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

Colin M Young; Casey Quinn; Mark R Trusheim

doi:10.1016/j.drudis.2021.09.001

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

Drug Discov Today. 2022 Jan;27(1):17-30. doi: 10.1016/j.drudis.2021.09.001. Epub 2021 Sep 17.

Authors

Colin M Young¹, Casey Quinn¹, Mark R Trusheim¹

Affiliation

¹ MIT NEWDIGS, 77 Massachusetts Avenue, Cambridge, MA 02139-4307, USA.

PMID: 34537333
DOI: 10.1016/j.drudis.2021.09.001

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 93000 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.

Keywords: Drug development; Gene therapy; Health economics; Healthcare financing; Horizon scanning; Markov chain Monte Carlo simulation.

Publication types

Review

MeSH terms

Biological Products* / economics
Biological Products* / pharmacology
Drug Approval
Drug Costs / trends*
Forecasting
Genetic Therapy* / methods
Genetic Therapy* / trends
Humans
Molecular Targeted Therapy* / methods
Molecular Targeted Therapy* / trends
United States

Substances

Biological Products