Problems in the study implementation often affect patient-reported out-comes

Asciminib is used for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in the chronic phase. In a benefit assessment, the German Institute for Quality and Efficiency in Health Care (IQWiG) has now examined whether asciminib offers benefits over the current standard of care for these patients.

However, the study data provided by the manufacturer reveal a key problem: The patient-reported outcomes (PROs) were only recorded incompletely. In the case of PROs, patients themselves assess how the use of a medical intervention affects, for example, their symptoms or their health-related quality of life. This is why PROs are highly relevant for patients and for assessing the benefits and harms of drugs. Nevertheless, the lack of recognition of PROs compared with other outcomes in some manufacturers’ studies is a recurring problem.

This is also evident from the example of asciminib: in both studies presented by the manufacturer, a baseline PRO score was recorded for only around 50 per cent of patients. However, without such baseline values, it is not possible to conduct meaningful analyses on PROs – meaning that half of the patients therefore remain unconsidered in the analyses. What is particularly incomprehensible is how such a problem could arise in both studies in exactly the same way, and that this was apparently only noticed at a late stage in the course of the studies.

Philip Kranz, Head of the Haemato-Oncology and Infectiology Division in IQWiG’s Drug Assessment Department, draws the following conclusion: “Patient-reported outcomes clearly do not always carry the same weight as clinical parameters in the design and implementation of studies. The perspectives of the study participants must urgently be given greater consideration.”