Germany’s IQWiG finds first tumor-agnostic drug provides no added benefit
Germany’s Institute for Quality and Efficiency in Health Care (IQWiG) this week said that Bayer’s Vitrakvi (larotrectinib), approved in the EU last summer, has not been proven to provide an added benefit.
Vitrakvi was the first medicine to be approved in the EU as a treatment for tumors with a specific mutation, regardless of where in the body the tumor originated. But the treatment’s safety and efficacy were evaluated in three single-arm trials, and IQWiG took issue with the lack of comparator arms in the trials.“
In exceptional cases, studies without comparator data may be sufficient for an approval if the aim is to show that the substance has an effect on the tumor. Explicit comparator data are needed, however, if the question is whether such an effect is associated with a patient-relevant benefit,” IQWiG said.
The institute also found that regarding overall survival, the differences between larotrectinib and other treatments observed so far “were not so large in any of the cancer diseases that they could not be caused by systematic bias alone. Regarding patient-relevant outcomes from the categories of morbidity, health-related quality of life and side effects, there were no suitable data or no data differentiated by tumor type either for the new drug or for the comparator therapies, so that it was also not possible to derive an added benefit.”
Deficiencies in the histology-independent analyses presented by Bayer were also noted, as were a lack of prespecified analyses and selective results reporting.
Bayer, however, told Focus via email that it does not share the view of IQWiG and is convinced of the added benefit “due to the exceptionally high effectiveness and good tolerability shown in clinical studies for patients with so-called TRK fusion tumors.”
Bayer also said that IQWiG’s methods “are not yet appropriate for appraising histology independent products, so this recommendation is not surprising.” The company also noted that the approval by the EMA is conditional so “further clinical evidence will be generated after the EMA approval via registries and non-interventional phase IV studies.”
RAPS: First published in Regulatory Focus™ by the Regulatory Affairs Professionals Society, the largest global organization of and for those involved with the regulation of healthcare products. Click here for more information.
Social image: IQWiG logo, Ralf Baumgarten