A2 - Biological drugs and gene therapy – Hot topics on regulation and policies affecting use


Organised by the FIP Social and Administrative Pharmacy Section in collaboration with the FIP Hospital Pharmacy Section, the FIP Industrial Pharmacy Section, the FIP SIG on Biotechnology and the FIP SIG on Regulatory Sciences


Cody Midlam (Willis Towers Watson, USA) and Jennifer Dressman (Institute of Pharmaceutical Technology Biocenter, Johann Wolfgang Goethe University, Germany)


This session focuses on new technologies and approaches in pharmacotherapy, including emerging ethical dimensions associated with their implementation. Advancements in drug discovery, formulation, compounding, delivery systems and mathematical modelling can be used to translate such innovations into better outcomes in populations and individual patients. The phrase “from bench to bedside” has been used to describe the process of taking advances in basic research and transferring them into clinical practice. Also referred to as translational medicine, this approach combines diverse disciplines, expertise and techniques, and has resulted in the development of therapeutic options for disease that were previously poorly managed and resigned to the use of existing therapies. This session discusses processes taking innovative biological drugs and gene therapy from bench to bedside.

Small patient populations and sophisticated post-marketing surveillance systems may result in faster approval of biological drugs and gene therapy, where safety and efficacy are determined more at the bedside and less at the bench. Even with fast approval, the costs of treating patients with biological drugs and gene therapy are typically extremely high. Furthermore, these novel pharmaceuticals may not be appropriate for future generic competition based on their complexity alone. The combination of these factors has the potential to change the pharmaceutical landscape, including how drugs are accessed, paid for and managed.
Considering this changing pharmaceutical landscape, this session will present hot topics on regulation and policies affecting the use of biological drugs and gene therapy, and explore the contribution that pharmacists make in this evolving area. It will review new approvals and the global pipeline to examine the current approval process for biological drugs and gene therapy and discuss future expectations in a rapidly advancing field.


  1. Introduction by the chairs
  2. Policies and programmes affecting the utilisation of increasingly complex drugs; examples from the US and the world
    Susan Cantrell (AMCP, USA)
  3. Hot topics on regulation and policies affecting the utilisation of biological drugs and gene therapy
    1. Risks and benefits of fast-track approval
      Prateek Jain (Decision Resources Group, India)
    2. 10:40 – 11:00 Biosimilars and Biobetters: Interchangeability issues for pharmacists, physicians and regulators
      Iqbal Ramzan (The University of Sydney, Australia)
    3. Challenges for biologics development from a pharmaceutical point of view
      Hanns-Christian Mahler (Lonza AG, Switzerland)
  4. Panel discussion on future perspectives with a focus on:
    1. Pricing of recent therapies around the world
    2. Feasibility of generic novel therapies (exon-skipping, CAR-T)

    Facilitated by Jennifer Dressman

Learning Objectives

At the end of this session, participants will be able to:

  1. Define the landscape of novel therapies, including biologics and gene therapies;
    2. Outline the regulatory framework and policies affecting the utilisation of biological drugs and gene therapy;
    3. Identify challenges associated with biological drugs and gene therapy concerning approval and managing costs;
    4. Chart the evolving complexity of developing generic or biosimilar alternatives to newer therapies.

Type of session: Knowledge-based