Sep 1, 2025
Selpercatinib in medullary thyroid cancer: RCT shows substantial survival advantages
Following the expiry of the first decision: Reassessment of the drug for the first-line treatment of RET-mutant medullary thyroid cancer shows an indication of a major added benefit.
Selpercatinib is approved for several therapeutic indications, including as monotherapy for the treatment of advanced RET-mutant medullary thyroid cancer in the first line. The drug blocks an enzyme that has mutated in the cancer cells and constantly sends them growth signals. The blockade is intended to prevent the cancer cells from dividing further.
The first benefit assessment in this therapeutic indication ended in 2023 with a temporary decision from the Federal Joint Committee ( G‑BA), because an ongoing randomized controlled trial had not yet delivered any results.
The relevant data are now available, so that the Institute for Quality and Efficiency in Health Care (IQWiG) has been able to conduct a further benefit assessment. The result: Compared with the appropriate comparator therapies vandetanib or cabozantinib, selpercatinib as monotherapy shows numerous and, in some cases, clear advantages. Consequently, there is an indication of a major added benefit of selpercatinib monotherapy.
Advantages in many outcomes
The phase 3 study LIBRETTO-531 was already underway at the time of marketing authorization and the first benefit assessment, but results were not yet available. Informative study results are now available and for the outcome of overall survival there is an indication of a clear added benefit. There are hints of an added benefit in several other outcomes such as fatigue, pain and health-related quality of life. “While over 16% of the patients included in the comparator arm died, only 5% died in the intervention arm,” explains Katrin Nink, Head of the Oncology Division (solid tumours) at IQWiG’s Drug Assessment Department. “Such a clear difference is remarkable.”
The G‑BA decides on the extent of the added benefit
The dossier assessment is part of the early benefit assessment in accordance with the Act on the Reform of the Market for Medicinal Products (AMNOG) supervised by the G‑BA. After publication of the dossier assessment, the G‑BA conducts a commenting procedure and makes a decision on the extent of the added benefit.