Measuring patient-centered outcomes in neurologic disease. Extending the Q-TWiST method
C. E. Schwartz, B. F. Cole and R. D. Gelber
Frontier Science and Technology Research Foundation Inc, Brookline, Mass., USA.
BACKGROUND: 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. METHODS: 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. CONCLUSION: We discuss applications to other
clinical research and other chronic diseases.