Statistical consideration when adding new arms to ongoing clinical trials: the potentials and the caveats

Area of Science:

• Clinical Trials
• Biostatistics
• Drug Development

Background:

• Platform trials offer flexibility in drug development by allowing arms to be added or dropped.
• While benefits are known, statistical implications of adding arms in ongoing studies require further examination.

Discussion:

• This study details statistical considerations for incorporating new interventions into ongoing platform trials.
• It covers methodology for robust inference, control arm changes, patient eligibility complexities, and operational bias.

Key Insights:

• Recent methodological developments address statistical challenges in platform trial design and analysis.
• Robust inference is achievable through enhanced design and analysis approaches.

Outlook:

• Platform trials present opportunities for efficient intervention evaluation.
• Available statistical methods support robust and efficient platform trial design and analysis in various settings.