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Mar 14, 2022 10:00 AM - Mar 16, 2022 3:15 PM

(US Eastern Standard Time)

Latin America Regulatory Conference

DIA Your Way! Join us virtually from the comfort of your home/office March 14-16 or watch later at a time that works for you. This event provides access to the materials for a full two months!

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

Maria Antonieta Tony Roman, MPharm

Head Regulatory Policy Emerging Markets LATAM

Novartis, Mexico

Sonia  Viejobueno, LLM

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


Ilan  Irony, MD

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

Miriam  Fuchs, PhD

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

Joao  Batista Silva Junior, MHS, RAC

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

Patrick  Celis, PhD

Post Marketing Activities in Advanced Therapies

Patrick Celis, PhD

European Medicines Agency, Netherlands

Scientific Administrator

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