Evolution of CIDP Clinical Trials and Patient Protection

Clinical research in chronic inflammatory demyelinating polyneuropathy (CIDP) has advanced substantially over the past two decades, moving from exploratory, investigator-driven studies toward more refined, patient-centered trial designs. Historically, the lack of FDA-approved therapies forced reliance on small, heterogeneous studies that often included patients with uncertain disease activity. Modern CIDP trials now incorporate stricter inclusion criteria, objective biomarkers, and stronger patient-safeguard frameworks to ensure meaningful data collection while minimizing participant risk. Still, unique challenges remain—particularly around identifying patients in true disease remission and managing the ethics of treatment withdrawal during placebo or washout phases.

In this episode of Advancing CIDP Care, Amit Sachdev, MD, discusses how these trials have evolved to better balance scientific rigor with patient safety. He explains how withdrawal periods in earlier study designs created recruitment hurdles and long-term concerns, and how more recent protocols have shifted toward flexible, rescue-based approaches. Dr. Sachdev also emphasizes what neurologists should communicate to patients when discussing trial participation—especially the importance of recognizing treatment plateaus, reassessing goals, and considering clinical trial enrollment when existing therapy no longer meets disease-control needs.