Benefits and Challenges of Transitioning From a Centralised to a Decentralised Model for the Manufacturing of Investigational Cell&Gene Therapy Medicinal Products

Cell&Gene therapies can respond to therapeutic unmet needs, but they are often difficult for patients to access. The development of a decentralised model for the production of Cell&Gene Therapies at the patient's bedside to support clinical protocols, treatment in hospital exemption and also the widespread distribution of authorised products, can be a valid tool for putting the patient's needs back at the centre.

Although the current Cell&Gene therapies available on the market have demonstrated a disruptive effectiveness in treating orphan diseases, only a very small proportion of patients can access the treatment. The imbalance between supply and demand is magnified in minority and vulnerable populations. Limited access is multifactorial and in part a result of factors directly related to the cellular product such as cost, complex logistics and manufacturing limitations.

The recent possibility of having multi-product manufacturing systems that are fully automated and effectively connected to a central site for process supervision and batch release allows clinical centres to have fresh, highly effective products available and to focus on the patient's needs.

Decentralised production with closed, automated systems at the patient's bedside also helps to reduce manufacturing costs, address treatment failures related to complex logistics and sterility failures.

The speaker has worked actively in a central site for the coordination of decentralised production and its authorisation by a European regulatory authority. She also supports hybrid models of decentralised production in the USA with central production logistically located to serve numerous clinical centres.