A Swedish physician specialising in health economics has examined the issue of quality of life in Duchenne muscular dystrophy (DMD), a component that is becoming increasingly important in the light of therapeutic advances in the field, for clinicians, researchers, regulators and payers alike.
- Current instruments for measuring quality of life in DMD are far from being efficient but can still be useful either in the context of patient follow-up or during a clinical trial.
- It is all a question of choice and context, between generic scales on the one hand, and disease-specific scales on the other.
- The author recommends the use of KIDSCREEN in children and the SF-36 in adults, but also the EQ-5D and the HUI.
However, a major methodological research effort remains to be made in this field.
