Clinical Trial Design in Rare Diseases: Special Considerations - UMD

Clinical 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

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Outline

? Application of US regulatory framework to rare disease

product development

? Challenges in trial design for rare diseases

¨C Natural history

¨C Statistical considerations

¨C Dose selection

¨C Ethical considerations in pediatric clinical investigations

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Definitions

?

Rare disease: disease or condition affecting < 200,000 people in the United States

?

Orphan Drug Designation:

¨C

Investigational product must be intended to treat a rare disease

¨C

Adequate demonstration of a medical plausibility for the drug¡¯s expected benefit

OR

¨C

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

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