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Orphan Drug Development Challenges: Case Studies
Session Chair(s)
Daniel Mazzolenis
Senior Medical Director, Global Oncology Hematology
INC Research, Argentina
In this symposium, three different case studies will showcase the multiple challenges in the successful development of new therapies for orphan diseases. The first case is about adjuvant treatment for phenylketonuria (PKU) and will center on systematic analysis of the current evidence and subsequent gap analysis as the basis to inform decision-making and future research. The second case focuses on hemophilia, a field with a large corps of knowledge regarding pathophysiology, therapeutic interventions and even comprehensive drug development guidelines. We intend to showcase precisely how the paradigms on which those guidelines are based may require revision in light of recent therapeutic developments, at the risk of regulation being an impediment to the continuous improvement. Finally, the last case will showcase how strategic planning and operational efficiency is critical for efficient and successful development of an orphan drug program.
Learning Objective : Discuss critical issues in drug development for rare/orphan diseases, from the review of evidence and gap analysis, the critical analysis of regulations and guidelines, and how they interact with development path lines; Describe how a comprehensive development strategy would serve therapy development.
Speaker(s)
Connecting the Dots for Fast-Track Approval for Rare Disease and Orphan Drugs
Michelle Petersen, MSc
Medpace, Inc., United States
Clinical Trial Manager
Challenges and Opportunities in Systematic Reviews of Rare Diseases: Adjuvant Treatment for Phenylketonuria
Christine Chang, MD
AHRQ, United States
Showcasing Hemophilia as a Rare/Orphan Disease: Do We Need to Update Regulatory Paradigms?
Pablo Rendo, MD
Pfizer Inc , United States
Senior Director, Physician Clinician, BeneFIX Global Clinical Lead
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