge techniques such as gene editing and cell therapy, has been recognized as one which imposes considerable challenges to regulators. This is so because these therapies bring about medical hopes to patients and clinicians fighting rare or resistant conditions, but are at the same time at an early stage of development and may trigger serious adverse reactions. One example of these regulatory challenges comes from the domain of CAR-T cell therapies: products derived from gene editing techniques and aimed to fight resistant types of cancer. These products have proven satisfactory and efficacious in clinical trials and early medical treatments but to a considerable proportion of patients, they have caused dangerous reactions which may require long periods of intensive care. As this is a very recent therapeutic domain, the regulation of the treatment has evolved alongside the development of products. In the UK, a clinical network has been formed to deliver CAR-T therapies. It is formed of hospitals able to sign off a strict accreditation process through which clinical competence and infrastructure readiness must be displayed. Currently, twelve hospitals have joined the network but a significant expansion is underway which will almost double the number of participating centres. Because of the regulatory and governance difficulties posed by this initiative, NHS England, the body responsible for its coordination, has mobilised a range of requirements to steer operations in the network. Apart from the national regulations that apply to advanced therapies in the country, four groups of standards are paramount here. First, there are professional standards formulated by institutions such as the accreditation committee of the European Society for Bone and Marrow Transplantation. Second, there are industry standards elaborated by the companies manufacturing CAR-T products. Third, there are technical standards, which are created by bodies such as ISO and manage to have considerable global diffusion. Finally, there are some clinical standards created by medical and pharmacy staff engaged in the CAR-T network. This proliferation of standards, as well as the interplay between standards and regulations in this domain, reveal important aspects of current technology regulatory governance. Some complex technologies emerge and are framed as a desirable societal and technological path. At the same time, the regulatory activity around them becomes fragmented, with a series of stakeholders formulating either de facto or de jure requirements. In these accelerated and fragmented scenarios, some markets can emerge and thrive while collective risks and liabilities are dealt with by means of specific contracts which are frequently kept as confidential. In this way, the field of advanced therapies in general, and the UK CAR-T cell network more specifically, open a space for the analysis of key regulatory governance challenges pertaining to regulatory innovation, efficacy and public accountability.