Premium
Using global statistical tests in long‐term Parkinson's disease clinical trials
Author(s) -
Huang Peng,
Goetz Christopher G.,
Woolson Robert F.,
Tilley Barbara,
Kerr Douglas,
Palesch Yuko,
Elm Jordan,
Ravina Bernard,
Bergmann Kenneth J.,
Kieburtz Karl
Publication year - 2009
Publication title -
movement disorders
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 3.352
H-Index - 198
eISSN - 1531-8257
pISSN - 0885-3185
DOI - 10.1002/mds.22645
Subject(s) - clinical trial , parkinson's disease , quality of life (healthcare) , disease , multivariate statistics , multivariate analysis , medicine , statistical significance , psychology , computer science , machine learning , nursing
Abstract Parkinson's disease (PD) impairments are multidimensional, making it difficult to choose a single primary outcome when evaluating treatments to stop or lessen the long‐term decline in PD. We review commonly used multivariate statistical methods for assessing a treatment's global impact, and we highlight the novel Global Statistical Test (GST) methodology. We compare the GST to other multivariate approaches using data from two PD trials. In one trial where the treatment showed consistent improvement on all primary and secondary outcomes, the GST was more powerful than other methods in demonstrating significant improvement. In the trial where treatment induced both improvement and deterioration in key outcomes, the GST failed to demonstrate statistical evidence even though other techniques showed significant improvement. Based on the statistical properties of the GST and its relevance to overall treatment benefit, the GST appears particularly well suited for a disease like PD where disability and impairment reflect dysfunction of diverse brain systems and where both disease and treatment side effects impact quality of life. In future long term trials, use of GST for primary statistical analysis would allow the assessment of clinically relevant outcomes rather than the artificial selection of a single primary outcome. © 2009 Movement Disorder Society