SC-26 - How to design a pharmacoepidemiologic study of biologics including biosimilars and cell & gene therapies

1. Course Aim

This workshop will provide participants with information on the following topics:
• Background on biologics, biosimilars, and cell and gene therapies (CGTs)
• Regulatory, methodological, and clinical considerations when designing a pharmacoepidemiology study with respect to selecting the most suitable study design and real world data, including either primary data collection or secondary use of available data sources such as registries, claims data and electronic medical records
• How to design a real-world study to explore pattern of use, comparative effectiveness and safety of biologics including biosimilars through interactive case study discussion
• Unique elements of treatment with CGTs and their implications on long-term safety and effectiveness monitoring using RWD
• Special emphasis will be given to the methodological and operational issues srelevant to regulatory agency post-authorization requirements

2. Requisites Statement
This should state a) whether other courses or level of expertise (Entry, Intermediate, Advanced level) is expected and b) any precourse materials need to be accessed or reviewed

Entry level. Fundamental understanding of pharmacoepidemiology required, but biologic-related topics will start from the basics.

3. Course Objectives

1. To gain an understanding of biologics including biosimilars and CGTs with the related regulatory landscape
2. To learn how to design real-world studies for studying comparative benefit-risk profile of biologics in post-marketing setting in different therapeutic areas
3. To become familiar with the special challenges associated with the necessary long-term follow up of CGTs

4. Syllabus Outline

1. Biologics: Regulatory and clinical considerations
Students will be introduced to biologics/biosimilars terminology, the big picture, complexities in the marketplace that challenge researchers. An introduction to regulatory requirements for biologics including biosimilars will be discussed along with some recommended tips regarding planning pharmacoepidemiologic research studies will also be described.
2. Methodological considerations when studying biologics/biosimilars using prospective data, U.S. secondary data sources, and multiple data sources to generate evidence
This presentation will discuss stakeholder needs to generate fit-for-purpose evidence. Students will learn about different study design options and specific considerations when designing a pharmacoepidemiologic study of biologics. This presentation will also focus on the use of U.S. secondary data sources, including administrative claims and electronic health records, for conducting comparative effectiveness and safety studies of biologics / biosimilars. The presentation will include examples from published literature.

3. Requirements and potential limitations of observational studies of biosimilars in real world settings through secondary use of healthcare databases in Europe.
Overview of observational studies on originator biologics and biosimilars that have been carried out or are actually ongoing in Europe also through large-scale distributed claims database network and linkage with clinical registries will be given. In particular, potential limitations of different data sources (e.g. claims databases and electronic medical records from various EU countries in terms of addressing clinically relevant questions concerning originator biologics and biosimilars will be discussed. Specific issues to consider will include interchangeability of biological originator and biosimilar products, and immunogenicity associated with switching between biological medicines. Some examples of database studies will be provided.

4. Special considerations pertaining to post-authorization safety and effectiveness evaluations for Cell and Gene Therapies
CGTs are frequently administered only once but have direct and indirect lifelong impacts on the health and well-being of patients. This presentation will underscore the required breadth and depth of real-world data needed to assess the long-term benefit-risk profile of CGTs. Operational and tactical aspects of conducting such studies will be discussed.

5. Interactive Case Study activity
We will have two interactive case studies to apply learnings from the course lectures. Participants will be presented with a problem and task for each of the case studies. Participants will be asked to answer a series of questions using the polling system to prompt group discussion. Following the case studies, there will be time for open discussion and additional Q&A.