Product characterisation/Quality by Design
Quality by Design (QbD) is a science- and risk-based approach to pharmaceutical
development. Applied to manufacturing processes, it is intended to ensure a rational and cost-
effective approach to product quality. Both the EMA and FDA have systematically assessed
QbD elements of regulatory filings and, especially since the FDA’s 2011 Process Validation
Guidance and the ICH’s Q8 through Q11, it has become an intrinsic part of regulatory guidance.
However, understanding and adoption of the principles of QbD has remained relatively limited
in the field of cell and gene therapy. We strongly believe that its methodologies are critical to
the successful development of ATMPs and that they should be incorporated as early as possible
into the science of manufacturing. This workshop will both demystify the fundamentals of QbD
and provide concrete examples of its application to this field. You will leave with a crystal-clear
understanding of P Diagrams, RAMM matrices, the QTPP and many more tools which you can
immediately use to improve your products and processes.
Clinical trial design for ATMPs
The considerations when initiating a clinical trial for cell-based therapies are diverse and
numerous. Alternative approaches to traditional clinical development based on sequential,
seamless design are justified, including exploratory (phase I/IIa) trials examining both safety and
efficacy while using an extensive biomarker portfolio, whenever applicable. Great emphasis
should nevertheless be placed on patient safety, particularly in designing appropriate GMP-
grade product and risk-benefit related stopping criteria. In this session some of these aspects
will be discussed using real-life examples.
In this session, Professor Dr Abou-El Anein will be joined by his colleague, Mr Daniel Kaiser
who will discuss the characteristics of a clinical grade product to be used in clinical trials.
Authoring the Common Technical Document (CTD) Module 3 (CMC/Quality)
The ICH Common Technical Document was developed to standardise the way information is
shared with regulators, and is accepted or mandatory by agencies following ICH (e.g. EU, US,
Japan, Canada etc). The CTD is used for all types of medicinal product but the purpose of each
section may not always be clear for ATMP. This workshop aims to clarify:
What the purpose of each section is
Whether the section is descriptive or data-driven
What sorts of data are expected
Tips and considerations for style, use of figures and tables, etc.