Datamonitor Healthcare Strategy: Gene Therapy Commercialization – Opportunities and Barriers
Datamonitor Healthcare Strategy: Gene Therapy Commercialization – Opportunities and Barriers
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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 having also occurred in the EU.
Author: Amanda Micklus
Publisher: Datamonitor Healthcare / Biomedtracker
- Highlights
- Description
- Contents
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 a 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?
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 having 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 manufacturing guidelines on gene therapy in the US and EU.
CONTENTS
6 OVERVIEW
8 APPROVED GENE THERAPY LANDSCAPE
8 Datamonitor Healthcare insights and recommendations
8 Gene therapy: a field in existence for 50 years, but setbacks have slowed progress
8 Across the globe, 11 gene therapies are approved to date
14 Bibliography
15 PRICING AND REIMBURSEMENT OF GENE THERAPIES
15 Datamonitor Healthcare insights and recommendations
16 Therapy costs of select approved gene therapies
18 Certain challenges make reimbursement of gene therapies an issue
23 Hemophilia gene therapies may benefit the most in showing offset of costs
27 Payment options for gene therapies
35 Early engagement with US and EU payers would be beneficial
37 The use of registries and real-world evidence in long-term follow-up will be important for payers
40 Patient advocacy will be important in supporting access for gene therapies
42 Bibliography
48 US REIMBURSEMENT
48 Datamonitor Healthcare insights and recommendations
49 Value frameworks for gene therapy do not yet exist, but ICER’s role is growing
52 Commercial payer coverage policies for gene therapies
56 Medicare coverage of CAR-T therapies
59 Pricing for initial gene therapies in the US is perceived as high in some cases, lower in others
67 Bibliography
75 EU REIMBURSEMENT
75 Datamonitor Healthcare insights and recommendations
75 Progress of gene therapies has been slow because of price and access restrictions
78 The next big test in Europe will be CAR-T therapies
81 In HTA assessments, European payers are unlikely to penalize products with data from single-arm trials and/or small sample sizes
86 Bibliography
90 MANUFACTURING GUIDANCE AND VECTOR SUPPLY CONSIDERATIONS
90 Datamonitor Healthcare insights and recommendations
91 Regulatory guidance
95 Challenges and barriers in vector manufacturing and supply
105 Bibliography
112 ISSUES IN MANUFACTURING EX VIVO/CELL AND GENE THERAPIES
112 Datamonitor Healthcare insights and recommendations
113 With the process as the product, cell and gene therapies represent a major paradigm shift
114 Bedside CAR-T processing considerations
115 Process variability in raw/starting materials is of particular importance
117 Manufacturing logistics in CAR-T therapy
136 Bibliography
143 APPENDIX
143 Scope
143 Methodology
LIST OF FIGURES
13 Figure 1: Approved gene therapies by country, September 2018
14 Figure 2: Gene therapy pipeline by therapy area
19 Figure 3: Key challenges to reimbursement of gene therapies
41 Figure 4: Ethical concerns related to hypothetical gene therapy for brain-related conditions
51 Figure 5: The Alliance for Regenerative Medicine’s suggested value framework for gene therapies
52 Figure 6: Top-level additional inputs for payers to consider when assessing regenerative medicines
62 Figure 7: Kymriah and Yescarta are forecast to pass $2bn in peak sales, 2018–26
96 Figure 8: Gene therapy pipeline by vector category and viral vector type
114 Figure 9: Process and unit operations in autologous CAR-T therapy manufacturing
122 Figure 10: Heat map of Kymriah and Yescarta CAR-T therapy certified centers in the US, August 2018
134 Figure 11: Open versus closed systems: benefits and challenges
LIST OF TABLES
9 Table 1: Approved gene therapies worldwide, September 2018
17 Table 2: Therapy costs of select approved gene therapies
25 Table 3: Late-stage hemophilia gene therapies in the pipeline
28 Table 4: Financing and payment options for gene therapies
40 Table 5: Summary of the main recommendations on utilization of registry data in supporting regulatory benefit-risk evaluations for CAR-T therapies
53 Table 6: Major US payer restrictions or selection criteria on gene therapies
59 Table 7: Medicare outpatient rates for CAR-T therapies
63 Table 8: ICER discounted lifetime costs and outcomes for Kymriah and Yescarta versus comparators
63 Table 9: ICER threshold analysis for Kymriah and Yescarta
64 Table 10: Estimated total cost of CAR-T treatment at the low and high end
67 Table 11: ICER incremental cost-effectiveness ratios for Luxturna
76 Table 12: Market access decisions for approved gene therapies in the five major EU markets
83 Table 13: Select gene therapies in the pipeline with pivotal single-arm trial designs
91 Table 14: Summary of EMA guidance on gene therapy manufacturing
94 Table 15: Summary of FDA guidance on gene therapy manufacturing
99 Table 16: Select group of CMOs and CDMOs with gene therapy vector supply capabilities
101 Table 17: In-house and external cell and vector manufacturing efforts by select gene therapy developers
117 Table 18: Vein-to-vein time comparison for CAR-T therapies
120 Table 19: Select companies that specialize in cell and gene therapy biopreservation
123 Table 20: US treatment centers for Kymriah and Yescarta, August 2018
131 Table 21: Select companies specializing in software tracking tools for cell and gene therapy
132 Table 22: Potential innovations in improving scale-out of cell and gene therapies
135 Table 23: Select companies that offer closed cell-processing systems or technologies
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