Accelerating Time To Market: A Guide To CGT Commercialization
By Lung-I Cheng, VP and Head of Cell & Gene Therapy Service Line, AmerisourceBergen
Propelled by several landmark approvals and a robust pipeline, the cell and gene therapy (CGT) sector is entering a new era – with new modalities reaching the market and an influx of new therapies, including those that target larger patient populations on the horizon.
In the United States, there will be regulatory decisions on about 10 CGT products by the end of this year. And with more than 2,000 clinical trials underway globally, that figure is poised to grow in the next five to 10 years, offering tremendous promise for patients worldwide.
While the growing pipeline offers substantial potential, the path to commercialization is complex and lined with challenges that can delay market entry and impact product success. Successful commercialization requires timely orchestration among all stakeholders as well as careful planning and execution across a number of critical areas— patient access and support, regulatory strategy, supply chain design, market access, stakeholder engagement, and commercial distribution. A team of experts at AmerisourceBergen, where I work, developed a step-by-step guide to CGT commercialization to help developers overcome potential barriers and successfully bring their medicines to market.
Reducing Access Barriers
With CGT, several unique factors and challenges can impact access – from identifying eligible patients to complex reimbursement processes. While designing their pivotal trial, developers should establish a patient services strategy and a comprehensive plan for delivering their products to patients. The detailed roadmap, particularly critical for autologous cell therapies, will help developers and their partners anticipate and prepare for potential barriers and establish more efficient operations, ultimately helping expedite time-to-therapy.
Through a tailored but flexible patient service program, developers and their partners can reduce potential barriers across the treatment journey, simplify the care coordination process and enable an enhanced patient and provider experience.
Creating a Well-Defined Regulatory Strategy
Similar to patient services, developers should establish a regulatory strategy early in the clinical development process to prepare for scrutiny from regulators globally. A well-defined strategy will help to mitigate the risk of delays and optimize the pathway to reach the market efficiently.
While developing their plan, companies should consider three key components: comparative analysis, Chemistry, Manufacturing and Controls (CMC) framework, and master protocols. For example, the CMC framework requires comprehensive information about the manufacturing process, validation, data control, and analytical methods, underscoring the importance of thorough documentation and adherence to guidelines.
To navigate the regulatory landscape efficiently, developers can work with partners that can facilitate productive interactions with regulatory authorities, assist in the preparation and submission of regulatory documents, and support the design and execution of long-term follow-up studies.
Building a Robust Logistics Platform to Enable Secure, Reliable Transport
While the specific logistical requirements vary based on the product, CGTs generally require a heightened focus on three key elements: time criticality, chain of custody (COC) and identity (COI), and temperature-control storage and transport, such as cryogenic logistics.
For example, autologous cell therapies must move to and from a manufacturing site while maintaining COC, COI and Good Manufacturing Practices controls. These therapies also have rigorous temperature-control requirements and must be delivered within a narrow timeframe – and, in some cases, over a long distance. Cryopreservation of cell-based therapies can extend the critical timelines required for commercial delivery of both allogeneic and autologous therapies. However, some therapies require fresh transfer to ensure cell viability or product integrity, compressing acceptable transportation timelines.
Developers and their partners should build a comprehensive logistics strategy—tailored to the product’s unique requirements—that entails importation, cryogenic storage capacity, transportation capabilities, including dry shippers and contingency planning, and processes to maintain COI and COC. Through early planning and collaboration, CGT developers and their partners can overcome the unique logistical challenges across the clinical and commercialization journey and ensure these innovative therapies reach the patients who need them.
Demonstrating Value to Payers, Providers and Patients
Given the relatively high cost of CGTs, payers require sufficient follow-up time and evidence to assess their long-term value. Developers should initiate discussions with payers early to foster open dialogue, understand their expectations, and plan the evidence package accordingly. With their evidence package, developers should showcase the therapy’s potential benefits and justify its cost while also addressing the concerns and priorities of key stakeholders, such as:
- Clinical evidence: present robust clinical trial data showcasing the therapy’s durability, safety and efficacy;
- Cost-effectiveness analysis: Compare the therapy’s cost to that of standard of care and incorporate quality adjusted life year estimates;
- Patient perspective: Highlight the therapy’s impact on improving patient quality of life and reducing disease burden.
As part of the market landscape analysis, biopharma companies should actively seek input from providers and patients – in addition to payers – to address potential challenges. For example, patient advocacy groups can offer valuable insights into the patient journey. Meanwhile, providers need to understand which patients can benefit from specific therapies, how to direct them to appropriate care facilities, and how to provide ongoing support throughout their follow-up treatment.
Ensuring a Smooth Transition from Clinical to Commercial
Making the right choice in channel strategy is vital to achieve commercial success and ensure patients can receive the therapies they need.
Developers should answer five key questions to inform their distribution strategy:
- Where are patients treated today?
- What clinical and logistical considerations should they include in their Center of Excellence selection criteria?
- What is the ideal distribution network?
- What is the launch area?
- How might the strategy change over time?
The last question speaks to the importance of a channel strategy and distribution network that can evolve, as needed, based on new factors, such as receiving approval for additional indications and needing to expand to additional sites of care.
Capitalizing on the Momentum
The next decade holds tremendous promise for CGT. Based on the current pipeline, it’s possible that between 54 and 74 CGT products will be approved in the U.S., alone, by 2030.
But to achieve that potential and bring these innovative therapies to patients around the world, CGT developers must successfully navigate the challenges along the path to commercialization – from patient access and alignment on regulatory requirements to manufacturing complexities and market access. A successful CGT launch should involve cross-functional perspectives during strategy development. This entails initiating crucial conversations and engaging partners as early as possible, all the while comprehending the interdependencies between R&D and commercialization. Through early planning and collaboration, CGT developers can navigate the market and unlock the potential of their therapy.
You can access AmerisourceBergen’s CGT Commercialization Guide here: https://www.amerisourcebergen.com/insights/essential-guide-to-cell-and-gene-therapy-commercialization
About the Author
Lung-I Cheng, PhD, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen. In his role, Cheng is responsible for leading a unified CGT go-to-market strategy that addresses external customer needs and provides integrated solutions that deliver the right resources at the right time to position therapies for success. Before joining AmerisourceBergen, Cheng spent a decade in biopharma and most recently led Takeda Oncology’s global value and access team in cell therapies and pipeline. https://www.linkedin.com/in/lungicheng/