The field of gene therapeutics is really beginning to deliver, with dozens of pipeline treatments promising potentially revolutionary advances in patient outcomes. However, the innovative nature of this emerging category presents formidable challenges for developers, regulators, payers and patients.
In this report, Clarivate experts look at the calculations stakeholders are making to move these products from bench to bedside, ensure safety and efficacy, understand their value and make them accessible to the patients that most stand to benefit from them.
Download the report to learn more about:
- How developers are solving for longevity of gene expression and immune evasion, and placing bets on multiple modalities
- How smaller companies are partnering with contract manufacturers to get up to speed
- The knotty problems of pricing and access companies are facing as they move beyond ultra-niche indications
- How payers are making risk-benefit and value assessments as they weigh coverage decisions around these expensive therapeutics
- The roles outcomes-based contracting and utilization management are playing in this evolving payer ecosystem
- How the regulatory landscape is shifting in response to these innovative treatments