Ibrutinib, trade name Imbruvica, is approved by the FDA to treat chronic lymphocytic leukemia in patients who have already received other treatment, mantle cell lymphoma in patients who have already received other treatment, and Waldenström macroglobulinemia, a form of non-Hodgkin lymphoma.
Upon a request from the German Federal Joint Committee (G-BA), Johnson & Johnson’s Janssen Pharmaceutical division, the manufacturer of Ibrutinib, has now submitted a new dossier. This is because the turnover of the drug in the statutory health insurance exceeded 50 million euros in the preceding 12 months.
The German Institute for Quality and Efficiency in Health Care (IQWiG) therefore examined in an early benefit assessment whether the drug offers an added benefit for patients with these diseases in comparison with the respective appropriate comparator therapies.
As per the findings, there is no added benefit in chronic lymphocytic leukemia (CLL) and Waldenström macroglobulinaemia.
In the case of refractory mantle cell lymphoma, however, there is an indication of major added benefit of ibrutinib for patients for whom temsirolimus is the individually optimized treatment option. An added benefit is not proven for patients for whom temsirolimus is no or only a secondary option.
Refractory Mantle Cell Lymphoma
MCL is an aggressive type of B-cell non-Hodgkin lymphoma (NHL) that usually occurs in older adults. The disease typically begins in the lymph nodes, but can spread to other tissues, such as bone marrow and the liver. Ibrutinib targets the B-cell receptor pathway, an important pathway in malignant B-cell growth and proliferation.
In the United States, there are approximately 5,000 new cases of MCL each year.
The G-BA separated relapsed or refractory MCL patients into two subpopulations. Those were patients for whom temsirolimus is the individually optimized treatment, and patients for whom this is not the case.
Because of a lack of data, added benefit of ibrutinib is not proven for patients for whom temsirolimus is no or only a secondary treatment option.
For the other patient group, however, the manufacturer presented data from the study MCL3001, in which ibrutinib was compared with temsirolimus. There was no statistically significant difference between the study arms regarding overall survival.
Ibrutinib had positive effects in the outcomes “health status” and “side effects,” which were not offset by negative effects in other outcomes. Overall, there is therefore an indication of major added benefit for patients for whom temsirolimus constitutes the individually optimized treatment.
According to the Wall Street Journal, in January 2016 ibrutinib, a specialty drug, cost US$116,600 to $155,400 a year wholesale in the United States. In spite of discounts and medical insurance, the prohibitive price causes some patients to not fill their prescriptions.
A German version of the extract of dossier assessment can be found here.
Image: Michele Angelo Petrone, Wellcome Images