Paying For Gene Therapy: Biggest Cost Challenges Two To Three Years Away?
Only about one-quarter of the 40 gene therapies projected to launch by the end of 2025 would have potential US patient populations of more than 50,000. But a handful of treatments could target millions of individuals.
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Based on the current pipeline of durable therapies, MIT research indicates ‘it’s not a big lift to cover the children and the adults in Medicaid for these products,’ Mark Trusheim says.
The partnership worth up to $1.75bn removes the significant risk of going it alone in a competitive market as Regenxbio awaits late-stage data for its gene therapy.
US FDA is ‘willing to look at anything’ but sponsors need to demonstrate an effect on pain as drug development challenges remain. Accompanying chart describes 32 drugs in Phase II and Phase III clinical trials and one with a pending biologics license application.