Table 1 Outcomes of interest

Prevalence (any timeframe)

Generic quality of life scores measured via the following tools:

EQ-5D

HUI-1, HUI-2/HUI-3

SF6/SF6D/SF12, SF36

MOS, RAND12 or RAND 36

PedsQL

Incidence (any timeframe)

Utility values derived from generic preference-based instruments listed above

Disease natural history—disease progression

Disease-specific quality of life scores measures (any)

Mortality

Mapping studies, from disease-specific to generic preference-based measures or between different generic preference-based measures

Disutilities associated with adverse events e.g. pruritus

Caregiver utility values

Descriptive summary of health states, and/or change in health status/QoL results