Design of brain injury rehabilitation treatment research

Rehabilitation is a key service component in the context of significant brain injury, yet many of the treatments and services employed in rehabilitation are not based on rigorous evidence. Treatment research in rehabilitation, like in other fields of healthcare, relies on a developmental sequence of studies that address different questions, including safety, proof of principle, definitive efficacy, and real-world effectiveness. Each of these steps faces challenges specific to the rehabilitation domain, where many treatments are nonpharmacologic, and outcomes of interest are highly varied and complex. This chapter frames the research questions addressed by different phases of treatment research, identifies some of the particular challenges in answering these questions, and takes a hypothetical treatment through the sequence to illustrate the process.