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Development of Orphan Biologics: Challenges and Opportunities

Webinar sponsored by Catalent

18 April 2023 @ 3:00 pm 4:00 pm CEST

The development of therapies for rare diseases comes with distinct challenges, including patient recruitment, setting meaningful clinical endpoints, manufacturing and supply and finding the regulatory and market pathway to ensure patients can access therapy.

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.

These challenges sit within a wider regulatory and legislative framework for orphan medicinal products (OMPs) that defines whether companies can successfully develop a novel product to market and thus the decisions made for selecting therapeutics and target diseases early in development.

During this webinar, speakers cover aspects of clinical trial design for orphan biologics products, key strategies for ensuring clinical trial supply throughout clinical development and the regulatory and legislative framework for delivery to patients.

Key Learning Objectives

  • Considerations in identifying meaningful clinical endpoints for orphan diseases
  • Orphan clinical trial design considerations and strategies to ensure sufficient patient recruitment
  • Manufacturing and clinical supply strategies to ensure supply of clinical trial materials while collecting data needed to support NDA submission
  • Regulatory and legislative frameworks that guide decision making for innovation in rare diseases


15:00: Welcome and introduction – Claire Skentelbery, Director General EuropaBio

15:05: Current Development of Orphan Drugs in EuropeMatthias Schoenermark, Professor, President and CEO, SKC Beratungsgesellschaft mbH

Prof. Schönermark opens the session with an overview of the current status of orphan drug development in the EU. He discusses the main challenges for market entry of new orphan drugs into the EU market and highlights key recommendations to drug developers to address these challenges.

15:20: Orphan Biologics and CGT Development – CMC and Clinical Trial Supply Considerations – Wai Lam Ling, Ph.D., Vice President, Science and Technology,  Catalent

Dr. Ling discusses the difference in CMC challenges encountered during an orphan biologics vs CGT programme, with a focus on product quality as the most significant hurdle to approval. She addresses manufacturing approaches to ensure regulatory compliance and factors behind a successful product, particularly robust, reproducible and cost-effective pathways. Finally, supply chain liabilities are discussed, with risks to production of biologics and their clinical supply.

15:45: Successful Strategies and Business Model For Companies Bringing Their Orphan Drug To Market Thomas Bols, Head of Government Affairs and Patient Advocacy, EMEA & APAC, PTC Therapeutics

Thomas Bols addresses the spectrum of opportunities and barriers for innovation within orphan drug development, particularly across different regulatory territories, and the critical role of smaller companies in addressing unmet medical needs. He focuses on the legislative and regulatory pathways that exist in Europe for orphan drugs and the potential impact of the upcoming pharmaceutical and OMP legislation revisions for company innovation and patient benefits.

16:00: Close


+32 2 735 03 13

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