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Session 8: Orphan Drugs and Advanced Therapies for Rare Diseases: Bringing Transformational Treatments to Patients with Unmet Medical Needs
Session Chair(s)
Maria Antonieta Tony Roman, MPharm
Head Regulatory Policy Emerging Markets LATAM
Novartis, Mexico
Sonia Viejobueno, LLM
US and Latin America Policy Lead, Global Regulatory Policy and Intelligence
Johnson & Johnson Innovative Medicine, Argentina
Orphan Drugs and Advanced Therapies aimed at treating rare and ultra-rare diseases, represent one of the fastest growing sectors in the biopharmaceutical space by offering the promise of personalized treatments for patients with highly unmet medical needs. However, the use of increasingly innovative technologies and regulatory tools, such as real-world data (RWD) and real-world evidence (RWE), also provides for operational and regulatory challenges during the course of development of these highly complex therapies. In this session, we will discuss the global rare disease landscape and identify existing opportunities for accelerating clinical development with innovative regulatory pathways and the use of modern regulatory tools, including RWE to facilitate development and patient access to orphan drugs and advanced therapies.
Learning Objective : At the conclusion of this session, participants should be able to:- Recognize the global and regional regulatory landscape to address rare diseases
- Identify the unique features and main challenges of cell/gene therapy product development and their specific regulatory landscape
- Describe the use of RWD and RWE for regulatory decision-making applied to orphan drugs and advanced therapies
Speaker(s)
FDA Approach to Orphan Rare Diseases and Gene Therapy Development/Industry Perspective
Ilan Irony, MD
Janssen Pharmaceutical Companies of Johnson and Johnson, United States
Senior Director, Global Regulatory Leader
Role of RWE for Evidence Generation in Rare Diseases/Advanced Therapies
Miriam Fuchs, PhD
Novartis, Switzerland
Global Therapeutic Area Lead, Reg. Affairs, Oncology Cell & Gene Therapies
AT and Orphan Regulatory Pathway in Brazil
Joao Batista Silva Junior, MHS, RAC
ANVISA, Brazil
Manager of Blood, Tissues, Cells, Organs and Advanced Therapy Products Office
Post Marketing Activities in Advanced Therapies
Patrick Celis, PhD
European Medicines Agency, Netherlands
Scientific Administrator
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