Title: Monte Carlo modified profile likelihood in models for clustered data,
Abstract: The main focus of the analysts who deal with clustered data is usually not on
the clustering variables, and hence the group-specific parameters are treated
as nuisance. If a fixed effects formulation is preferred and the total number
of clusters is large relative to the single-group sizes, classical frequentist
techniques relying on the profile likelihood are often misleading. The use of
alternative tools, such as modifications to the profile likelihood or
integrated likelihoods, for making accurate inference on a parameter of
interest can be complicated by the presence of nonstandard modelling and/or
sampling assumptions. We show here how to employ Monte Carlo simulation in
order to approximate the modified profile likelihood in some of these
unconventional frameworks. The proposed solution is widely applicable and is
shown to retain the usual properties of the modified profile likelihood. The
approach is examined in two instances particularly relevant in applications,
i.e. missing-data models and survival models with unspecified censoring
distribution. The effectiveness of the proposed solution is validated via
simulation studies and two clinical trial applications.