2023 cell and gene therapy industry survey

The survey results highlighted the top three critical aspects of clinical trial management: site selection/trial design, patient recruitment, and clinical trial management system. More than 35% of respondents chose site selection/trial design as the most critical aspect of clinical trial management for cell and gene therapies. On the other hand, contrary to CGT innovators and health care providers, CDMOs selected electronic data capture as the third most critical aspect of clinical trial management. Overall, the findings underscore the need to effectively manage clinical trials to optimize the development of CGTs.

The survey revealed that nearly 40% of respondents are only somewhat confident, slightly confident, or not confident in their current manufacturing capabilities to meet demand within the next two to three years. The lack of confidence is a result of the main challenges faced when scaling manufacturing, including the need for more access to inputs, the absence of standardized or proven manufacturing technology, and inflated cost of capital or limited access to capital.

• 71% of CDMOs highlighted the absence of standardized or proven manufacturing technology as the primary barrier to scaling up manufacturing
• 35% of respondents emphasized the critical role of good quality apheresed and processed leukopaks as a limiting factor in their manufacturing capacity

Furthermore, the survey results identified existing accreditations, prior experience collecting raw materials, and capacity as the top three factors driving the selection of apheresis centers. In fact, 33% of respondents selected existing accreditations as the most important factor driving apheresis center selection.

These results underscore the challenges and considerations associated with scaling up manufacturing in the CGT industry. Barriers such as limited access to inputs, lack of standardized technology, and capital constraints must be addressed. The dynamics of outsourcing within the manufacturing value chain highlight the reliance on external partners, particularly in plasmid production.

The survey results revealed the top three strategies CGT organizations were considering to support the commercialization of their products: seeking partnerships, raising capital, and commercializing independently. These results highlight two diametrically opposed strategies between organizations, with almost half (48%) seeking partnerships and one-third (33%) choosing to commercialize independently.

Clinical-stage innovators are more likely to seek partnerships (61%) and raise capital (54%) to support their commercialization efforts. In contrast, only 20% of commercial-stage innovators are seeking partnerships or raising capital for commercialization.

Looking ahead, respondents identified three key factors when considering the forces that will have the most impact on the commercialization of cell and gene therapies in the next 12–18 months: payer and reimbursement challenges, the ability to raise capital from public and private sources, and manufacturing capacity or quality consistency constraints.

One of the crucial technological capabilities for CGT companies is the ability to capture and use real-world evidence (RWE) data for more informed decision-making. The survey results revealed the top three barriers for RWE collection: the lack of biomarkers and surrogate endpoints to quantify efficacy, the burden associated with data collection over time, and unclear regulatory guidelines.

The survey results revealed that more than 33% of CGT innovators and CDMOs plan to establish their patient/therapy digital orchestration platform by leveraging partnerships. Another 33% of CGT innovators and CDMOs plan to develop end-to-end bespoke capabilities for their digital orchestration platform. The results demonstrate the varying approaches and priorities in embracing digital and technology advancements to improve patient-centric services.

The top three areas in the cell and gene therapy value chain that are perceived to have regulatory ambiguity are chemistry, manufacturing, and control (CMC); comparability and biosimilarity to reference products; and vector or plasmid guidelines. More than 40% of respondents selected CMC as the area of the cell and gene therapy value chain with most regulatory ambiguity.

The survey results revealed that 54% of CGT leaders either lack specific regulatory training or don’t have in-house regulatory expertise specific to cell and gene therapy. Developing and acquiring the necessary regulatory expertise and knowledge within organizations is crucial to meeting regulatory requirements and ensuring compliance. These insights underscore the importance of having clarity in regulatory requirements and the need to develop talent with this expertise within the CGT industry.