Real-world external control arm for the single-arm LIBRETTO-001 trial of selpercatinib in RET-mutation-positive medullary thyroid cancer: RECALIB-RET

Key Points

• The study evaluates the effectiveness of selpercatinib in patients with RET-mutation-positive medullary thyroid cancer compared to traditional treatments.
• Utilized retrospective real-world data to match with prospective trial data.
• Focused on treatment-naïve patients in the first-line setting.
• Assessed progression-free survival (PFS) outcomes against standard of care.
• Selpercatinib showed significant improvement in progression-free survival for treatment-naïve patients.
• Results were inconclusive for patients in the second-line treatment setting.
• The approach demonstrated feasibility of external control arms for single-arm trials.