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Session 5, Track A: Orphan and Rare Disease Management: Are Patients In LatAm Getting the Advanced Therapies They Need?
Session Chair(s)
Carolina Sian, PharmD, RAC
Regulatory Affairs Director
CAEME, Argentina
Lawrence Liberti, PhD, RAC
Director, D.K. Kim International Center for Regulatory Science
The Kim Center/ USC DRQS, United States
As the identification of rare diseases become more sophisticated, so too do their therapies. The growing focus on advanced medicinal therapeutic products (ATMPs), biologics and specialized gene therapies for orphan diseases poses not only challenges during development but also for their regulatory assessments. This session will investigate challenges such as the use of early clinical data and decisions from other NRAs based on phase 2 or small, yet fit-for-purpose datasets. Is import testing of ATMPs being optimized or are products which are developed in precious small quantities being needlessly wasted? What do patients expect from developers and regulators to meet their unique needs?
Learning Objective :
At the conclusion of this session, participants should be able to:
- Define the scope of rare diseases in LatAm
- Characterize the similarities and diversities among regulatory approaches to assess rare disease therapeutics
- Identify opportunities to optimize regulatory approaches to the efficient review of rare disease therapeutics
- Understand the needs of patients affected by these rare diseases
Speaker(s)
Orphan and Rare Disease Management: Are Patients In Latam Getting the Advanced Therapies They Need?
Roberta Eleonora Anido
FADEPOF, Argentina
Patient Advocate
Orphan and Rare Disease Management: Are Patients in LatAm Getting the Therapies They Need?
Maria Antonieta Roman, MPharm
Novartis, Mexico
Head Regulatory Policy LaCan
Regulation of Advanced Therapies in Argentina: Status, Experience, and Challenges of ANMAT
Gabriela Beatriz Bravo
ANMAT Ministry of Health, Argentina
Biologics Regulatory Reviewer