Clinical Trial Design in Rare Diseases: Special Considerations - UMD
嚜澧linical Trial Design in Rare Diseases:
Special Considerations
Patroula Smpokou, MD, FACMG
Clinical team leader
Division of Gastroenterology and Inborn Errors Products
Office of New Drugs
Center for Drug Evaluation and Research
Food and Drug Administration
FDA Clinical Investigator Training Course
November 13, 2018
1
Disclaimer
The views expressed in this presentation are those
of the speaker and do not represent an official
FDA position.
I have no financial interests to disclose.
2
Acknowledgments
Laurie Muldowney, MD
Yeh-Fong Chen, PhD
Christine Yuen-Yi Hon, PharmD
Yow-Ming Wang, PhD
Robert (Skip) Nelson, MD
Dragos Roman, MD
Julie Beitz, MD
3
Outline
? Application of US regulatory framework to rare disease
product development
? Challenges in trial design for rare diseases
每 Natural history
每 Statistical considerations
每 Dose selection
每 Ethical considerations in pediatric clinical investigations
4
Definitions
?
Rare disease: disease or condition affecting < 200,000 people in the United States
?
Orphan Drug Designation:
每
Investigational product must be intended to treat a rare disease
每
Adequate demonstration of a medical plausibility for the drug*s expected benefit
OR
每
A product for which there is no ※reasonable expectation§ that the development costs would be
recovered from U.S. sales
?
Orphan Designation is an incentive to encourage drug development
?
Orphan designation applies to specific product AND specific disease
5
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