Date: April 18, 2023
Time: 14:00 GMT/ 15:00 CET/ 09:00 ET

Webinar in collaboration with Europabio

Abstract

The development of therapies for rare diseases comes with distinct challenges such as patient recruitment, difficulty in setting meaningful clinical endpoints, as well manufacturing and supply challenges. For biologic products(growth factors, monoclonal antibodies, other complex proteins), development and production costs tend to be higher due to the complexity of the drug substance. To manage costs, the clinical development and manufacturing and Chemistry, Manufacturing and Controls (CMC) teams need to be aligned to ensure drug product is delivered on time to meet clinical trial needs. During this session, speakers cover aspects of clinical trial design for orphan biologics products as well as key strategies for ensuring clinical trial supply throughout clinical development.

Key Learning Objectives

1. Considerations in identifying meaningful clinical endpoints for orphan diseases
2. Orphan clinical trial design considerations and strategies to ensure sufficient patient recruitment
3. Manufacturing and clinical supply strategies to ensure supply of clinical trial materials while collecting data needed to support NDA submission.

FEATURED SPEAKERS

Wai Lam Ling, Ph.D.,
Vice President, Scientific Advisory
Catalent

Dr. Ling brings with her more than 25 years of experience in biologics development, including cell and gene therapies, and joins Catalent from Rocket Pharma, where she held the role of Head of Manufacturing, Science and Technology. Prior to Rocket, Dr. Ling worked at Merck & Co. (MSD), where she led biologics CMC development and supported regulatory filing. Prior to Merck, she was at Schering-Plough Corp., where she worked on adenoviral gene therapy development and manufacturing prior to transition to biologics. She earned her Bachelor of Science degree in Biochemistry from the University of California, Los Angeles, and her Doctorate degree in Pharmacology from the Rutgers University. Dr. Ling also completed her post-doctoral research fellowship at the Albert Einstein College of Medicine, New York. 

Thomas Bols
Head of Government Affairs and Public Policy for EMEA & Asia Pacific region,
PTC Therapeutics

Thomas joined PTC in March 2020, continuing his career in the biopharmaceutical industry, having held positions for Amgen, Merck, Biogen and, most recently, at the global association, DIA. He has worked in the interconnected areas of market access, government affairs, public affairs, health policy, corporate communications, and patient advocacy. In each role, Thomas has spearheaded initiatives that are crucial to bringing much-needed medicines to patients. Throughout his career, Thomas has advised and represented companies, as well as industry federations in engagements with policy makers, patient representatives, media, regulators, payers, and other national and European health policy platforms. Thomas held board positions at several European associations, such as Europabio and EUCOPE, chaired a variety of working groups related to regulatory and access issues within EFPIA, and has been a member of the European Commission High Level Pharmaceutical Forum (2005-2008) and its Process on Corporate Responsibility in the Field of Pharmaceuticals (2010-2012).

MODERATOR

EuropaBio represents corporate and associate members across sectors, plus national and regional biotechnology associations which, in turn, represent over 2600 biotech companies, 2300 out of them are SMEs. EuropaBio is a founding member of the International Council of Biotech Associations (ICBA).