On CAR-Ts, decentralized in-house models, and the hospital exception. Routes for sustainable access to innovative therapies.

Chimeric Antigen Receptor T cells, or CAR-Ts, are a novel class of gene and cell 'one-shot' therapies based upon collecting, reprogramming, and using patients' own immune cells to treat their cancer. The article discusses the status, prospects, and some relevant legal issues of this frontier of personalized medicine. In particular, it explores the legitimacy of 'in-house' CAR-Ts, ie treatments manufactured and delivered to patients within the same clinical center by relying upon automated cell processing systems, a decentralized model which is very different from the one currently adopted for manufacturing existing commercial CAR-Ts. A few legal routes are envisioned for legitimately developing CAR-Ts within decentralized, non-commercial operational sets. In more detail, the article explores, firstly, the issue of 'academic' CAR-Ts (ie therapies developed and administered to patients as experimental drugs). A focus is then provided on what is known as the 'hospital exception' (HE), a special feature of current EU pharmaceutical regulation for non-routine preparations of custom-made advanced therapy medicinal products. Conclusions support a regulatory convergence on shared models of decentralized manufacturing, also through a broader and clearer application of the HE, to enhance a virtuous complementarity between in-house autologous and commercial allogeneic CAR-Ts, for the benefit of patients, pharmaceutical R&D, and sustainable healthcare systems.