A thoughtful overview from Dr Richard Zajicek (Medicine and Process Delivery Leader, GSK) at the CAR-TCR Europe Summit2020, outlining the merits of an integrated plan and close collaboration across CMC and Clinical, to accelerate delivery of cell therapies to the clinic setting.

Understanding your fundamental philosophy of approach is crucial to ensure clear thinking and decision points. Are you adopting a ‘fast fail’ strategy, where the goal may be to assess proof of concept around safety and efficacy? Alternatively, is it about building for the future and armour plating your phase 1, to use as a foundational stepping stone?

Either way, Richard advocates for the logical and well established integrated assessment processes used as standard in the bio pharmaceutical industry, to be applied to gene and cell therapy development to accelerate decision making and efficient investment allocation. A series of core questions should then be considered including: what is the minimum efficacy to enter into the clinic setting in the first phase?; how do you transition to 2nd and 3rd generation points?

An overarching theme advocated by Richard was to engage Payers and Regulators for scientific advice, early and often, to support target product profile development and rationale for changes to approach, on the basis of the science and potential improved outcomes.