A 2018 report from the trade group PhRMA states that nearly 300 cell and gene therapies are currently in development. Cancer is the largest target with 111 agents in the pipeline, but a variety of other diseases are targeted as well, including hemophilia, neurologic disorders, autoimmune diseases, and even Alzheimer’s. While a sizeable portion of these therapies will likely fall off at some point in the development phase, the gene/cell therapy revolution appears to be growing rapidly. That may be a problem for payers and our current healthcare system, which is incentivized by short-term returns and immediate cost avoidance rather than long-term benefits of improved patient health.

In prior market research done by Precision for Value, 20 health plan and 5 IDN stakeholders were surveyed regarding their thoughts on gene therapy. Ideas on how to pay for cell- and gene-based therapies were almost as varied as the therapies themselves. Outcomes-based agreements were the clear winner, garnering support from nearly half of respondents, but a variety of other methods, including payment over time, payment following the patient, and high-risk pools, were suggested as well. These data indicate a market trying to come up with answers as a healthcare revolution speeds toward it.

Gene/cell therapy is here to stay. To justify the high cost, manufacturers need to communicate both short-and long-term, comprehensive value stories. This will require longer-term follow-up and data on durability. In addition, manufacturers should explore new payment models with payer partners, both government and commercial, to ensure therapy access to the patients that need them.