At Rallying to the Challenge 2015, clinical trial outcomes were put under the microscope.
Clinical trials put great emphasis on measurement scales such as UPDRS (United Parkinson’s Disease Rating Scale). These are questionnaire-based scales and provide a snapshot of a patient’s well-being at any one time. Because of the constantly changing nature of Parkinson’s, even within individuals, it is increasingly accepted that most clinically used measures of outcome in clinical trials give only a partial and non-specific picture of patient well-being. Moreover most existing outcome measures focus on movement components (e.g. stiffness, slowness, tremor), with less emphasis on non-movement symptoms (e.g. fatigue, sleep, pain).
For patients, outcomes that relate to quality of life are more important. Movement symptoms per se influence but do not solely determine quality of life. There is increasing evidence that non-movement symptoms have a particularly strong effect on quality of life for patients.
Variability between individual patients and fluctuations in symptoms over time means that measurements such as UPDRS fails to capture the variability of the condition with time, a critical component of the management of Parkinson’s.
The results of our 2015 Rallying survey formed the basis for the focused discussion at the meeting at Grand Rapids. This meeting looked closely at the variability of individual’s symptoms and how they can inform outcomes in clinical trials, where outcomes can be improved and how technology may be useful in developing new ways of measuring. The emphasis throughout was on improving the patient experience.