煌めく治験の未来　～医師主導治験を成功に導く生物統計学の実践～

抄録

Physician–initiated clinical trials constitute a critical framework for advancing therapeutic innovation, particularly in areas where existing treatments are inadequate. In neurological diseases―many of which are rare or refractory to standard care―such investigator–driven studies offer essential pathways for developing novel interventions. The conduct of these trials requires careful attention to multiple components, including precise study design, ethical oversight, appropriate funding, and rigorous data analysis. Biostatistics plays a foundational role in ensuring methodological integrity and scientific validity throughout this process. This article examines the essential contributions of biostatistics to physician–led clinical research, illustrated by selected case studies that highlight both practical applications and broader implications for future trial design.