CMC Readiness Challenge in case of Expedited Programs for Biotechnological Products

To ensure that therapies for serious conditions are approved and available to patients as soon as it can be concluded that the therapies’ benefits justify their risks, regulatory agencies have developed specific processes to speed the availability of new therapies, especially when there are no satisfactory alternative therapies, while preserving appropriate standards for safety and effectiveness.

As expedited development does not lessen the quality expectations, one challenge is to provide adequate Chemistry, Manufacturing and Controls (CMC) information required to support a marketing authorization of a biological product on a timely manner. An accelerated clinical development program is usually a prerequisite for acceptance onto an accelerated access scheme project. This decreases the amount of time available for the development and understanding of the drug substance, the drug product and their associated processes and analytical methods. Therefore, there must be a strategy to ensure that the critical aspects of CMC provide assurance that safety and quality are not compromised. This strategy must also assure the flexibility needed to deliver consistent and reliable supplies of product to patients in a less predictable environment.

Early access approaches create challenges to complete quality and manufacturing development and data requirements during development of products. They require the manufacturing organization to reconsider traditional approaches to product and process development and undertake their own resource-intensive, cross-functional team approach to ensure a sustained supply of safe and efficacious product at the time of approval.

The Master Class will delineate potential adaptations to the traditional approach to product development, product characterization, specification setting, process validation, stability setting, manufacturing and timepoint for full completion of certain quality data packages.