Market access for cell and gene therapies: defining value for one-off treatments

An Expert View from Georgi Makin, Editor, Phacilitate.

It’s very easy to be enamored by the promise of cell and gene therapies – the one-dose treatments that can cure some of the cruelest of diseases – but there remains a gap between scientific prowess and feasible, marketed therapeutics, and everyone knows it. Scientific advancement is exceeding market and reimbursement expectation, with some markets more prepared than others for meeting demand for both the companies ready to launch approved therapies, as well as patient populations.

The contentious issue of ‘market access’ lies in the fact that one day, every successful developer must eventually cross the barrier from lengthy development, arduous approval and into successful delivery of a therapy on a commercial scale, satisfying the cost of production and meeting a defined value proposition. It then falls to ‘payers’, or bodies responsible for reimbursing this process, to have the ultimate final decision for entry into a specific market.