Related Experiment Video
Updated: Jun 11, 2025
Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
Published on: February 15, 2019
The health economics of cell and gene therapies
1Health Economics and Market Access, Cell and Gene Therapy Catapult(†), London, United Kingdom.
Abstract:
The health economics of cell and gene therapies is complex; due to resource-intensive manufacturing, high prices are required for commercial viability that are challenging for healthcare systems to accommodate. Despite high prices, cell and gene therapies can provide value when they deliver substantial clinical benefits and displace long-term healthcare costs compared with existing treatment options. In this chapter, the cost utility approach of economic evaluation is discussed, focusing on the considerations that occur more commonly in cell and gene therapies compared to conventional medicines, how these considerations create challenges in interpreting the evidence and coming to conclusions on value, and what tools exist for understanding the level of decision uncertainty. A summary of the economic evaluation of onasemnogene abeparvovec for spinal muscular atrophy is provided as a real-world example that features the considerations discussed.
Related Concept Videos
00:59Gene Therapy
00:49What is Genetic Engineering?
00:57Embryonic Stem Cells
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
01:22iPS Cell Differentiation
01:58Cell Specific Gene Expression
02:49Cancer Therapies
However, cancer treatments can pose several challenges, as therapies used to kill cancer cells are generally also toxic to normal cells. Moreover, cancer cells mutate rapidly and can develop resistance to chemical agents or radiation therapy. Besides, all types of cancer cells may not respond to the same therapy. Some cancer cells respond to one...