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CMC Forum Europe 2017: Scientific Program
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To view the final scientific program, click here.



Program Agenda

MONDAY, 22 MAY 2017

08:30 – 12:30
EBE Satellite Session

Concept Paper 2016 Update
   1. A Risk-based Approach to Setting Sterile Filtration Bioburden Limits
   2. A Biopharmaceutical Industry Perspective on the Control of Visible Particles in Biotechnology-derived Injectable Drug Products
   3. Drug-device Combination Products or Antibody-drug Conjugates

ATMP – Specific Challenges in Development and Commercialization

EBE (European Biopharmaceutical Enterprises) of EFPIA has recently again kicked-off several position papers and is working on others. An update will be given on the position papers that have recently been published “A Risk-Based Approach to Setting Sterile Filtration Bioburden Limits” and made some considerable progress such as “Drug Device Combination Products / “A Biopharmaceutical Industry Perspective on the Control of Visible Particles in Biotechnology-Derived Injectable Drug Products”. An update will also be given on an ongoing work stream on Drug Device or Antibody Drug Conjugates.

A subsequent session comprising four presentations and followed by a panel discussion will be dedicated to current topics in Advanced Therapy Medicinal Products. In the past few years, we do see a substantial increase of investment worldwide into Regenerative Medicine including Gene and Cell Therapies. New developments comprise, for example, the retroviral transduction of B-Lymphocytes, the expansion, stimulation or transduction of T cells ex-vivo, the production of individualized tumor vaccines after an analysis of neoepitopes in tumors or Gene-Editing using CRISPR-Cas9 technology. The number of Scientific Advice Procedures of EMA-CAT has increased essentially in the past two years. With the possibility of conditional MAAs, PRIME and Adaptive Pathways EMA has created tools to move these innovative therapies with high potential to address unmet medical needs fast to market. Finally, following Article 5 of Regulation (EC) No 1394/2007, the initiative of the European Commission strives to define their own GMP requirements for ATMPs and ATiMPs.

14:00 – 17:30
Regulatory Updates from Around the World
Session Chairs: Kowid Ho, F. Hoffmann-La Roche Ltd. and Alistair Kippen, IPSEN Biopharm Limited

This session is to provide a high level regulatory update from around the world. The session starts with three or four short presentations that will provide us with an overview of changes and/or updates in their respective areas and after the presentation we will have a panel discussion. The panel discussion will include more Health Authorities from around the world and during this time questions from the audience or questions listed below will be discussed.


TUESDAY, 23 MAY 2017

09:00 – 12:30
The Continuing Evolution of Product Characterization 
Session Chairs: Brendan Hughes, Bristol-Myers Squibb Company and Mark Schenerman, MedImmune, A member of the AstraZeneca Group

Product characterization is a key element of product development throughout the lifecycle. This session will address some of the challenges that are encountered with characterization throughout development and includes case studies of how they were resolved.  Some of the challenges include characterization of complex biological formulations (e.g., co-formulation of two monoclonals), migrating from pattern similarity to attribute measurement (gels to attribute measurement and criticality assignment), and value and limitations of cell-based potency assays for blocking antibodies (an opportunity for QRM?). This session will include case studies and practical experience from industry sponsors as well as regulatory perspectives in presentations and in the accompanying panel discussions.

14:00 – 17:30
Manufacturing Process Development and Control Strategies
Session Chairs: Ralf Gleixner, Merck and Ronald Imhoff, Janssen Biologics BV

The goal of manufacturing process development is to establish a commercial manufacturing process capable of consistently producing drug substance of the intended quality. The intended quality of the drug substance is generally determined through consideration of its use in the drug product as well as from knowledge and understanding of its physical, chemical, biological, and microbiological properties or characteristics, which can influence the development of the drug product.

Guideline ICH Q11 and Q8 have introduced new approaches in manufacturing process development and determination of the Control Strategy, in particular the concept of a design space, however, the Quality Target Product Profile (QTPP), Critical Quality Attributes (CQAs) and linkage to Critical Process Parameters (CPP) and ultimately the determination of an overall Control Strategy (CS) are generally considered minimal deliverables from manufacturing process development.

The current session provides ICH Q 8/11-related implementation examples of manufacturing process development and lessons learned from products recently approved.



09:00 – 12:30
Statistical Tools in CMC
Session Chairs: Niklas Ekman, Finnish Medicines Agency and Martin Schiestl, Sandoz Biopharmaceuticals

Proper use of statistical tools could facilitate data evaluation and decision making, especially in complex situations where intuition fails. The use of statistics beyond simple descriptive data plots is becoming more common in many areas within CMC. This workshop will focus on the use of statistical tools in setting meaningful limits for manufacturing controls, and for comparing quality attributes in the evaluation of manufacturing changes and biosimilar candidates. This is a contentious field where we aim to increase a common understanding between industry and regulators on the proper use of statistics.

Proper use of statistics requires understanding of the scientific problem to be solved. Therefore, we will not go into details in statistical methodology but focus on the questions to be solved and on the interface between process experts and statisticians.

Case studies from industry and regulators will set the floor for the workshop discussion.

14:00 – 17:30
Opportunities and Obstacles for Accelerated CMC Development
Session Chairs: Chana Fuchs, CDER, FDA and Jason Hampson, Amgen Inc.

CMC development activities for biological products have long lead times and require significant financial investment. Hence they are typically “critical path” items which determine how quickly new (investigational) medicines can reach patients, both in terms of initiation of clinical trials, and commercial availability. Given that an estimated ~80%1 of investigational medicines entering Phase 1 studies will not be advanced into Phase 3, there is a strong incentive to “fail quickly and fail early”, before financial investments escalate, and to free up capacity in the development pipeline to bring other molecules forward into the clinic. Later in development, it is typically necessary for CMC Development activities for commercial supply to be initiated at risk, often 12 months or more prior to pivotal clinical data read-out.  Given a Phase 3 failure rate of ~40%1, this necessary approach can result in a significant waste of resources.

In this session, presenters from industry and academia will be invited to identify challenges / obstacles to accelerated development, and present case studies and proposals for how CMC development lead-times can be reduced and/or taken off the critical path, in a phase-appropriate manner, while continuing to ensure that medicinal products are manufactured to appropriate quality standards. Regulators will be invited to provide their perspectives on the degree of flexibility that may be available at different phases of development, within the boundaries of current regulatory requirements.


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