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Medullary thyroid cancer (MTC) is frequently driven by alterations in RET. The multi-kinase inhibitors (MKIs) vandetanib and cabozantinib — the current standard-of-care for advanced MTC — were approved over a decade ago based on a progression-free survival (PFS) benefit over placebo in phase 3 randomized clinical trials. Recently, the highly selective RET inhibitors selpercatinib and pralsetinib were approved for advanced RET-mutant MTC based on encouraging activity in phase 1–2 trials, with overall response rates of 60% to 70% in RET-mutant MTCs (N Engl J Med 2020; 383:825; Lancet Diabetes Endocrinol 2021; 9:491).
In this industry-funded, phase 3, randomized, controlled trial, researchers compared the efficacy of selpercatinib to physician'…