15/16 December 2020
The EMA/CHMP offers assistance and support in the form of the Scientific Advice to the developing or approving company or institution for the development and approval of drugs. According to the EMA, this can concern the following questions:
In the context of ATMP, the Scientific Advice particularly frequently raises questions about comparability in the case of changes in manufacturing processes or the introduction of a new or additional production site. This may involve changes to equipment, raw materials and critical starting materials such as cells or vectors or their suppliers, scale of the manufacturing process or product stability. Such changes often occur during the early stages of product development. In such cases, an appropriate comparability program is necessary to capture and evaluate the criticality and impact of the changes.
Due to the heterogeneity and the usually high complexity and dynamics of cell- or tissue-based ATMP, the evaluation of changes is not always easy. Therefore, functional / biological properties of the product are the key to define the level of comparability achieved and to define the range of non-clinical and/or clinical data to be generated.
In order to provide additional help and information, the EMA published a Q&A document in December. It is intended to address specific issues related to demonstrating comparability at the level of quality aspects for ATMPs, because changes in manufacturing requirements that have been registered under an ATMP approval must be submitted and, where appropriate, reviewed through change procedures. This approach also applies to the approval of an ATMP for clinical trials for which substantial changes must be submitted.
This document includes about a dozen questions ranging from "What do comparability and comparability studies stand for, or "How does the ICH Q5E Directive, which addresses comparability of biological biotechnological medicinal products, apply to ATMPs?" to "Is a comparability study required when changes are made in the manufacture of starting materials?
To find out all the questions and answers, please read the EMA document : "Questions and answers: Comparability considerations for Advanced Therapy Medicinal Products (ATMP)".