Operational and path dependencies impede the feasibility of hospital-based production of individually engineered...

Hospital-based production of gene therapies is a promising new approach for patients with high unmet medical needs, particularly those with rare diseases. However, operational and technical path dependencies (e.g. supply of materials, production timelines, and infrastructure limitations) and regulatory ambiguity impede hospitals from expediting implementation. We describe two distinct pathways including dependencies and uncertainty in manufacturing: complete in-house production and procurement of the active pharmaceutical ingredient. To realise the potential of hospital-based production at scale, future research should quantify infrastructure and staffing needs, analyse the alignment of production capabilities with patient demand, and scale up scenarios to improve delivery of therapies.