The POWER-tool: a new effective method that helps involving patients in clinical trial design

Research on rare diseases is often difficult to perform mainly because there are only a small number of cases to study, resulting in a vaguely representative statistical analysis of the data. For these conditions, patient samples are not sufficiently big to measure real variations between individuals. The situation is even worse for ultra-rare diseases. Besides, the disease is sometimes so unfamiliar for the scientific community that very few or no outcome measures have been yet identified. Here is where patients come into the picture.

In recent years, researchers and clinicians have realized that patient involvement in research is vital to develop effective treatments. In this context, patients and/or caregivers should be asked about which characteristics concerning their disease they consider important to be measured during the performance of a new clinical trial, because they are the ones that know more about the symptoms and development of their own condition. In order to accelerate and facilitate this process, Gaasterland and cols have developed a tool to involve patients in the determination of outcome measures and the choice of measurement instruments. To do so, researchers collaborated with a group of rare disease patient representatives which had been educated on clinical research. The tool was tested during an ongoing clinical trial and evaluated during a focus group meeting. The POWER-tool (Patient participation in Outcome measure WEighing for Rare diseases) consisted of three steps: 1) Preparation, 2) Consultation and 3) Follow-up. This new tool was designed to provide guidelines for researchers that will help them include the patient’s opinion in the choice of outcome measures during the clinical trial design stage.

The first experiences of the tool, based on existing literature and input from patient experts, were positive. According to the authors, the POWER-tool can be further settled in future trials, before the trial is even performed. In these cases it could be a useful tool for participation, changing the direction from research in patients towards research with patients.


Link to the specific scientific publication:

Gaasterland CMW, et al. (2018) The POWER-tool: Recommendations for involving patient representatives in choosing relevant outcome measures during rare disease clinical trial design. Health Policy.

Patient and doctor