Measuring patient-centered outcomes in neurologic disease. Extending the Q-TWiST method

Current clinical research and outcomes assessment on multiple sclerosis rely on an approach to disability measurement that is heavily influenced by ambulation. Not only is this strategy often insensitive to the clinical changes affected by pharmacotherapeutic or rehabilitative interventions but it also disregards the symptoms that patients seem to consider most enervating. We propose a new method for evaluating clinical interventions in terms of their impact on the symptoms of multiple sclerosis, side effects, parameters of exacerbation, and disease progression, while considering the patient's perspective.

The extended Q-TWiST method yields an estimation of treatment trade-offs in terms of Quality-adjusted Time Without Symptoms and Toxicities (Q-TWiST). An illustration of this method is presented by using a hypothetical clinical trial of two treatments. The trade-offs between the two treatments are highlighted to facilitate treatment decision making by using individual patient importance weights.

We discuss applications to other clinical research and other chronic diseases.