Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues

TitleDurable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues
Publication TypeJournal Article
Year of Publication2022
AuthorsYoung, C, Quinn, C, Trusheim, M
JournalDrug Discovery Today
Volume27
Issue1
Start Page17
Date Published01/2022
Other Numbersdoi.org/10.1016/j.drudis.2021.09.001
KeywordsDrug development, Gene therapy, Health economics, Healthcare financing, Horizon scanning, Markov chain, Monte Carlo simulation
Abstract

Durable cell and gene therapies potentially transform patient lives, but payers fear unsustainable costs arising from the more than 1000 therapies in the development pipeline. A novel multi-module Markov chain Monte Carlo-based model projects product-indication approvals, treated patients, and product revenues. We estimate a mean 63.5 (54–74 5th to 95th percentile range) cumulative US product-indication approvals through 2030, with a mean 93 000 patients treated in 2030 generating a mean US$24.4 billion (US$17.0B–35.0B, US$73.0B extreme) list price product revenues not including ancillary medical costs or cost offsets. Thus, the likely dozens of durable cell and gene therapies developed through 2030 are unlikely to threaten US health system financial sustainability