Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System

Casey Quinn; Colin Young; Jonathan Thomas; Mark Trusheim; MIT NEWDIGS FoCUS Writing Group

doi:10.1016/j.jval.2019.03.014

Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System

Value Health. 2019 Jun;22(6):621-626. doi: 10.1016/j.jval.2019.03.014. Epub 2019 May 17.

Authors

Casey Quinn¹, Colin Young², Jonathan Thomas², Mark Trusheim²; MIT NEWDIGS FoCUS Writing Group²

Affiliations

¹ Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA. Electronic address: caquinn@mit.edu.
² Center for Biomedical Innovation, Massachusetts Institute of Technology, Cambridge, Massachusetts, USA.

PMID: 31198178
DOI: 10.1016/j.jval.2019.03.014

Abstract

Objectives: To estimate, at the indication level, durable gene and cellular therapy new product launches in the United States through 2030, and the number of treated patients.

Methods: A statistical analysis of clinical trials pipeline data and disease incidence and prevalence was conducted to estimate the impact of new cell and gene therapies. We used Citeline's^® Pharmaprojects^® database to estimate the rates and timing of new product launches, on the basis of the phase of development, duration in phase, and probability of progression. Disease incidence and prevalence data were combined with estimates of market adoption to project the size of reimbursed patient populations.

Results: We project that about 350 000 patients will have been treated with 30 to 60 products by 2030. About half the launches are expected to be in B-cell (CD-19) lymphomas and leukemias.

Conclusions: Cell and gene therapies promise durable clinical benefit from a single treatment course. High upfront reimbursement for these products means that the total costs could exceed what the healthcare system can manage. This creates a need for precision financing solutions and new reimbursement models that can ensure appropriate patient access to needed treatments, increase affordability for payers, and sustain private investment in innovation.

Keywords: cell and gene therapies; drug pipeline; financing healthcare; managed care; pharmaceuticals.

MeSH terms

Cell- and Tissue-Based Therapy / economics*
Cell- and Tissue-Based Therapy / methods
Cell- and Tissue-Based Therapy / trends
Economic Development / trends
Genetic Therapy / economics*
Genetic Therapy / methods
Genetic Therapy / trends
Humans
United States