Patients with symptomatic anaemia associated with chronic kidney disease who are on chronic maintenance dialysis can now also receive vadadustat instead of an erythropoiesis-stimulating agent such as darbepoetin. The German Institute for Quality and Efficiency in Health Care (IQWiG) has now examined in an early benefit assessment whether the new drug offers an added benefit for these patients in comparison with the appropriate comparator therapy (ACT). The (German-language) report was published in September 2024, an English translation in November 2024.
No overall advantage or disadvantage of vadadustat can be established based on the available results of the two studies cited by the manufacturer in its dossier; individual positive and negative effects can be seen in side effects. An added benefit of vadadustat in comparison with the ACT is therefore not proven.
Studies with virtually no informative benefit outcomes
Anaemia is often accompanied by symptoms such as fatigue, dizziness, headache and physical weakness. The resulting reduction in physical performance also has an impact on patients’ quality of life. However, informative outcomes on symptoms and outcomes in the health-related quality of life category were not recorded in the studies. It therefore remains unknown what effect the consistently lower haemoglobin levels during treatment with vadadustat have on the patients’ wellbeing.
In smaller studies, the results of which were not included in the benefit assessment, the manufacturer had previously asked patients about their wellbeing, thus demonstrating that patient-reported outcomes can and should be recorded during treatment with vadadustat. Daniela Preukschat, who heads the Chronic Diseases Division in IQWiG’s Drug Assessment Department, says: “The study data used for the benefit assessment say nothing about how patients feel during and after treatment. It would have been perfectly feasible to record patient-relevant outcomes. The opportunity was missed to collect important data for patients and treatment decisions.”
G-BA decides on the extent of added benefit
The dossier assessment is part of the early benefit assessment according to the Act on the Reform of the Market for Medicinal Products (AMNOG) supervised by the G-BA. After publication of the dossier assessment, the G-BA conducts a commenting procedure and makes a final decision on the extent of the added benefit.