Measurement strategies in progressive disease

Cancer patients with progressive disease are confronted with the outlook of a limited survival, along with an increasing symptom burden. Evaluating both survival and health-related quality of life (HRQoL) outcomes allows investigators to determine the net clinical benefit of a treatment strategy. Nevertheless, missing data is a major problem in patients with progressive disease, hampering the statistical analysis and thus the value of the information obtained. To improve the current standards, we aim to develop guidelines on the design, collection and analysis of HRQoL data in cancer clinical trials in the progressive disease setting.

The research protocol needed for the semi-structured interviews with patients, their proxies and healthcare professionals is being approved in the Netherlands. Interviews with patients, proxies and health care professionals with start. Other countries will follow as soon as the ethical process is completed. Moreover, two literature reviews are currently being performed.

Current status:

Recruitment of patients, proxies and health care professionals.

Start with the interviews in all countries, finishing the literature reviews, start with the data analyses, and write the manuscripts and guidelines.

Patients with cancer may face tumor growth or spread of their disease throughout their body, thereby limiting their chances of survival. At the same time, their symptoms typically increase, thereby limiting their overall quality of life. When developing new treatments, the impact of this treatment on both the survival and the functioning of the patient needs to be evaluated. For this, data on the functioning and well-being of patients is collected with questionnaires which have to be completed by patients. However, if patients have a poor health status, they typically do no longer complete these questionnaires, limiting the amount of data that is available for analysis. To ensure that sufficient data is available that is necessary to draw reliable conclusions on the impact of the treatment under investigation, new strategies need to be developed to better collect and assess health-related outcomes in cancer patients with progressive disease.

This project aims to develop guidelines on the design, collection and analysis of health-related outcomes in clinical studies for patients with progressive disease.