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FDA Releases New Draft Guidance for Cell and Gene Therapy Products

Updated: Feb 16



The Food and Drug Administration (FDA) has recently issued a draft guidance document addressing the management of manufacturing changes and comparability for human cellular and gene therapy (CGT) products. This guidance aims to provide recommendations and strategies for ensuring the safety and effectiveness of these complex and evolving therapies.  The manufacturing of CGT products poses unique challenges due to their inherent complexity. These therapies often involve the use of living cells, making it difficult to control and standardize the manufacturing process. Any changes made during manufacturing can potentially impact the quality and performance of the final product. Therefore, it is crucial to carefully manage and evaluate manufacturing changes to ensure continued product safety and efficacy.

The Food and Drug Administration (FDA) has recently issued a draft guidance document addressing the management of manufacturing changes and comparability for human cellular and gene therapy (CGT) products. This guidance aims to provide recommendations and strategies for ensuring the safety and effectiveness of these complex and evolving therapies.


The manufacturing of CGT products poses unique challenges due to their inherent complexity. These therapies often involve the use of living cells, making it difficult to control and standardize the manufacturing process. Any changes made during manufacturing can potentially impact the quality and performance of the final product. Therefore, it is crucial to carefully manage and evaluate manufacturing changes to ensure continued product safety and efficacy.


Key Takeaways from the FDA's Draft Guidance:

  1. Assessing the Risk of Manufacturing Changes: The draft guidance emphasizes the need to assess the risk associated with each manufacturing change. Factors such as the nature of the change, its potential impact on product quality, and the availability of data to support the change should be considered.

  2. Conducting Comparability Studies: Comparability studies are recommended to evaluate the effect of manufacturing changes on product quality. These studies involve comparing the pre-change and post-change products in terms of safety, purity, potency, and other quality attributes.

  3. Reporting Manufacturing Changes to the FDA: Sponsors of Investigational New Drug Applications (INDs) and applicants of Biologics License Applications (BLAs) for CGT products are required to report all manufacturing changes to the FDA. This ensures transparency and regulatory oversight throughout the product development process.


Guidance on Conducting Comparability Studies:

  • Lifecycle Approach to Comparability:

The draft guidance highlights the importance of adopting a lifecycle approach to comparability. This approach considers the entire product lifecycle, from manufacturing to clinical use, to assess the impact of changes on product quality.

  • Risk-Based Approach to Comparability:

A risk-based approach should be employed to evaluate the critical quality attributes of the product and the potential impact of manufacturing changes on these attributes. This helps identify areas that require closer evaluation and monitoring.

  • Variety of Methods for Assessing Comparability:

Various methods, including analytical, animal, and clinical studies, can be utilized to assess comparability. These methods enable a comprehensive evaluation of the impact of manufacturing changes on product quality.


Other Noteworthy Points from the Draft Guidance:


1. Comprehensive Quality Management System:

  • The FDA recommends that sponsors establish a robust quality management system (QMS) to ensure the quality and consistency of CGT products throughout the manufacturing process.


2. Risk-Based Approaches for Identifying and Assessing Risks:

  • Sponsors are encouraged to use risk-based approaches to identify and assess the risks associated with manufacturing changes. This helps prioritize resources and focus on critical areas.


3. Communication and Collaboration:

  • The guidance underscores the significance of ongoing communication and collaboration between sponsors and the FDA. Regular dialogue throughout the product lifecycle facilitates the exchange of critical information and ensures regulatory compliance.


The FDA's draft guidance for manufacturing changes and comparability in CGT products serves as a valuable resource for sponsors of INDs and BLAs. It offers clear recommendations and strategies to manage the complexities involved in manufacturing these advanced therapies. By emphasizing the importance of quality management and risk assessment, the guidance supports the development and production of safe and effective CGT products throughout their lifecycle.


Contact BioBoston Consulting or visit our website to learn more about how we can support your organization.


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