The past year has marked a turning point for cell and gene therapies (CGTs), with the Food and Drug Administration (FDA) approving seven new therapies in 2023, bringing the total number of available treatments to 34. While the industry is slightly behind the FDA’s original prediction of 10 to 20 new approvals annually by 2025, there has been a significant uptick in approvals and growth in the pipeline.
This isn't just a numbers game – it shows that the industry is finally cracking the code on safety, efficacy, and scalable commercialization. However, challenges such as healthcare infrastructure readiness, manufacturing, scalability, high costs, and evolving market dynamics must be addressed as these therapies advance.
Below, we'll explore a few key trends shaping the commercialization of CGTs and strategies needed to navigate this rapidly changing landscape.
Shifting priorities: The growing importance of rare diseases and autoimmune therapies
Oncology still comprises the lion's share of cell and gene pipeline activity, but there's a noticeable pivot toward rare diseases and autoimmune conditions.
Why?
The science is evolving, and there is growing recognition that specific cell therapies, such as chimeric antigen receptor T-cell (CAR-T) therapy, have not fully met their initial high expectations. The market is moving fast, and cutting-edge gene-editing technologies are starting to eclipse older, less effective approaches.
The focus on autoimmune conditions marks a bigger trend: companies are reassessing the viability of early cell therapies and redirecting their resources toward these emerging therapeutic areas, which have huge potential for innovation and commercial success. New gene-editing technologies fuel this trend, offering more effective and commercially attractive options than alternative autologous cell technologies.
Infrastructure and healthcare system readiness
A key challenge in the commercialization of CGTs is the site readiness of healthcare systems to manage these complex treatments. Unlike traditional drugs, CGTs require specialized patient management, including ultra-cold storage/logistics and unique administration processes. This has spurred the distribution channel – including third-party logistics providers, specialty distributors, specialty pharmacies, and logistics and storage companies – to adapt their business models in order to support the unique characteristics of CGTs.
To ensure site readiness, consider the following:
- Infrastructure and storage: Facilities must have appropriate ultra-cold storage and infrastructure to effectively handle and administer these therapies. However, it's important to note that each CGT will have distinct handling requirements relative to the manufacturing and quality release standards. CGTs could have various temperature requirements, from liquid nitrogen to dry ice to regular refrigeration. The goal is to continue to streamline logistics, reduce costs, and make administration more accessible regardless of specialized handling requirements
- CGT brand-specific staff and patient training: Clinical staff must be trained on new protocols, while patients need clear guidance on the treatment process, potential risks, and follow-up care
- Regulatory and risk management: It is crucial to stay compliant with evolving FDA regulations and have risk management plans to address adverse reactions and post-therapy monitoring
- Alternative acquisition pathways and optionality: Given the high cost of CGTs, manufacturers must ensure that sites have optionality in their acquisition pathways, whether through a buy-and-bill model, external specialty pharmacy partnerships, or innovative payment schemes
Cost and value considerations in a shifting landscape
The high cost of CGTs, often reaching millions of dollars per treatment, remains a significant barrier to broader adoption. With the global CGT market projected to hit US $117.46 billion by 2034, driven by expanding treatment options and growing demand, addressing pricing challenges is critical. As these therapies advance, questions around who will bear the cost and how value-based pricing (VBP) models can be effectively applied become increasingly important.
Aligning cost with value is essential, where clear patient outcomes and benefits to the healthcare system balance financial investments. Artificial intelligence (AI) and advanced data analytics can play a crucial role in streamlining development and potentially lowering costs, thereby making these therapies more economically viable. The move toward VBP highlights the need for innovative pricing strategies that reflect the long-term value these therapies provide. Furthermore, careful patient selection ensures that these costly treatments are reserved for those most likely to benefit, optimizing both clinical outcomes and cost-effectiveness.
The role of AI in advancing commercialization
AI is still relatively new to the world of clinical trials and commercialization for CGTs, but it is already proving to be a game-changer in reducing costs and improving patient selection. One of AI's greatest strengths lies in its ability to quickly analyze vast datasets, which can improve patient selection by identifying individuals earlier in their disease progression and matching them with the right therapies sooner, boosting the chances of success. AI also optimizes trial design by predicting which patients are most likely respond to specific treatments, streamlining the development process, and cutting overall costs.
However, AI's impact extends beyond patient selection – it also addresses logistical challenges in producing and delivering these complex therapies. The production of CGTs is intricate, often requiring tailored processes and strict timelines. AI can help navigate these challenges by forecasting supply chain needs, optimizing manufacturing schedules, and ensuring timely delivery to treatment centers. This approach reduces waste, improves resource allocation, and ensures that therapies reach patients exactly when needed.
AI traceability is also becoming a critical component in the landscape of CGTs. This capability allows us to track and understand AI-driven decisions throughout the treatment process, from patient selection to therapy delivery. In a field that relies heavily on personalized treatments, traceability ensures patient safety by monitoring the chain of custody for cellular materials, aids in regulatory compliance with transparent processes, and supports the ongoing improvement of AI algorithms. It links data from preclinical studies to clinical trials and manufacturing records, allowing for long-term tracking of patient outcomes.
For example, in CAR T-cell therapy AI traceability can optimize manufacturing schedules while closely monitoring each step, from cell collection to final delivery, thereby enhancing both safety and efficacy. This level of oversight builds confidence in AI-driven processes and propels the advancement of CGTs.
The future outlook: Overcoming challenges and expanding access
The future of CGT commercialization hinges on tackling today's infrastructure and cost challenges while making these therapies accessible to more patients. To get there, pharma companies, healthcare providers, and regulators must collaborate closely to streamline delivery processes and build physician confidence. Sharing real-world successes and outcomes will drive adoption and trust among healthcare professionals.
The path forward isn't just about overcoming existing hurdles but also redefining what is possible. By confronting these challenges head-on and embracing the latest technologies, the industry can ensure that these groundbreaking therapies are not merely breakthroughs on paper but real solutions that reach the patients who need them most.