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A flexible multi-domain test with adaptive weights and its application to clinical trials.
- Source :
-
Pharmaceutical statistics [Pharm Stat] 2020 May; Vol. 19 (3), pp. 315-325. Date of Electronic Publication: 2019 Dec 30. - Publication Year :
- 2020
-
Abstract
- The design of a clinical trial is often complicated by the multi-systemic nature of the disease; a single endpoint often cannot capture the spectrum of potential therapeutic benefits. Multi-domain outcomes which take into account patient heterogeneity of disease presentation through measurements of multiple symptom/functional domains are an attractive alternative to a single endpoint. A multi-domain test with adaptive weights is proposed to synthesize the evidence of treatment efficacy over numerous disease domains. The test is a weighted sum of domain-specific test statistics with weights selected adaptively via a data-driven algorithm. The null distribution of the test statistic is constructed empirically through resampling and does not require estimation of the covariance structure of domain-specific test statistics. Simulations show that the proposed test controls the type I error rate, and has increased power over other methods such as the O'Brien and Wei-Lachin tests in scenarios reflective of clinical trial settings. Data from a clinical trial in a rare lysosomal storage disorder were used to illustrate the properties of the proposed test. As a strategy of combining marginal test statistics, the proposed test is flexible and readily applicable to a variety of clinical trial scenarios.<br /> (© 2019 John Wiley & Sons Ltd.)
- Subjects :
- Data Interpretation, Statistical
Double-Blind Method
Functional Status
Humans
Models, Statistical
Mucopolysaccharidosis I diagnosis
Mucopolysaccharidosis I physiopathology
Mucopolysaccharidosis I therapy
Recovery of Function
Treatment Outcome
Endpoint Determination statistics & numerical data
Randomized Controlled Trials as Topic statistics & numerical data
Research Design statistics & numerical data
Subjects
Details
- Language :
- English
- ISSN :
- 1539-1612
- Volume :
- 19
- Issue :
- 3
- Database :
- MEDLINE
- Journal :
- Pharmaceutical statistics
- Publication Type :
- Academic Journal
- Accession number :
- 31886602
- Full Text :
- https://doi.org/10.1002/pst.1993