Regenerative medicines have made significant advancements in patient care, especially for rare diseases and small patient populations who no longer have to fear being told there are no other options. Unlike small molecules and even some biologics, cell and gene therapies have a more challenging road to commercialization and success for many reasons, including the nature of the modality – the promise that some therapies are one-time cures – and the price of such therapies – how do you simultaneously enable patients to access these expensive treatments, but also reward developers for innovation and investment in an arduous research process. These are all critical considerations as predictions have put 40-60 product launches in the US and over 500k patients treated by 2030, according to Peter Marks, the director of FDA’s Center for Biologics Evaluation and Research, and global sales forecasted at $13.23bn by 2023, based on estimates from market research agency ResearchAndMarkets.
Arguably, one of the biggest hurdles in getting cell and gene therapies over the finish line to becoming a sustainable...
