Composite endpoints, including patient reported outcomes, in rare diseases.

ABSTRACT:

Background

When assessing the efficacy of a treatment in any clinical trial, it is recommended by the International Conference on Harmonisation to select a single meaningful endpoint. However, a single endpoint is often not sufficient to reflect the full clinical benefit of a treatment in multifaceted diseases, which is often the case in rare diseases. Therefore, the use of a combination of several clinically meaningful outcomes is preferred. Many methodologies that allow for combining outcomes in a so-called composite endpoint are however limited in a number of ways, not in the least in the number and type of outcomes that can be combined and in the poor small-sample properties. Moreover, patient reported outcomes, such as quality of life, often cannot be integrated in a composite

SUBMITTER: Verbeeck J 

PROVIDER: S-EPMC10474650 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature