Key Lessons for Future Cell & Gene Therapies
By Heather Drexler, Anna May Fitch, Caleb Rhodes, Carolyn Zheng, Olivia Okoli, Seth Henks, Ellen Edenberg, and Bobby Moy
The first cell and gene therapy products have launched, but the development pipeline suggests a potential explosion in the space moving forward with hundreds of assets in clinical and preclinical development across a variety of platforms and indications. Cell and gene therapies have the potential to fundamentally reshape the treatment landscape for life-threatening disorders; however, there are many unique considerations about developing and commercializing these agents that cannot be learned from standard small molecules or biologics. Early decisions are critical in a space where demonstrating durable response is crucial, and significant investments may need to be made before traditional milestones are met.
To help our clients wrestle with strategic decisions in the growing cell and gene therapy space, we need to understand what attributes lead to a strong path forward for these complex approaches and why. Here, we offer some key lessons learned from the gene and cell therapies that have already launched.
- One-time/long-lasting therapies will result in new models of competition. Some disorders have multiple gene or cell therapies in development, which may also be competing with small molecule or antibody-based approaches. Particularly for therapies that have the potential to be curative, competition is critical even during clinical trials as there may only be one opportunity to treat each patient. A strong, clear vision of how and why product choices will be made is required to ensure meaningful differentiation among future cell and gene therapies.
- Patients and caregivers are key decision makers. Successful uptake requires focusing on the patient and their needs, including efficacy and safety, but also convenience, availability, route of administration, and more. While not unique to these approaches, an emphasis on education and clear messaging for patients is even more critical for gene and cell therapies given the complex science and potentially invasive administration for such approaches. Where patients or caregivers have a choice, understanding the optimal patient experience, including their needs and acceptable tradeoffs, is critical.
- Payment models for costly one-time potentially curative therapies are still evolving. The anticipated movement towards outcomes-based contracting or annuity payments will likely become better defined as new one-time therapy approvals expand into larger populations. Understanding an evolving reimbursement landscape across geographies is critical to ensure long-term success and sustainability.
- Risks of adverse events and long-term unknowns may leave the door open for second-generation therapies. Recent concerns around the safety of some cell and gene therapies have halted some ongoing clinical trials or commercial distribution. Efforts to make these agents safer may displace the earlier entrants in the clinic, especially for non-life-threatening disorders where patients are otherwise managed with chronic therapies.
- While safety and efficacy are paramount, manufacturing is also critical. At all stages of development and commercialization – through clinical trials, regulatory approval, and eventual adoption in the clinic – manufacturing influences product success. Preparing for commercial manufacturing early in the development process is imperative for successful launch and commercialization.
Understanding the above lessons will help to provide a strong path forward for companies developing gene and cell therapies. At Clarion, we look forward to continuing to help our clients navigate the complex and evolving space of cell and gene therapy commercialization to best enable life-changing treatments for those in need.