Considerable added benefit of ipilimumab in advanced melanoma, study suggests

ScienceDaily (Nov. 9, 2012) The humanized antibody ipilimumab (trade name Yervoy) has been approved since August 2011 for the treatment of adult patients with advanced melanoma (black skin cancer) who have already been treated. The term "advanced" means that the melanoma can no longer be removed by an operation or that metastases have formed. The German Institute for Quality and Efficiency in Health Care (IQWiG) has examined the added benefit of the drug pursuant to the Act on the Reform of the Market for Medicinal Products (AMNOG). According to the findings, the drug offers major advantages with respect to overall survival, but is accompanied by major potential harm in the form of side effects. The overall conclusion is that ipilimumab offers considerable added benefit.

"Best supportive care" as appropriate comparator therapy

As specified by the Federal Joint Committee (G-BA), ipilimumab was to be compared with so-called "best supportive care." This means the best possible supportive therapy, optimized for the individual patient, which guarantees alleviation of the symptoms and improvement in the quality of life.

The only study relevant to this benefit assessment compared ipilimumab in combination with "best supportive care" with "best supportive care" alone. This study was informative (randomized controlled trial, RCT) and included three parallel study arms. Patients in arm 1 were treated with ipilimumab. Patients in arm 2 were treated with ipilimumab and an experimental tumour vaccine (gp100), which has been examined in studies since 1996, but which has not yet been submitted for approval. The patients in the comparator arm (arm 3) were given a placebo and the tumour vaccine. All patients in the study were also given "best supportive care."

In its assessment, the Institute came to the conclusion that the administration of gp 100 had no relevant influence on the effects of ipilimumab measured in this study, in comparison to "best supportive care."

Longer survival means major added benefit

The assessments performed by IQWiG always concentrate on patient-relevant outcomes, such as survival time, symptoms and complaints and quality of life. The study provided an indication that ipilimumab can prolong life. Whereas half of the patients who had not received ipilimumab already died after 6.5 months, half of the patients who had received ipilimumab died after 10 months. This prolongation of life provides an indication of major added benefit of ipilimumab in combination with "best supportive care" in comparison to "best supportive care" alone.

Symptoms and complaints caused by the disease (morbidity) were not examined in the study. Thus, added benefit for ipilimumab for these outcomes is not proven.

There was no relevant difference between ipilimumab and placebo with respect to quality of life (general state of health, functionality, symptoms such as fatigue, nausea, pain, digestive disorders etc.). Thus there is no proof of added benefit.

Major potential harm in the form of side effects

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Considerable added benefit of ipilimumab in advanced melanoma, study suggests