Short Courses

Pre-Conference Short Course*
SC1: Quality By Design

Monday, April 4, 9:00 am – 12:00 pm


This short course presents an intensive study of the application and benefits of this new approach by experienced Industry experts including an FDA representative, to enable investigators to decide on a QbD-implementation strategy. It weighs the initial investment required against the ultimate efficiencies in process development and life-cycle management, and presents practical approaches for determining critical process parameters, design space and control strategy.

9:00 Chairperson’s Opening Remarks

Ron Taticek, Ph.D., Senior Director and Head, Commercial Product Quality Stewards, Biologics, Genentech, Inc.

9:05  What is Quality by Design?

Ron_TaticekRon Taticek, Ph.D., Senior Director/Head, Commercial Product Quality Stewards, Biologics,
Genentech, Inc.

The first part of this presentation will examine the terminology used and its meaning, particularly regarding design space and means of delineating it. It will examine the benefits and draw backs in terms of the initial investment and future rewards in terms of potential improvements in consistency and quality of the product, especially clinical trial material, the long term simplification of maintaining product quality, its application to all stages of development, the impact on risk analysis, and the potential smoother running with the regulatory authorities.

9:45   Critical Quality Attributes – What Do They Mean and How Are They Defined?

Ziping_WeiZiping Wei, Ph.D., Fellow, Analytical Biochemistry, MedImmune, Inc.

Identification of critical quality attributes (CQAs) is essential for the establishment of design space and control strategy for biopharmaceutical products. This presentation will describe a process for determining CQAs through the evaluation of the impact on safety and efficacy during product development. The focus will be on how prior product knowledge, laboratory, non-clinical and clinical experience may be used to determine CQAs and define their acceptable ranges. The presentation will also discuss how to link quality attributes to control strategy.

10:15   Refreshment Break

10:30   Regulatory Perspectives on Quality by Design for Biologics

Michele_DoughertyMichele K. Dougherty, Ph.D., Product Quality Reviewer, Division of Monoclonal Antibodies,

11:00  Life-Cycle Management and QbD

Ron Taticek, Ph.D., Senior Director and Head, Commercial Product Quality Stewards, Biologics, Genentech

The second part of this presentation will describe Genentech’s experiences with the practical application of Quality by Design. It will outline the parameters that need to be tested to define and establish the design space such as the pH, the temperature, the media components for cell culture and means of elution, and will outline the role of QbD in release assays and bioassays. We will describe Genentech’s experiences with pilot programs and offer advice on seeking and getting QbD approval from the FDA leading to BLA filing. Finally an industry experience on current guidelines will be presented.

11:40   Discussion

12:00   End of Short Course


Post-Conference Short Course*
SC2: Strategy for Entering the Biosimilars Market

Thursday, April 7, 1:30 pm – 4:30 pm


A short course led by experts in the field examining the current opportunities, profit potential and penetration strategies for biosimilar products. It offers advice on collecting a robust 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.

1:30    Chairperson’s Opening Remarks

Chris Holloway, Ph.D., Group Director, Regulatory Affairs, ERA Consulting Group

 1:35   Strategy for Entering the Biosimilars Market

Christian_ClausslLouis-Christian Clauss, Ph.D., Principal Consultant, RA & QA Healthcare, Atheln Biomed

A review of the global biosimilars market including  the regulated (the EU), the to be regulated (Japan, UA, NZ, Australia, Canada etc.) and the unregulated. It will examine:

  • Biosimilars in the pipeline and already licensed
  • Current opportunities that make this option worthwhile
  • What is required to bring a biosimilar to market
  • The challenges
  • Studies required to demonstrate pharmaceutical equivalence and “similarity”
  • Potential costs and profits
  • The choice between comparability development or stand alone

In addition, the following will be discussed:

  • BioSimilars Lesson learnt to date
  • How the USA and EU and Global market compare
  • The challenges and opportunities for market entry
  • The hurdles for BioSimilars manufacturers
  • The data requirements for a BioSimilar in the global market
  • Commercial consideration  from recently marketed biosimilars
  • More complex proteins e.g. blood products and monoclonals

2:05   Biosimilars in Europe: 5 years of Successes and Failures

 Chris_HollowayChris Holloway, Ph.D., Group Director, Regulatory Affairs, ERA Consulting Group

The presentation will begin by explaining this legal basis and its ramifications for products claimed to be "biosimilar". The concept of "similar biological medicinal products", or "biosimilars" as they have become known, was first introduced in the EU in 2003 but the legal basis was not established until 2004. The directives were followed by a series of guidelines issued by the European Medicines Agency in 2005, which address quality, non-clinical and clinical requirements, and provide some product-specific guidelines. More recently, a draft guideline has been issued for non-clinical and clinical development of biosimilar monoclonal antibodies and a concept paper for biosimilar interferon-beta has also been published by the European Medicines Agency. These guidelines will be discussed in the light of experiences to date, both successes and failures.

The fact that several biosimilars have been approved, demonstrates the "achievability" of a marketing authorisation through the biosimilar route. The "failures" are interesting and important to illustrate the limitations of the biosimilar paradigm and the issues that need to be considered by sponsors during development, particularly with regard to comparability in the context of "equivalent safety". The presentation will conclude with an overview of the ongoing challenges facing the "biosimilar industry".

2:35   Managing the Life-Cycle of Biologics in a World with Biosimilars

Gillian R. Woollett, M.A., D.Phil., Chief Scientist, Engel & Novitt, LLP

As biosimilars have enter the global market place in other highly regulated markets, we all know that the US will not be far behind. Whether sponsors choose to use the new biosimilar pathways in BPCIA or not, the biologics market is changing and how originator biopharmaceuticals are researched and developed must take this into account. We will discuss what aspects of the development of any biologic will give opportunities to manage and predict the likely competitive environment that it will face, and how to consider these from the very beginning of development to avoid surprises later.

3:05  Refreshment Break

3:20  Exploring the Interplay Between the Biosimilar Pathway and Patent Law for Biologics

 Kathleen_WilliamsKathleen Williams, Partner, Intellectual Property, Edwards Angell Palmer & Dodge LLP

This presentation focuses on the issues that face companies developing therapeutics which are biologics. Whether new, generic or follow-on biologic therapeutics, the commercial markets are significant, and the regulatory pathways still uncertain. How do intellectual property issues such as patent protection as well as third party patent infringement arise and get resolved for biologics? How do concerns as to equivalency--bioactivity, glycosylation, half-life, immunogenicity play a role in IP? What have we learned from history? EPO (Amgen/TKT; Amgen/Roche), Avonex, Pergonal? How does one create a clear pathway as to one's patent strategy and make statements to the FDA in seeking product approval that is clear and consistent? 

4:00   Discussion with Short Course Speakers including:

Brian Harvey, M.D., Ph.D., Vice President, US Regulatory Policy, Global Regulatory Affairs, Sanofi-Aventis


Keith Chidwick Keith Chidwick, Ph.D., Senior Pharmaceutical Assessor, Biologicals Unit, MHRA


4:30 End of Short Course

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