Industries: Pharma / Biotech

Biosimilars – Understanding Regulatory Challenges™

Course Director: John Geigert, Ph.D.

Course Description

In the United States, much excitement has been generated since the passage of the Biologics Price Competition and Innovation Act (BPCI) of 2009. This law establishes an abbreviated market approval pathway for biological products that are demonstrated to be ‘highly similar' (biosimilar) to, or ‘interchangeable' with, a FDA-licensed marketed biologic. However, much confusion has arisen over exactly what this new abbreviated regulatory pathway actually means, and what it will look like when fully implemented by the FDA.

To better understand what the developing FDA biosimilar pathway might look like, this course will closely examine the existing regulatory guidelines from the European Medicine Agency (EMA) and the World Health Organization (WHO), and compare them to the draft FDA guidances. Insights and practical suggestions to help your company enter the biosimilar biological product market will be provided by examining what has worked, and what has not, for the existing market-approved EMA biosimilars and FDA follow-on-proteins.

Who Should Attend

This biosimilars training course is designed for senior management, directors, managers, supervisors, project planners and professional staff seeking to develop or implement a regulatory strategy for entering into biosimilars, including Project Managers, Regulatory Affairs, Manufacturing and Quality personnel.

Additionally, this course will be of benefit to those needing to understand Food & Drug Administration (FDA), European Medicines Agency (EMA) and World Health Organization (WHO) perspectives on biosimilars, and the differences in how these agencies view these products.

Course Agenda

First Day

Biosimilars Are Not "Bio-Generics"

  • Defining the terminology
  • Highly similar, not equivalent
  • Highly similar vs interchangeable
  • Market application dossier content comparison

Regulatory Pathways For Biosimilars

  • U.S. Public Health Service Act pathway to biosimilars
  • U.S. confusion over protein enzymes and hormones
  • Biosimilar pathway in Europe
  • Patent protection and marketingexclusivity

Biosimilar Meetings With Regulatory Agencies

  • FDA (PDUFA vs BsUFA) compared to EMA
  • Filing fees for review and meetings
  • Meeting opportunities for biosimilars
  • Biosimilar market approval review timelines

General Principles For Biosimilars:

  • Key purpose of biosimilarity studies
  • Biological products being considered for biosimilars
  • Reference biological product comparators
  • Scientific approach required – stepwise and totality of evidence

Second Day

Biosimilarity Step 1: CMC Comparability

  • Challenge of being blinded to innovator's CMC development
  • Fingerprint, state-of-the-art protein characterization
  • Limitations of CMC similarity
  • Justifying observed CMC differences

Biosimilarity Step 2: NONCLINICAL Comparability:

  • Linkage to CMC biosimilarity findings
  • Risk-based stepwise approach to Nonclinical comparability
  • Key elements of the Nonclinical biosimilarity study
  • Which Nonclinical studies are not needed for biosimilars

Biosimilarity Step 3: CLINICAL Comparability:

  • Linkage to both CMC and Nonclinical biosimilarity findings
  • Risk-based stepwise approach to Clinical comparability
  • Pre-defined acceptance criteria for human PK studies
  • Equivalence vs non-inferiority Clinical study design

Biosimilar Lessons Learned and the Future

  • Successful EMA marketed biosimilar products
  • Status of FDA biosimilar products in clinical development
  • EMA biosimilar successes and failures
  • Crystal-balling – price reductions, biosuperior

Learning Objectives

At the end of the 2 day course you will:

  • Gain a firm understanding of the CMC, Nonclinical and Clinical regulatory requirements and expectations (FDA, EMA and WHO) for biosimilar biological products
  • Appreciate why biosimilars are not treated by the regulatory authorities as bio-generics, and the significant differences between biosimilars and generic chemical drugs
  • Learn the totality of what is to be present in the submitted comprehensive, scientific biosimilarity exercise – what has worked, what has not, in bringing biosimilars into the marketplace.

Frequently Asked Questions

Does the course cover FDA and international concerns?

The course covers all aspects of biosimilar requirements and guidance from the FDA, European Medicine Agency (EMA), and World Health Organization (WHO).

Does the course cover CMC, Nonclinical and Clinical concerns?

The course covers expectations for Chemistry, Manufacturing & Control (CMC), Nonclincal (i.e., animal studies) and Clinical (i.e., human studies) biosimilarity studies.