Overcoming six challenges in commercialising cell and gene therapies

Cell and gene therapies (CGTs) are living up to their promise, with more than two dozen treatments licensed from the FDA’s Office of Tissues and Advanced Therapies in areas such as cancer and hematology. Hundreds more CGTs are moving through research pipelines, signaling the arrival of a new treatment paradigm.
To date, however, many CGTs have failed to meet their commercial goals due to missteps that might have been avoided with earlier strategic planning and execution. Emerging pharma companies tend to focus on regulatory hurdles at the expense of formulating early and robust commercialization strategies. When therapies fail post-launch, there are impacts beyond the loss of shareholder value. Patients may be deprived of potentially life-altering treatments. And without a critical mass of commercial successes, it is difficult for industry to sustain scientific and engineering innovation.
How can we ensure that a greater percentage of these important products meet their commercial goals? Syneos Health recently analyzed a cohort of FDA-approved CGTs that met with widely divergent levels of success. In the process, we identified six key commercial challenges, along with preemptive measures and actions companies can take to mitigate the obstacles. These key learnings are summarized below. For a more detailed discussion of the challenges, along with relevant data and use cases, please explore our whitepaper titled Success Factors for Commercializing Cell and Gene Therapies.

Challenge #1: Complex value proposition
Companies often fail to fully grasp the types of pre-approval and post-approval data required by key stakeholders including payers. Without such understanding, it’s hard to accurately assess and drive belief in the benefit/risk profile of a novel CGT.
• Strategic imperatives:

• Develop an integrated evidence generation plan leveraging market insights early in development and updating stakeholders as additional data becomes available.
• Explore opportunities to leverage systems (e.g., hubs) to compliantly generate real-world evidence.
• Establish a platform narrative to tell the long-term value story (including plans to build infrastructure, lifecycle management and other factors).

Challenge #2: Paradigm shift from chronic treatment to potential cure
Product innovators realize that the emergence of CGTs has altered referral networks and administration/process requirements, emphasizing centers of excellence (COEs) and in some cases limiting utilization. However, innovators need to think holistically to address this paradigm shift in the patient journey.
• Strategic imperatives:

• Help HCPs change how they think about “patient ownership” and educate them on the changing role of monitoring and management.
• Increase HCP comfort level with referrals to COEs and assist in developing referral networks while advancing regional high-value influencer engagement and promotional efforts.

Challenge #3: Burdensome Site Setup
IT system set-up requirements including mandatory certifications can be a significant burden, and the challenge grows as medical teams juggle systems for more than one CGT based on differences in how they are stored, extracted or infused.
• Strategic imperative:

•  Reduce system setup burden where possible by integrating existing IT systems/platforms that are already in use.
• Consider the need for enhanced provider training to improve ease of use.

Challenge #4: Uncertainty around addressable population
Patient identification can be a challenge, especially in rare diseases. Even in established disease areas, it may be hard to determine the right patient type in conformance with reimbursement considerations, guidelines and availability of existing standard of care therapies. Eventually this may present hurdles to anticipating potential uptake in a fast-evolving market.
• Strategic imperatives:

• Minimize patient drop-off/leakage by understanding the overall patient journey and referral patterns.
• To avoid “stock-out,” develop a robust forecast based on understanding uptake across patient profiles, optimizing manufacturing and inventory and other measures.

Challenge #5: Manufacturing, administrative and regulatory/compliance hurdles
CGTs are more individualized than other treatments, requiring closer collaboration between HCPs and manufacturers. This draws additional regulatory and compliance scrutiny regarding information flow. Delays in treatment delivery may arise from longer manufacturing turnaround times. And shifts in patients’ clinical condition between diagnosis and the start of treatment sometimes render them ineligible for the therapy. Overall, complex logistics plus lack of process familiarity at some centers may limit CGTs availability.
• Strategic imperatives:

• Reduce turnaround time from patient sample collection to product administration while maximizing manufacturing efficiency.
• Build a system/platform for hospital ordering and tracking to facilitate information exchange.
• Set up an operating model with an optimum mix of field personnel to support every step of the process.

Challenge #6: Reimbursement hurdles linked to high therapy costs
Payers have voiced concerns about high wholesale acquisition costs for CGTs, especially in the absence of long-term data. Medicare has shown interest in bundled diagnosis-related group (DRG) approaches to hold down costs on CAR-T and other CGTs, but these strategies may not be sufficient, and hospitals may be reluctant to take on risk.
• Strategic imperatives:

• Consider innovative reimbursement models, including value-based approaches to help address cost concerns.
• Early in the development cycle, take stock of payer expectations for coverage and reimbursement to ensure key elements are collected as part of evidence generation.

The hurdles discussed above can diminish the value CGTs bring not only to treatment developers but also to patients. Commercial strategies at large pharma companies have evolved over time to meet these challenges, bringing many benefits to stakeholders. With the right strategic partnerships in place, companies can anticipate hurdles and emulate strategies that some larger players have implemented with success. At Syneos Health, our mission is to partner with ambitious CGT innovators, sharing and disseminating insights and best practices in this fast-evolving field.