Abstract
Rare diseases can have a considerable impact on patients’ health-related quality of life (HRQoL). The National Institute for Health and Care Excellence’s (NICE) Highly Specialised Technologies (HST) process evaluates treatments indicated for very rare diseases. For interventions that have the potential to restore normal physiology in rare disease sufferers, capturing long-term impact on HRQoL in economic evaluations may be particularly challenging, due to sparsity of data. The aim of this study is to explore how long-term HRQoL benefit was modelled in NICE HSTs of treatments that could restore patient health to near that of the general population.
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