Cell and Gene Therapies

The emergence of cell and gene therapies (CGTs) in the last decade has been one of the most captivating advancements in the field of life sciences. Chronic and fatal diseases that were once incurable can now be treated with a single administration. This almost resembles science fiction. While there have been numerous challenges to overcome when brining CGTs to market, we now have valuable reference points to learn from. Here are a few select lessons from CGT launches so far:

• Starting early: CGT trials are typically small, and as companies enter the clinical phase, commercialization is not far behind. This accelerates the traditional planning timeline.

• Generating evidence beyond pivotal studies: CGTs often come with remarkable long-term, potentially curative claims, but these claims are based on only a few years of clinical studies. It is crucial to consider early on how durability of response (and other data gaps) will be addressed through additional evidence generation activities.

• Access and pricing: Innovative access and payment models have been discussed since the inception of CGTs, but their implementation has been limited so far. The key lesson here is that there is no one-size-fits-all solution. Each payer system operates within its own legal framework, incentive structures, and cultural preferences. What may appear as a promising idea on paper (5-year annuity model?) may not find support from payers in the US, Germany, or France.

• Centers of Excellence (COE) model: CGT launches have been complex procedures that require specific expertise for successful administration. Identifying the right sites for administration becomes crucial. The primary criterion for site identification and prioritization is often expertise related to the product, overall procedure, patient population, and other CGTs. Once high-expertise sites are identified, the next step is determining the optimal number of sites to onboard to meet the demand adequately. In this case, less tends to be more: onboarding too many sites too quickly introduces execution risks, low volume per site, and suboptimal resource allocation.

• Manufacturing capacity: CGTs involve a complex system where many puzzle pieces need to align. This challenge has become evident with CAR-T therapies, where patients face significantly long wait times due to insufficient manufacturing capacity, despite demand, prescription willingness, and coverage.

• Patient support: Successful commercialization of CGTs goes beyond the therapy itself and encompasses the entire end-to-end process. For many launched CGTs, patients often need to travel to distant treatment centers multiple times and may have to stay for an extended period. This imposes substantial emotional, logistical, and financial burdens on patients and caregivers. It is crucial for manufacturers, especially in the US, to provide comprehensive support throughout this process.