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Optimizing Orphan Drug Development and Using Appropriate Methodology: Key Tips for Success

Track:
Rare/Orphan Diseases

Day & Time:
June 18, 1:30PM - 3:00PM (Pacific Standard Time)

Session Number:
375

Room Number:
32AB

Level:
Intermediate

Type:
Symposium

Title:
Optimizing Orphan Drug Development and Using Appropriate Methodology: Key Tips for Success

Chair(s):
Gopalan Narayanan, MD, MRCP, FFPM, FRCP
Biologicals and ATMP Expert
NDA Regulatory Science Ltd., United Kingdom

Description:
Around 7,000 diseases are considered rare/orphan diseases, and licensed medicines are available for only a fraction of these. The rarity of a disease makes novel drug development even more of a complex process. Navigating drug development for regulatory submissions requires special knowledge and expertise in order to make efficient use of resources, while at the same time maintaining a high standard of any new medical intervention. This symposium will cover key aspects of orphan drug development with tips on clinical development and regulatory strategy, using examples to illustrate the points. We will provide advice on how to overcome practical problems such as choosing the best CRO for the clinical trials, for example. It will also explore new methodologies in gathering relevant and required data to strengthen the evidence base, including use of registries and past research records.

Learning Objective(s):
Describe how to plan the clinical development of orphan products including program risk assessment and possible solutions for gaps; Plan an efficient global regulatory strategy; Use optimum methodology for gathering evidence.

Presentation(s) & Speaker(s):
Developing Products for Orphan Diseases: Perspectives of an Ex-Regulator
Gopalan Narayanan, MD, MRCP, FFPM, FRCP
Biologicals and ATMP Expert
NDA Regulatory Science Ltd., United Kingdom

Key Tips for Orphan Product Development
David G Shoemaker, PhD
Senior Vice President, R&D
Rho, Inc., United States

Management of Rare Diseases: Orphan Drug Discovery Combining Evidence-Based Medicine and Genetic Information
Manish Khatri, MS
Senior Consultant - Business Strategy, Life Sciences
Cognizant Business Consulting, United States