Pre-Conference Short Course*:
Monday, March 11 • 9:00 am – 12:00 pm
SC1: Impurities: Measurement, Characterization and Impact
Instructors:
Nanda Subbarao, Ph.D., Senior Consultant, Analytical CMC, Biologics Consulting Group - Biography
Matthew Seefeldt, Ph.D., CSO, Barofold, Inc. - Biography
Jun Liu, Ph.D., Senior Scientist and Senior Group Leader, Late Stage Pharmaceutical Development, Genentech, Inc. - Biography
This short course will examine the nature of process and product related impurities that can be present in biotechnology products, and their clinical impact. It will provide an overview of current regulatory expectations and advice on 'best practices' for impurity assessment during different stages of product development. Three leading types of impurities will be examined in greater detail. The short course will begin with an overview of the Impurities that occur in Biotechnology and Biosimilar products. The differences in the approach to specification setting for these two classes of product s will be discussed. Host Cell Protein testing presents unique problems both for Biosimilars and NCEs which will be discussed. Regulatory expectations for testing Particulates have been changing over the past few years. Particulate testing sections in the CMC often do not have all the studies expected by the CMC Reviewer. The current expectations for analytical methods and common mistakes during testing by sponsors in this area will be addressed. Extractables leachables testing for Biologics is an area that presents challenges for Sponsors. The Extractables/Leachables sections of CMC continue to be deficient and feature often in Deficiency letters. Each of these key areas of Impurity testing will be addressed in detail during this course with case studies selected from literature.
9:00 Overview of the New Impurity Hot Topics: Topics that the FDA is currently worried about; Overview of E/L and current concerns; examples from meetings and the literature.
Nanda Subbarao, Ph.D., Senior Consultant, Analytical CMC, Biologics Consulting Group
9.45 Overview of Aggregates: Causes of aggregation; characterization; current concerns in the context of immunogenicity; examples from meetings and the literature.
Matthew Seefeldt, Ph.D., CSO, Barofold, Inc.
10.30 Coffee Break
10.45 Recent experiences and Case studies of critial process and prodcut related impurity
Jun Liu, Ph.D., Senior Scientist and Senior Group Leader, Late Stage Pharmaceutical Development, Genentech, Inc.
11.30 Discussion with Q & A
12:00 End of Short Course
SC2: Bioassays for Innovators and Biosimilars: For Release Testing, Stability and Comparability
Instructors:
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc. - Biography
Xu-Rong Jiang, Ph.D., M.D., Associate Director, Analytical Biochemistry, MedImmune, Inc.
This interactive short course will enable attendees to develop methods and strategies for developing and validating bioassays that support the identification and development of their biotherapeutics products. It will include coverage of assays to test both activity and potency, including cell-based and biochemical based systems.
9:00 Bioassay in Product Life-Cycle Management
Xu-Rong Jiang, Ph.D., M.D., Associate Director, Analytical Biochemistry, MedImmune, Inc.
9:20 Antibody Fc Effector Function Assessment and Control Strategy: an Industrial Perspective
Xu-Rong Jiang, Ph.D., M.D., Associate Director, Analytical Biochemistry, MedImmune, Inc.
9:40 Rapid Bioassays for Monitoring Fab & Fc Functions
Xu-Rong Jiang, Ph.D., M.D., Associate Director, Analytical Biochemistry, MedImmune, Inc.
10:00 Quality by Design for Analytical Methods
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc.
10:30 Break for Tea/Coffee
10:45 Managing Variability in Bioassay
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc.
11:15 Continuous Verification and Bioassay Control Strategy
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc.
11:45 Open Discussion with Q&A
12:00 End of Short Course
Wednesday, March 13 • 5:30 – 8:30 pm (Dinner will be provided)
Dinner SC3: Implementation of Quality by Design
Instructors:
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc. - Biography
Michele Dougherty, Ph.D., Product Quality Reviewer, Monoclonal Antibodies, FDA - Biography
This short course will examine the benefits of QBD in terms of establishing a risk-based life-cycle management approach, and outline approaches for identifying critical quality attributes and acceptable ranges and identification of critical process parameters. It will clarify the challenges of QBD implementation, describing the tools required, and include examples and case studies regarding determination of CQA and design space, applications to existing cell culture and manufacturing processes, control of the process, control of the presence of impurities and application for comparability purposes. In addition, the regulatory benefits will be outlined together with advice on putting together a QBD application and working together with the regulatory authorities.
5:30 Quality by Design and Risk-Based Decision Making
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc.
6:00 Tools for Managing Risk throughout the Product and Analytical Lifecycle
Timothy Schofield, M.A., Senior Fellow, Regulatory Sciences, MedImmune, Inc.
6:30 Break for Dinner
6:50 FDA Experience with Implementation of Quality by Design for Biotechnology Products
Michele Dougherty, Ph.D., Product Quality Reviewer, Monoclonal Antibodies, FDA
Part 1 - Identifying Critical Quality Attributes - Regulatory Perspectives
Part 2 - Approaches to Designing a Risk-Based Control Strategy
8:00 Open Discussion with Q&A
8:30 End of Short Course
Dinner SC4: Strategy for Entering the Biosimilars and Biobetters Market
A short course led by experts in the field examining the current opportunities and penetration strategies for biosimilar products. It offers advice on collecting a robust characterization data package for the authorities, clarifies current regulatory pathways around the globe, and provides strategies for dealing with the remaining uncertainties. It also examines the complex legal issues and advises on how to work out freedom to operate and how to clear the path for development. This short course will arm investigators with the information required for entering this exciting and potentially highly rewarding field.
5:30 Strategies for Entering the Biosimilars Market
Christopher J. Holloway, Ph.D., Dr.rer.hum.biol.habil., Group Director, Regulatory Affairs & Chief Scientific Officer, ERA Consulting Group - Biography
Since the implementation of a legal basis and guidelines for biosimilar products in Europe, there have been notable successes, but also some failures, from which useful lessons can be learned. Currently, a "new wave" of biosimilars, based on "blockbuster" monoclonal antibodies, is underway. Compared with the "first wave" of biosimilars, these products present particular challenges, to be discussed, along with consideration of what a sponsor for successful development of a biosimilar product. Globally, there are further challenges, owing to differing requirements, which will be addressed. Finally, there is the challenge of positioning a biosimilar product in the market.
- The track record of biosimilars in Europe: learning from successes and failures
- The "next wave" of biosimilars, their challenges, and current players
- What does a sponsor need for successful development of a biosimilar product?
- Can there be a global approach?
- Bringing biosimilars to market; experiences to date in Europe
- So, are biosimilars worth the effort?
7:30 Legal Issues to Consider for Biosimilars
Donald L. Zuhn, Jr., Ph.D., Partner, McDonnell Boehnen Hulbert &Berghoff LLP - Biography
This part of the short course will review the patent-related aspects of the biosimilar regulatory pathway and the FDA's corresponding guidance on biosimilars, discuss recent attempts to limit the scope of the regulatory pathway, describe how to work in the patent environment and analyze freedom to operate, and provide an update on relevant legal precedent.
6:30 Break for Dinner with Discussion
7:00 Analytical Tools for Characterizing Biosimilars, Biobetters and Next Generation Antibodies with Enhanced Properties
Alain Beck, PhD, Senior Director, Antibody Physico-Chemistry, Centre d'Immunologie Pierre Fabre and Associate Editor, mAbs - Biography
This part of the short course will introduce the strategies of therapeutic antibodies in-depth characterization by mass spectrometry (MS) and discuss analytical comparison of biosimilar candidate to originator mAbs, with the cases of trastuzumab, rituximab, cetuximab and etanercept. In addition, the structural and functional insights gained both by state-of-the art and emerging MS methods used for biobetters and next generation antibodies design and optimization will also be highlighted. These methods include native MS, Ion Mobility MS, ETD, MALDI-TOF N and Ct sequencing, H/D X MS and absolute MS quantitation.
7:30 Biologics Price Competition and Innovation Act of 2009: FDA's Overview of the Regulatory Guidance for the Development and Approval of Biosimilar Products in the US
Marjorie A. Shapiro, Ph.D., Chief, Laboratory of Molecular and Developmental Immunology, Division of Monoclonal Antibodies, CDER/FDA
The Biologics Price Competition and Innovation Act of 2009 promotes the development of biosimilar products for the US market. In February 2012, the FDA released three draft guidances focused on the development of biosimilar products for therapeutic proteins. This presentation will provide an overview of the legislation and guidance documents as well as the FDA's current thinking on biosimilar product development.
8:00 Discussion with Q&A
8:30 End of Short Course
Thursday, March 14 • 1:00 – 4:30 PM
SC5: Comparability Protocols for Innovator Products and Biosimilars
Instructors:
Christopher J. Holloway, Ph.D., Dr.rer.hum.biol.habil., Group Director, Regulatory Affairs & CSO, ERA Consulting Group - Biography
Sheila Magil, Ph.D., Senior Consultant, Bioprocess Technology Consultants, USA - Biography
Nanda Subbarao, Ph.D., Senior Consultant, Analytical CMC, Biologics Consulting Group - Biography
Manufacturing changes can impact on quality attributes of biologics, and may affect efficacy and/or safety of the product. For that reason, a thorough comparability exercise is required, to assess the impact of the change and whether CMC data alone will suffice to support the change. In the case of biosimilar products, an extensive comparability exercise is required to substantiate the claim of biosimilarity. This interactive short course will consider comparability exercises during development, as well as post-approval, addressing regulatory and technical requirements. This should provide the attendee with the knowledge on how to prepare a comparability package for discussion with regulatory agencies, towards acceptance of the proposed change to the process/product.
1:00 Comparability – the Regulator’s View
Sheila Magil, Ph.D., Senior Consultant, Bioprocess Technology Consultants, USA
1:45 Methods for Biotech/Biosimilar Product Characterization and Comparability
Nanda Subbarao, Ph.D., Senior Consultant, Analytical CMC, Biologics Consulting Group
3:00 Break for Tea/Coffee
3:15 Interactive Exercises
3:45 Comparability, Change Management Protocols and the EU System of Variations to Marketing Authorisations
Christopher J. Holloway, Ph.D., Group Director, Regulatory Affairs & CSO, ERA Consulting
4:30 End of Short Course
* Separate registration required