Transforming Medicine: Strategies in Cell and Gene Therapy Development
Blog
Nov 13, 2024

Authors

Erin Finot, MS, MBA, Vice President, Immuno-Oncology and CAGT
Diego Correa, MD, PhD, Vice President and Global Head, Cell and Gene Therapy Center of Excellence

Cell and gene therapies (CAGTs) are rapidly changing the treatment landscape for genetic disorders, with significant advancements reflected in the surge of FDA approvals—38 therapies to date, including seven in 2023. Over 2,000 global trials are underway, with more than 50 new products expected in 2024, showcasing a promising horizon for patient care.

However, as CAGT innovations accelerate, so too does the complexity of the regulatory landscape of clinical research. Effective CAGT development requires careful attention to:

  1. Regulatory Approvals: Understanding varying jurisdictional requirements can affect timelines and documentation.
  2. Environmental Considerations: Awareness of local regulations is crucial for product distribution.
  3. Documentation Needs: Unique requirements must be anticipated during development.
  4. Long-term Safety Follow-up: Extended monitoring is essential for assessing therapy impacts.

In addition to regulatory complexity, CAGT clinical programs also face hurdles with:

  • Trial Design: Innovative approaches, including adaptive designs, are necessary for patient retention and statistical validity.
  • Patient Identification: Developing registries and prescreening protocols helps minimize screen failures.
  • Execution Logistics: Early attention to manufacturing and distribution can streamline therapy delivery.
  • Long-term Studies: Comprehensive planning for follow-ups ensures high-quality data collection.

Assessing a Country’s Capacity for Cell and Gene Therapy

In the UAE, the adoption of cell and gene therapies faces several hurdles:

  • Regulatory Harmonization: Aligning local regulations with international standards (like those from the EMA and FDA) is essential for ensuring that therapies meet safety and efficacy benchmarks.
  • Infrastructure Investment: Significant investments are needed to bolster healthcare facilities, ensuring they can handle the complexities of advanced therapies.
  • Public Education: Educating patients and the general public about the benefits and risks of these therapies is crucial for fostering acceptance and understanding.
  • Collaboration and Public Awareness: Collaborative efforts among government bodies, healthcare providers and educational institutions will enhance awareness and understanding, facilitating smoother adoption.

To address these challenges, countries who strive to implement cell and gene therapies should consider several factors to ensure readiness:

  1. Patient Availability: The success of any therapy hinges on the patient population's accessibility and willingness to participate in trials or treatments.
  2. Operational and Clinical Infrastructure: A robust healthcare infrastructure is essential for the administration and management of complex therapies like CAR T and gene therapy.
  3. Regulatory Landscape: Harmonizing regulations with established entities like the EMA and FDA is crucial to facilitate a smooth introduction of therapies.

Logistical Barriers: Efficient supply chains and logistical frameworks must be in place to handle the complexities of these advanced

Applying insights from CAGT to Immuno-Oncology

Transitioning from traditional oncology to a more integrated approach that includes immunology presents unique operational challenges. These can range from resource allocation to the need for specialized training. Healthcare facilities must invest in the necessary infrastructure to support immuno-oncology research. This involves setting up state-of-the-art laboratories capable of handling complex immunological assays and increasing data analytics capabilities for real-time patient monitoring. Facilities must also ensure they have the necessary equipment to safely manage and administer advanced therapies, which may involve building new clean rooms and updating existing spaces to meet stringent safety standards.

The move to integrate immunology into oncology practices demands an overhaul of training programs. This includes not only educating oncologists about immunological principles but also cross-training immunologists in cancer-specific challenges. Forming interdisciplinary teams of oncologists, immunologists, researchers, and nurses is key. Regular communication within these teams fosters a collaborative atmosphere, ensuring coordinated patient care that leverages the strengths of each discipline to enhance treatment outcomes.

Key takeaways from CAR T therapies

As we look to apply insights from CAR T therapies, which are a type of CAGT, to broader immuno-oncology strategies, several key takeaways stand out:

  1. Personalization: CAR-T therapy takes an individualized approach by using a patient's immune cells to fight cancer. This personalized method not only targets cancer more effectively but also reduces side effects, making treatments more manageable for patients. In the broader field of immuno-oncology, embracing such tailored strategies can significantly improve how we manage different cancers, ensuring treatments are as unique as the patients themselves.
  2. Continuous monitoring: With CAR-T therapy, real-time monitoring is key. It allows doctors to adjust treatment plans based on how the patient is responding, ensuring that care remains relevant and effective. This ability to adapt quickly is crucial in delivering the best possible outcomes. In immuno-oncology, using real-time data to guide treatment decisions ensures that patients receive the most responsive and personalized care available.
  3. Research and development: The success of CAR-T has sparked a wave of research into combination therapies. Integrating immuno-oncology with other treatment modalities can potentially enhance outcomes for patients with various cancer types.

To support the evolving CAGT landscape, IQVIA Biotech combines regulatory expertise with operational delivery through a dedicated study management team. This integrated approach enhances the trial lifecycle and accelerates therapy availability, emphasizing the importance of collaboration across various sectors. Our deep industry knowledge and innovative solutions position them as a vital partner in navigating the complexities of CAGT development.

Conclusion

It's clear that cell and gene therapies are not just changing the rules—they're transforming how we approach treating genetic disorders. This rapid evolution highlights the importance of having smart regulatory and operational frameworks in place. As these therapies grow more complex, we must be agile, ensuring they reach patients both safely and quickly.

For stakeholders, the key is to tap into these insights to help drive the development of CAGT, leading to breakthroughs in patient care. At IQVIA Biotech, we're committed to being at the heart of this exciting journey. Our role involves delivering integrated scientific and clinical solutions, refining our offerings to meet the unique challenges of this field, and, importantly, accelerating the path to delivering these advanced therapies to those who need them most.

Want to learn more? Download our Insight Brief, “Designing and Delivering Cell and Gene Therapy Clinical Trials: A strategic approach to CAGT study conduct and operations".