Ned Sharpless weighs in on gene therapy pricing debate, suggesting 'the messaging got lost'
PHILADELPHIA — You know the drug pricing debate has reached a new height when the acting FDA commissioner starts to chime in on a public forum.
Ned Sharpless NIH In response to a question on gene therapies posed during a fireside chat at the annual BIO convention, Ned Sharpless acknowledged the importance of discussing cost — though it doesn’t fall under the FDA’s purview — but said he’s “a little disappointed” in the coverage of the recent approval of Novartis’ spinal muscular atrophy treatment, Zolgensma. The treatment was priced at $2.12 million spread out over a 5-year installment.
“This is a completely novel, almost magical miracle that ends a devastating disease for lots of little kids and the thing you care the most about is the price? I mean, really? If you’re so cynical you can’t see how wonderful and great that is […] you need to re-wear your happy hat,” he said.
Then he hinted at how the price of these “shockingly expensive” therapies might eventually come down as manufacturers optimize their processes.
“You have to remember when we first made penicillin, it was so expensive to make,” he said. “They would filter the urine of the people who’d take it and crystallize it. We got better at making penicillin eventually.”
The topic was brought up again in the Q&A session when a patient advocate in the audience — whose 8-year-old son has received $3 million worth of other treatments for SMA — asked how the “messaging got lost.”
Here’s Sharpless again:
Thank you so much for making that point. Because I think I’m with you. I think the messaging got lost. I was sort of imagining, it’s like […] at that wedding that Jesus was at where he turned water into wine, there was probably somebody who said hey, you know I wanted Chardonnay! This is red wine! […] But there is a legitimate issue here which is you know we have 40, 50, I don’t know how many INDs of these novel therapies in CBER and how are we going to support all of these things that get developed for these ultra rare indications and the society is going to figure that out.
The FDA is doing its part by coordinating with the CMS on specific areas such as antibacterials and CAR-T, integrating their work to the extent possible, he mentioned earlier in a dialogue that ranged from opioids and stem cell clinics to Alzheimer’s and depression.
Image: Ned Sharpless. Tom Williams CQ Roll Call AP
