What it Takes to Bloom
Cell and gene therapies can live up to their potential – the field just needs to transform medicine as we know it.
James Strachan |
When the first CAR-T cell therapy, Kymriah, was approved by the FDA in 2017, Commissioner Scott Gottlieb said that cell and gene therapies “hold out the potential to transform medicine.” Words like “groundbreaking,” “revolutionary,” and “transformational” are often applied to new technologies. It’s all part of the familiar hype cycle, where we overestimate the effect of a technology in the short run and underestimate the effect in the long run (Amara’s law). And right now, it’s possible that personalized medicine, including cell and gene therapies, are riding high on the “peak of inflated expectations” following the first CAR-T approvals.
It’s true that the number of eligible patients for currently approved cell and gene therapies is miniscule when compared with the number of people who suffer from cancer or blindness (key areas for cell and gene therapy development) – and the field should brace itself for a spell in the “trough of disillusionment,” as expectations fail to live up to the hype in the short term.But will the fall in visibility be followed by the “slope of enlightenment” and an eventual “plateau of productivity” described by the hype cycle of Amara’s law?
Much will depend on whether or not researchers can find a way to selectively target solid tumors and expand the number of indications treated by cell and gene therapies. This, in the view of many researchers, is simply a matter of time. But there are additional, perhaps more challenging, problems that lie beyond the clinic; the field must rise to the challenges of commercialization, scale-up, logistics and cost.
Healthcare systems are set up to order, dispense, and pay for off-the-shelf products, but personalized medicines require new best practices and regulatory approaches. As things stand, it’s difficult to imagine how apheresis centers and pharmacies would be able to cope with dozens of new protocols as more therapies reach the market (as discussed on page 20).
Then there’s the question of pricing and payment. Will payers and companies be willing to embrace new pricing and reimbursement models? And even if the industry develops new, closed systems for manufacturing cell and therapies – eliminating the need for complex and expensive logistics – how will regulators cope with the prospect of individual hospitals manufacturing therapies? How will they ensure compliance and prevent processes drift?
In short, the field of cell and gene therapies will have to transform medicine to succeed. I am optimistic, but only time will tell if the field can overcome the hurdles.
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