Disease modifying trials in Alzheimer disease: methodological and statistical issues.
In order to establish that a drug has an impact on disease progression, a clinical trial must study both the symptomatic and the disease modifying effect of the drug. Disease modifying trials raise some methodological issues with regard to the choice of the design (parallel arm versus two- period design), of the outcome (clinical versus surrogate) and of the statistical analysis (management of missing data).
Unite INSERM 558, Service d'epidemiologie, CHU Toulouse, France.
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Andrieu
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Rascol
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Lang
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Grandjean
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Vellas
D000544:Alzheimer Disease; D002986:Clinical Trials as Topic / Q000379:methods* / Q000706:statistics & numerical data*; D018450:Disease Progression; D006801:Humans; D015233:Models, Statistical; D012107:Research Design