Improving the analysis of composite endpoints in rare disease trials
Abstract Background Composite endpoints are recommended in rare diseases to increase power and/or to sufficiently capture complexity. Often, they are in the form of responder indices which contain a mixture of continuous and binary components. Analyses of these outcomes typically treat them as binar...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
BMC
2018-05-01
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| Series: | Orphanet Journal of Rare Diseases |
| Subjects: | |
| Online Access: | http://link.springer.com/article/10.1186/s13023-018-0819-1 |