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Session 8, Track A: Regulation of Medicinal Products for Rare Disease: Challenges and Opportunities
Session Chair(s)
Viktoria Magyar, LLM, MSc
Doctoral Student, Department of Regulatory and Quality Sciences
USC Alfred E. Mann School of Pharmacy and Pharmaceutical Sciences, United States
Cammilla Horta Gomes, MA, MPharm
Latam Regulatory Policy Lead
Roche, Brazil
This session will discuss the need to adopt regulatory framework, pathways, and tools to allow for timely access to medicinal products for rare diseases. Attention will be drawn to regulatory tools to prioritize or expedite the assessment and registration of treatments benefitting patients with rare diseases in Latin America. A discussion of international best practices and regional trends in the regulation of treatments for rare diseases, and how to bridge regulatory gaps through fostering discussions among regulators and industry representatives, will also be covered.
Learning Objective : - Recognize concepts and international best practices in the regulation of rare diseases
- Identify current regulatory trends in Latin America
- Understand the potential for evolving regulatory frameworks to facilitate greater access to innovative therapies for rare diseases
- Capture the essence of a case study illustrating the use of priority registration pathway in Brazil
Speaker(s)
FDA Regulatory Considerations for Drug Development in Rare Diseases
Daniela Varela Luquetti, MD, PhD
FDA, United States
Lead Clinical Analyst, CDER
Rare Disease in Brazil: Case Study
Raquel Pinheiro, MBA, RPh
Johnson & Johnson, Brazil
Regulatory Affairs Sr. Manager
Speaker
Claudia Saidman, DrMed, MD
ANMAT Ministry of Health, Argentina
Director of Clinical Research and Management of the Medicines Registry
Speaker
Marlene Esquivel, RPh
DINAVISA, Paraguay
Department of Biological and Radiopharmaceuticals
Speaker
Indhira Johanna Bernuy Zagaceta
DIGEMID, Peru
Executive Director of Pharmaceutical Products
Speaker
Karina Cuadra, RPh
Ministry of Public Health - Uruguay , Uruguay
Head of Evaluation Sector
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