Overview
Commercialization of modern-day gene therapies is now a reality. Next-generation modalities such as chimeric antigen receptor T-cell (CAR- T) therapies are fully in launch mode in the US, with final approvals have also occurred in the EU. The first in vivo gene therapy for an inherited disease is also now available in the US. With a slew of gene therapies in the pipeline, across ex vivo and in vivo applications, and several indications including oncology and rare disorders, more treatments are advancing into later-stage development and onto potential regulatory approval over the next several years. Gene therapy developers can take advantage of a multitude of manufacturing guidelines on gene therapy in the US and EU.
Highlights
- The high, and potentially prohibitive, cost of gene therapies is the biggest challenge in pricing and reimbursing this class, along with the great uncertainty around the long-term benefit, especially of one-time therapies.
- Several novel payment mechanisms have been proposed to address the issue of price and uncertainty around long-term efficacy, but each carries its own set of advantages and disadvantages. Payers suggest some models, such as pay for performance, may work better than others.
- Hemophilia provides a good example of an area where companies may be able to clearly show how gene therapy could offer great savings and value compared with the standard of care, with payers agreeing that the longer and larger the amount of efficacy data available, the better.
- US payers suggested that it is possible they will drive utilization to one chimeric antigen receptor T-cell (CAR-T) therapy brand over another based on the average additional hospital costs incurred.
- European HTAs are not likely to be negatively impacted by single-arm trial design, but payers will pay close attention to endpoints and relevance in the real world.
- One of the biggest concerns for gene therapy developers will be securing an adequate supply of vectors for their products, with demand growing as a larger number of companies begin to scale out manufacturing for commercial production.
Key Questions Answered
- Of the key alternative payment models that have been suggested to address the high cost and uncertainty of gene therapies, which do payers believe offer the most potential?
- In what ways do payers practically see long-term patient follow-up put into place?
- What are the most important topics that manufacturers should discuss with payers to help aid in later assessments and negotiations?
- What are the key lessons learned from the first gene therapies approved in Europe?
- What unique unit operations in ex vivo autologous therapy have room for improvement?
- Which key company players have in-house and partnered manufacturing capabilities, and how are these beneficial?
Table of Contents
1. Overview
