Introduction

Vandetanib (trade name: Caprelsa) has been approved in Germany since February 2012 for the treatment of advanced medullary thyroid cancer in adults. Since December 2016 it has also been approved for use in children over the age of five. The drug is an option for thyroid cancer if the course of the disease is aggressive and symptomatic.

When a malignant tumor is found in the thyroid gland, the tumor or the entire thyroid gland is removed surgically. But sometimes surgery is no longer possible because the tumor is already too large or has spread to other parts of the body (metastasis). This is known as advanced cancer.

There are different types of thyroid cancer. The medullary type is relatively rare. In some people with advanced medullary thyroid cancer the tumor is more aggressive than in other people and also causes typical symptoms such as a feeling of pressure in the throat, pain, diarrhea, or skin redness. The drug vandetanib inhibits certain proteins (enzymes) to prevent further tumor growth.

Application

Vandetanib is available in the form of tablets, in two different doses (100 and 300 mg). Adults take 300 mg once daily at the same time each day independently of meals. In children, the dose depends on the patient's body surface area.

The dose can be adjusted depending on the stage of the disease and side effects. Treatment is stopped if the disease gets worse or if very severe side effects arise.

Other treatments

Best supportive care (BSC) is an option for patients with aggressive and symptomatic medullary thyroid cancer that is already advanced. Supportive care should be tailored to the patient's individual needs, relieve symptoms such as pain, and improve quality of life.

Assessment

In 2013 the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into whether vandetanib has any advantages or disadvantages for adults with advanced medullary thyroid cancer compared with the standard treatments. In 2017 they did the same for children over the age of five and teenagers.

The manufacturer did not provide any suitable data on children over the age of five or teenagers for whom vandetanib is a treatment option. So it was not possible to draw any conclusions about the advantages or disadvantages of vandetanib for children compared with supportive care.

The manufacturer provided one study involving adults for whom vandetanib is a treatment option. All participants received supportive therapy, and two-thirds also took vandetanib.

What are the advantages of vandetanib for adults?

  • Pain: The results from the study provided weak evidence that additional treatment with vandetanib can delay the onset of pain or the increase in pain by almost eight months on average. However, this only applies to patients under the age of 65. There was no difference here in older people.

What are the disadvantages of vandetanib for adults?

  • Serious side effects: The results from the study provided weak evidence that serious side effects are more common in people who have additional treatment with vandetanib, though it cannot be estimated how much more common.
  • Rash: The results from the study provided weak evidence that skin rashes are more common in people have additional treatment with vandetanib.

No difference

  • Life expectancy: The study did not provide any evidence that adult patients who had treatment with vandetanib lived longer.

What remains unanswered?

  • Quality of life: There was not enough data to assess the effect of treatment with vandetanib on quality of life.

More information

This information summarizes the main results of reviews produced by the Institute for Quality and Efficiency in Health Care (IQWiG, Germany). The reviews were commissioned by the German Federal Joint Committee (G-BA) as part of the “early benefit assessment of medications.” On the basis of the reviews and the hearings received, the G-BA passed a resolution on the added benefit of vandetanib (Caprelsa).

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