Raw material selection decisions early in the development of ATMPs are crucial and can impact the final product quality and ultimately patient safety. Therefore, it is imperative that developers employ a risk-based approach when selecting and qualifying materials to establish the necessary controls to ensure process robustness and safety of the product.

Material classification is key to a successful implementation of a risk-based approach. The same material (e.g. plasmids) may be classified differently according to its use in different products and thus warrant a different control strategy. Classification of materials should be considered early in development with input from regulators as appropriate. Current guidance from many regulatory and standards bodies including FDA, EU, ICH, PIC/S, USP, and Ph. Eur., provide a foundation though some inconsistencies and use of alternate terminology do exist.

Control strategies for materials should be commensurate to their classification and, therefore, their potential to affect critical quality attributes of the product. Suppliers are an extension of an ATMP developer’s manufacturing process and the rigor of suppliers’ qualification activities and controls can impact the manufacturing process and the product. As suppliers are often unfamiliar with therapeutic manufacturing requirements (e.g. the need to minimize or eliminate the use of animal-derived materials),a partnership mindset by both parties to ensure transparency and joint mitigation of risks is crucial.

Session Speakers:

Material Considerations for The Manufacture of Pluripotent Stem Cell Derived Products

Saran Karumbayaram, CBER, FDA

Challenges and Perspectives in the Manufacture of Synthetic sgRNA used in Cell and Gene Therapy

Joe Guiles, Agilent Technologies Inc.

Material Qualification from a CDMO Perspective: A Phase Appropriate Approach to Material and Component Risk Control

Athenesia Faggins, WuXi Advanced Therapies

Vandana Chauhan

Appearances