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Section 5 - Part

Published online by Cambridge University Press:  05 May 2012

Edited by
Bernard Ravina ,
Jeffrey Cummings ,
Michael McDermott  and
R. Michael Poole
Bernard Ravina
Affiliation:
Biogen Idec., Cambridge, MA
Jeffrey Cummings
Affiliation:
Cleveland Clinic, Lou Ruvo Center for Brain Health, Las Vegas
Michael McDermott
Affiliation:
University of Rochester
R. Michael Poole
Affiliation:
AstraZeneca PLC, Waltham, MA, US
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Summary

The evidentiary standards for the approval of drugs to treat human disease are set forth in the relevant sections of the Food, Drug, and Cosmetic Act. This chapter focuses on some of the more important and current issues related to the demonstration of effectiveness of drugs and biologics. In essentially all cases, drugs to treat neurological disease are approved on the basis of clinical trials that examine the drug's effects on a face valid measurement with clinical meaning. The FDA defines orphan diseases as those with a prevalence of less than 200000 in the US. In the case of a trial designed to establish the superior safety profile of one drug compared to another, it is also critical that the trials examine a full range of adverse events, and employ methods sensitive enough to adequately assess them.

Keywords

placebotherapeutic validationendpoint modelMRIneurology clinical trialsearly-stage clinical trial designbiomarkercomputed tomography
Type
Chapter
Information
Clinical Trials in Neurology
Design, Conduct, Analysis
 , pp. 197 - 214
Publisher: Cambridge University Press
Print publication year: 2012

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