PDA Webinar: ATMP Interest Group Meeting

Module 1: Statistical Solutions for the Data Challenges of ATMP
Speaker: Katherine Giacoletti - Synolostats
Duration: 30 minutes

A challenge with many biotherapeutics, especially cell & gene therapies or products having accelerated approval status is the limited data to develop acceptance criteria. This can lead to ranges that are too wide or too narrow, depending on the method used to compute the specifications. An adaptive approach using Bayesian statistical methods could use all available information to develop initial specifications. As more data accumulate, specifications would be updated using the prior information and new data. The typical statistical background of scientists is limited to Classical (Frequentist) methods. These methods are used in a variety of ways to define acceptance and operating criteria of drug substances and products. However, these methods are not optimal, and in some cases are completely inappropriate, in situations of small sample sizes or complex data relationships. Bayesian methods, which are not widely known in drug development, but routinely used for clinical trials and medical devices, can provide solutions in these challenging situations. Just as innovative science enables the development, manufacture and delivery of these products, innovative analysis of complex data permits confidence in quality and reduction of risk. In this session, the Bayesian approach routinely used for clinical trials and medical devices will be introduced and benefits will be illustrated by case example. Participants will gain insight into alternative methods of statistical analysis applicable to all product platforms, and particularly useful for the challenges presented by cell and gene therapy platforms and for accelerated approvals.

Module 2: Risk-Based Approach to Apheresis and Infusion Site Qualification
Speaker: David Geoghegan - Tmunity
Duration: 30 minutes

Clinical Study Start up activities in the cell therapy space require more ingenuity due to the complexity of autologous human cell collection. Companies working in this space must understand the complexity of their protocol requirements to ensure clinical sites selected for the program can adequately comply with the complexities.

Experiences with clinical study start-up activities in the cell therapy space. The goal will be to define key critical activities to better ensure the quality of the selected sites to support start-up activities as they relate to collecting and processing cellular material for infusion.

Discuss key components of qualification and compliances at a clinical site and what we should keep in mind during the qualification process. How do companies identify risk within a given protocol to focus the clinical site qualification process? How do companies determine regulatory compliance and what accreditations a clinical site should be certified? What does chain of custody look like in this environment and how do we ensure controls from vein to vein.

Ultimately, how do we test the system to ensure the site can manage the collection, cryopreservation and shipment to and from a manufacturing site.

Module 3: PDA ATMP Interest Group Update
Speaker: Darius Pillsbury
Duration: 30 minutes

General updates related to on-going and planned activities for cell & gene therapy at PDA (e.g., update on the ASN cryopreservation standard, ATMP AB call for topics for technical reports/white papers).