CMC Strategy Forum Europe 2021

The EU Medical Device Regulation (MDR), which came into full effect in May 2021, has brought in new requirements for drug delivery devices often used for biologic products. These range from pre-filled syringes, auto-injectors and on-body pumps to novel concepts, such as sensors and matrix combinations. An updated EMA Q&A guidance on implementation of the MDR was released in June and the final version of an EMA guideline on quality documentation for MAAs in July.

At the same time, the EU In Vitro Diagnostic Regulation (IVDR) is planned to come into effect in May 2022, bringing extensive changes for review of all IVDs and introducing a dedicated procedure for companion diagnostics at MAA. At the interface of medicines and IVD development clarification is required to facilitate precision medicine.
The EMA Regulatory Science Strategy to 2025, European Medicines Regulatory Network Strategy to 2025 and ongoing European Commission Pharmaceutical Strategy all recognize the increasing importance of the convergence of medicines and medical devices for optimizing treatments of the future. New relationships need to be developed with medical device competent authorities and the third party ‘notified bodies’ that form part of the EU regulatory system for medical devices/IVDs.

This session will look at the CMC impact of the rapidly evolving requirements, from the perspective of pharmaceutical industry, national and EMA regulatory agencies, and notified bodies.

Comparability is a well-established scientific concept that is pivotal to biological product realisation. It successfully enables product development and lifecycle management of existing products and the development of biosimilars.

In the more recent past, the setting of comparability/similarity criteria and the role statistics therein received more and more attention. After an initial draft in 2017, EMA published the final version of the reflection paper on that topic in July 2021. It is accessible online via - https://www.ema.europa.eu/documents/scientific-guideline/reflection-paper-statistical-methodology-comparative-assessment-quality-attributes-drug-development_en.pdf.

In this session, we will focus on the comparative assessment of quality attributes, discuss the reflection paper and how it will impact future comparability exercises for manufacturing changes and biosimilars. We will discuss what is necessary to achieve robust quality packages to demonstrate comparability and how statistical evaluations can improve decision making by providing a better understanding of similarity criteria. More specifically, we want to improve our understanding of:

• How to set pre-defined, clear and rational criteria for comparability.
• How a better understanding of the statistical performance (operating characteristics) supports the letting of meaningful criteria and how it may impact comparability packages.
• The relevance of product & process understanding and the control strategy as context for statistical evaluations.
• To which extent new concepts can contribute to efficient and scientifically meaningful approaches for comparability in a global environment.

ICH Q9(R1) – Quality Risk Management (QRM) is currently being revised, and step 2 is expected to be reached by end of 2021. The update of this guideline is anticipated to benefit ATMP by fostering increased use of science based QRM activities.
Advanced therapy medicinal products (ATMPs) are medicines that include a wide range of different products. Due to their complexity, the approaches used in the development, manufacture and control of well characterised biological products may not be fully applicable. A pro-active risk-based approach is essential to drive the product development and determine the extent of quality, non-clinical and clinical data required.
This session will provide an update on ICH Q9 development and discuss the importance of Risk based approaches and QRM in the development of ATMP.