New drug available for rare thyroid cancer on NHS in England

The National Institute for Health and Care Excellence (NICE) has recommended the use of the drug selpercatinib (Retevmo) for some people in England with a type of advanced thyroid cancer.

The drug is now available through the Cancer Drugs Fund, which helps provide access to promising new treatments, as an option for patients with advanced thyroid cancers that have abnormalities in a gene called RET.

Whilst the clinical trial for selpercatinib is still ongoing, current results are positive, with around 70 to 80% of recipients responding to the treatment. The drug is also likely to have fewer side effects than current treatments. However, uncertainties remain as more data is needed.

“This decision is good news for people affected by these rare and aggressive types of thyroid cancer,” said Kruti Shrotri, head of policy development at Cancer Research UK.

“Because this drug has been approved through the Cancer Drugs Fund, patients who could benefit from it in England will be able to access it quickly while more evidence is gathered on its effectiveness.”

Targeting a rare and aggressive cancer

Selpercatinib has been approved to treat patients with a rare, advanced thyroid cancer if they need additional therapy after initial treatment. This specifically includes:

• Adults with advanced RET fusion-positive thyroid cancer who need systemic therapy after sorafenib (Nexavar) or lenvatinib (Lenvima, Kisplyx);
• People 12 years and older with advanced RET-mutant medullary thyroid cancer who need systemic therapy after cabozantinib (Cometriq, Cabometyx) or vandetanib (Caprelsa).

These types of thyroid cancer are rare, and abnormalities in the RET gene are associated with more aggressive disease and poorer outcomes for patients.

Patient experts noted that thyroid cancer can be devastating for people and their families, not only because of the shock of the initial diagnosis, but also because of the relative lack of treatment options that are available.

Some of the current treatments, including the targeted drugs lenvatinib and sorafenib, are also associated with significant side effects.

As selpercatinib specifically targets RET abnormalities, clinical experts noted that it would likely provide benefit to patients whilst being less toxic than currently available treatments, potentially improving the quality of life for people with these cancers.

A chance to prove itself

The main evidence for selpercatinib’s effectiveness is from the LIBRETTO-001 study, an ongoing phase 1/2 trial in people with advanced solid tumours with RET abnormalities.

The results so far are positive, but uncertainties remain.

Currently, the trial shows that nearly 8 in 10 people with previously treated advanced RET fusion-positive thyroid cancer responded to the treatment, living on average 20 months without their disease progressing.

And almost 7 in 10 people with previously treated medullary thyroid cancer responded to treatment, but with data still be collected, it’s too early to estimate the average amount of time patients will live without their disease progressing.

This also means that it’s too early to estimate how long people live for after taking selpercatinib for both groups.

Because of this, and the fact that the trial doesn’t directly compare selpercatinib to current treatment options, and includes a relatively small number of people, the NICE committee consider the evidence to be uncertain.

Despite this, the potential benefit of selpercatinib is high, which is why NICE are making the drug available through the Cancer Drugs Fund so that more data can be collected.

Drugs approved by NICE for use through the Cancer Drug Fund in England are normally considered in Northern Ireland in line with existing arrangements for endorsement of NICE recommendations.

If more data shows that selpercatinib works well and is cost-effective, the drug will be considered for routine use in the NHS in 2024. If approved by NICE for use in England, this decision will likely then be adopted in Wales and Northern Ireland, while Scotland has a separate process for reviewing drugs.