The Importance of Using a Multidisciplinary Approach in Early-Stage Development for Cell and Gene Therapy Products

The development of cell and gene therapy products requires a highly specialized and multidisciplinary approach to ensure success. Early-stage development plays a critical role, and failing to develop the product correctly can result in costly delays or even the abandonment of potentially life-saving therapies. At BioBoston Consulting, we understand the importance of this approach and offer our expertise to ensure that cell and gene therapy products meet regulatory standards and patient needs effectively.?

Why a Multidisciplinary Approach is Vital for Success?

Dr. Tal H. Salz, a gene therapy reviewer at the FDA, highlighted key factors contributing to the success of cell and gene therapy products. He identified that Sponsors who do not implement a multidisciplinary approach tend to experience lower success rates, primarily due to:?

• Inadequate manufacturing processes?

• Limited materials for testing?

• Tight timelines?

• Poor quality control?

Given these risks, adopting a multidisciplinary framework during the early development stages can prevent delays and facilitate smoother progress through regulatory requirements.?

Establishing Effective Controls with a Multidisciplinary Framework?

In January 2020, the FDA issued guidelines on Chemistry, Manufacturing, and Control (CMC) information for gene therapy Investigational New Drug (IND) applications. These recommendations emphasize the need for a multidisciplinary framework to establish effective controls throughout the product lifecycle. At BioBoston Consulting, we recommend focusing on the end goal, aligning with patient needs and regulatory requirements, and establishing a roadmap that leads from development to regulatory approval.?

Building a Cross-Functional Team for Development Success?

One of the key elements of a multidisciplinary approach is creating a cross-functional team that is well-versed in the key areas of development, including manufacturing, quality control, regulatory oversight, and supply chain management. This collaborative team is crucial to successfully navigate the complexities of cell and gene therapy development.?

Here are the critical roles required in this team:?

• Development Team: The development team works to define biophysical characteristics and identify Critical Quality Attributes (CQAs), Critical Material Attributes (CMAs), and Critical Process Parameters (CPPs).?

• Quality Control: Ensures that the CQAs can be effectively tested and that the product adheres to the required standards.?

• Regulatory Team: Confirms the effectiveness of the CQAs and guides the product through regulatory requirements.?

• Formulation and Process Experts: Specialists in formulation and process optimization help identify critical process parameters and material attributes to maintain product quality, potency, and purity.?

• Supply Chain Experts: Develop a robust supply chain with reliable raw materials that can consistently support the manufacturing process.?

• Quality Assurance: Ensures that proper protocols are in place to handle deviations, changes, and mitigations effectively.?

• Operations and Engineering: Review the transition from bench-scale to commercial-scale processes, including automation, controls, and facility requirements.?

• Environmental Toxicologist: Evaluates the environmental impact of raw materials and ensures the process remains environmentally safe.?

Leveraging an Agile Approach for Effective Team Management?

Managing a multidisciplinary team requires an agile approach that allows for quick adaptation to changes and modifications in the development process. By utilizing agile methodology, your team can:?

• Break down the project into manageable deliverables and tasks?

• Minimize time spent on discussions and maximize time spent on development work?

• Quickly identify and address challenges that may delay progress?

This flexibility is essential in cell and gene therapy development, where changes can arise at any stage of the process.?

Why BioBoston Consulting is Your Ideal Partner?

Are you developing a cell and gene therapy product? Whether you are at the early stages or further along in the development process, it is never too late to implement a multidisciplinary approach. At BioBoston Consulting, we provide end-to-end support for cell and gene therapy product development, ensuring that your team has the expertise and guidance needed to succeed.?

Our Cell and Gene Therapy Center of Excellence brings together top experts in all areas of product development, from regulatory compliance to quality control and supply chain management. We work with you to develop a clear, actionable roadmap that takes your product from the lab to the clinic and beyond.?

Contact BioBoston Consulting Today?

If you are looking to enhance your cell and gene therapy development process, BioBoston Consulting is here to help. Our team can support you through every stage of development, ensuring a robust, compliant, and successful path to market.?

Contact BioBoston Consulting now to learn more about how we can help optimize your cell and gene therapy product development and get you closer to your next milestone.?