This blog uncovers the critical strategies, innovative distribution models, and key considerations that are driving the rapid transformation of cell and gene commercialization and ensuring these groundbreaking treatments reach patients effectively.

The Current Landscape

Cell and gene therapy (CGT) is emerging as one of the most dynamic and innovative fields in the pharmaceutical industry. The journey began in 2017 with the groundbreaking approval of Kymriah (tisagenlecleucel), a CAR-T cell therapy for acute lymphoblastic leukemia. Since then, the pace has only quickened. From 2017 to 2022, seven gene therapies were approved, with another five approvals in 2023 alone. Now, there are over 20 such products on the market, with many more expected by year-end, showcasing the remarkable strides and potential of this sector.

Strategic Planning & Early Engagement

In the CGT space, early engagement in the commercialization process has become a pivotal trend. Strategic partnerships with pharmaceutical companies are being formed up to 36 months before an anticipated product launch. This proactive approach is essential for navigating the intricate regulatory and distribution channels, allowing companies to foresee and address potential challenges, thereby smoothing the path to market. Moreover, early engagement enables better alignment of manufacturing capabilities and streamlining of supply chain logistics, ensuring faster patient access to these revolutionary treatments.

Innovative Distribution Models

The distinct characteristics of cell and gene therapies, including their high value, often one-time administration, and specialized handling needs, have spurred the creation of CGT-specific distribution models:

• Flash Title Model: This involves an intermediary (like a 3PL or Specialty Distributor) assuming ownership of the product title without physical possession during the supply chain. The manufacturer and distributor agree on who retains title and risk until the product reaches site of care. This model helps alleviate receivable risk and ensures safe delivery through specialized couriers.
• Specialty Pharmacy Drive-by Model: Here, the product stops briefly at a specialty pharmacy for verification and relabeling before heading to the treatment center. This is particularly relevant for high-value gene therapies administered in outpatient settings, as it ensures product integrity and proper handling while maintaining manufacturer control over delivery and offering an alternative channel option for treatment centers.

Challenges & Solutions in Government Pricing

Navigating government pricing for CGT requires strategic foresight due to the high costs and unique nature of these treatments. Early engagement in government pricing programs may be crucial for broad market access and understanding how commercial discounts may impact government pricing is critical to a successful strategy. Key considerations include:

• Government Programs: Understanding timing for when cell and gene therapies will be added to existing government contracts or government contracting timelines for emerging manufacturers is important to ensure broad market access.
• Commercial Discounts: Evaluating the financial impact of discounting scenarios for Medicaid, VA-FSS, 340B, Part B Average Sales Price (ASP) and IRA is necessary for projecting and understanding gross to net.
• Outcomes Based Agreements (OBAs): It’s important to proactively evaluate if a single Medicaid best price or multiple best price approach will be used for OBAs and how ASP may be impacted by OBAs.

Key Considerations for Industry Experts and Manufacturers

• Regulatory Compliance: Timely compliance with regulatory requirements is vital for successful commercialization. This includes meeting FDA approval standards and navigating state licensing and compendia submissions.
• Market Access: A comprehensive market access strategy is essential, involving understanding payer landscapes, reimbursement pathways, and patient support programs to enhance treatment accessibility and affordability.
• Data Management and Integration: Advanced data management systems are crucial for tracking product distribution, patient outcomes, and compliance metrics, streamlining operations and improving decision-making.
• Sustainability and Scalability: As the CGT market grows, maintaining sustainability and scalability in production, distribution, and patient care processes is critical. This involves optimizing manufacturing processes and ensuring a robust supply chain infrastructure.
• Patient Population and Site of Care Shifts: As these therapies expand to include more common conditions, there is a corresponding shift towards treatment in outpatient settings. Larger patient populations need access to environments like community practices, requiring different approaches and reimbursement models for pharmaceutical companies.

By concentrating on these areas, cell and gene therapy manufacturers can better navigate the complexities of commercialization, ensuring that these groundbreaking treatments efficiently and effectively reach patients.