Although Prior Knowledge is not formally defined, it is an established term reported in several ICH and various EMA and FDA guidelines. The term Prior Knowledge refers to an established tool used for informing decisions during pharmaceutical development and lifecycle management. In such settings, Prior Knowledge can account for internal knowledge from a company’s proprietary development and manufacturing experience (e.g., historical experience based on similar compounds, products and processes, including data modelling, application of ‘platform technologies’, knowledge from previous filings), external knowledge such as reference to scientific and technical publications, the application of established scientific principles. Knowledge also exists within regulatory agencies, and experience derived from such knowledge may inform regulators’ assessments.

Leveraging prior knowledge offers an opportunity to support risk-based development strategies. Structured use of Prior Knowledge can inform key early development decisions for ATMPs. As an example, making use of Prior Knowledge in regulatory applications, to support manufacturing and control strategies, may be justifiable in certain circumstances and may compensate for a deferral of certain data on a risk-proportionate basis. While Prior Knowledge can be applied to all stages of control strategy development, its application may be of particular relevance to significantly accelerate the start of the (first) clinical trials particularly in case of medicinal products developed in the context of unmet medical need, rare disease and accelerated pathway settings, which represent frequent development scenarios for ATMPs.

In this session, we will hear from relevant Stakeholders about their experience in the application/assessment of Prior Knowledge in the ATMPs space.

Session Speakers:

Considerations for Leveraging Prior Knowledge for Cell and Gene Therapy Products

Stella Lee, CBER, FDA

Designing a Modular CMC Platform for Lentiviral Gene Therapies

Diego Castellaro, Fondazione Telethon ETS

Modeling Strategies to Accelerate and De-Risk Viral Vector Development

Sandra Aedo, Johnson and Johnson Innovative Medicines

Additional Panelist:

Lauren Kokai, CBER, FDA

Diego Castellaro

Appearances