New Treatment for Thyroid Cancer Available Through Cancer Drugs Fund

Dawn O'Shea

November 05, 2021

NICE has published new guidance recommending selpercatinib for use within the Cancer Drugs Fund (CDF), as an option for: 

The main clinical evidence for selpercatinib comes from the ongoing single-arm, open-label, multicentre phase 1 to 2 LIBRETTO‑001 trial in people with advanced solid tumours, with RET activations. The primary outcome of the trial was objective response rate.

The company presented three analysis sets for people with RET-mutant medullary thyroid cancer. The primary analysis set included 55 people who had previously had either cabozantinib, vandetanib, or both. The supplementary analysis set included 88 people with RET-mutant MTC who had not had cabozantinib or vandetanib, and an integrated analysis set included 124 patients from the primary analysis set.

For people with RET fusion-positive thyroid cancer, the trial reported results for people whose disease has been previously treated with a systemic therapy (n=19) and those whose disease was untreated (n=8).

For people with previously treated MTC, the objective response rate was 69% and median progression-free survival (PFS) could not be estimated.

For people with previously treated advanced RET fusion-positive thyroid cancer, the objective response rate was 79%, and median PFS was 20.07 months (95% CI 9.4 to not estimatable).

Median overall survival (OS) in all subgroups for RET fusion-positive thyroid cancer and RET-mutant MTC were also not estimable.

However, NICE said that the clinical evidence is highly uncertain because it is based on an ongoing single-arm trial and not all subpopulations within the trial represent NHS practice. It was also felt that the results of an indirect comparison of selpercatinib with best supportive care were highly uncertain.

NICE concluded that the treatment could be cost-effective if more data from LIBRETTO‑001 shows improved overall survival, and real-life NHS data could add further value. Therefore, the treatment will be available on the CDF so that more data can be collected.


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