NDLI: Endpoints and Analyses to Discern Disease-Modifying Drug Effects in Early Parkinson’s Disease

Content Provider	PubMed Central
Author	Bhattaram, Venkatesh Atul Siddiqui, Ohidul Kapcala, Leonard P. Gobburu, Jogarao V. S.
Abstract	Parkinson’s disease is an age-related degenerative disorder of the central nervous system that often impairs the sufferer’s motor skills and speech, as well as other functions. Symptoms can include tremor, stiffness, slowness of movement, and impaired balance. An estimated four million people worldwide suffer from the disease, which usually affects people over the age of 60. Presently, there is no precedent for approving any drug as having a modifying effect (i.e., slowing or delaying) for disease progression of Parkinson’s disease. Clinical trial designs such as delayed start and withdrawal are being proposed to discern symptomatic and protective effects. The current work focused on understanding the features of delayed start design using prior knowledge from published and data submitted to US Food and Drug Administration (US FDA) as part of drug approval or protocol evaluation. Clinical trial simulations were conducted to evaluate the false-positive rate, power under a new statistical analysis methodology, and various scenarios leading to patient discontinuations from clinical trials. The outcome of this work is part of the ongoing discussion between the US FDA and the pharmaceutical industry on the standards required for demonstrating disease-modifying effect using delayed start design.
Related Links	http://dx.doi.org/10.1208/s12248-009-9123-2
Starting Page	456
File Format	PDF
ISSN	15507416
e-ISSN	15507416
Journal	The AAPS Journal
Issue Number	3
Volume Number	11
Language	English
Publisher	Springer US
Publisher Date	2009-09-01
Access Restriction	Open
Rights Holder	Springer US
Subject Keyword	Pharmaceutical Science Research in Higher Education
Content Type	Text
Resource Type	Article
Subject	Pharmaceutical Science

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