Sample size calculation in randomised phase II selection trials using a margin of practical equivalence.

ABSTRACT:

Background

In rare cancers or subtypes of common cancers, a comparison of multiple promising treatments may be required. The selected treatment can then be assessed against the standard of care (if it exists) or used as a backbone for combinations with new, possibly targeted, agents. There could be different experimental therapies or different doses of the same therapy, and either can be done in combination with standard treatments. A 'pick-the-winner' design is often used, which focuses on efficacy to select the most promising treatment. However, a treatment with a slightly lower efficacy compared to another treatment may actually be preferred if it has a better toxicity or quality of life profile, is easier to administer, or cheaper.

Methods

By pre-defining a margin of pra

SUBMITTER: Dehbi HM 

PROVIDER: S-EPMC7106856 | biostudies-literature | 2020 Mar

REPOSITORIES: biostudies-literature