25 September 2020 - There are several techniques for estimating health state utility values, each of which presents pros and cons in the context of rare diseases.
Direct approaches (e.g. standard gamble and time trade-off) may be too demanding for patients with rare diseases, since most of them affect young children or cause cognitive impairment.
The alternatives are using “vignettes” that describe hypothetical health states for the general public, which may not reflect the heterogeneous manifestations of rare diseases, or multi-attribute utility instruments (i.e. indirect techniques), such as EQ-5D, which may be less sensitive in capturing the specificities of rare diseases.