Selpercatinib in medullary thyroid cancer: RCT shows substantial survival advantages

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.