Table 1.
Methods used to measure and value quality of life in NICE appraisals that included preschool children.
| Appraisal (Year) | Condition | Main Measure(s) Used to Derive Utility Values | Where Measure Was Collected | Who Completed Measure | Who Completed Valuation Tasks |
|---|---|---|---|---|---|
| TA151 (2008) [8] | Diabetes | EQ-5D-3L | Observational study | Adults with the condition | UK patients with diabetes |
| TA300 (2013) [9] | Hepatitis C | EQ-5D-3L | Observational study | Adults with the condition | Non-UK general public (Swedish and Canadian value sets) |
| TA538 (2018) [10] | Neuroblastoma | HUI used to calculate % decrement applied to EQ-5D-3L general population values | Observational study | Adults and older children with the condition. Adult proxy completion for preschool | % decrements: Non-UK general public (Canadian HUI value set) General population norms: UK general public |
| TA566 (2019) [11] | Cochlear implants (update of TA166, 2009) | HUI3 | Observational studies | Unilateral implant populations: adults and older children with the condition. Adult proxy completion for preschool children Bilateral implant population: adults with the condition |
Non-UK general public (HUI 3 Canadian value set) |
| TA588 (2019) [12] | Spinal muscular atrophy | Direct elicitation of utility values from clinical experts | N/A | N/A | N/A |
| TA630 (2020) [13] | NTRK mutation positive cancer | EQ-5D-5L mapped to 3L and PedsQL mapped to EQ-5D-Y (applying adult value set) | Clinical trial | Adults and older children with the condition. Adult proxy completion for preschool children | UK general public (UK EQ-5D-3L adult value set) |
| HST2 (2015) [14] | Mucopolysaccharidosis type IVa | EQ-5D-5L mapped to 3L | Burden of illness survey | Adults with the condition Data collected for older children with the condition were not used in the model |
UK general public (UK EQ-5D-3L adult value set) |
| HST6 (2017) [15] | Hypophosphatasia | EQ-5D-5L mapped to 3L | Vignette study | Clinicians | UK general public (UK EQ-5D-3L adult value set) |
| HST7 (2018) [16] | Adenosine deaminase deficiency/severe combined immunodeficiency | Utility decrement from HUI applied to EQ-5D-3L general population values | Observational study | Adult proxy completion for older children with a similar condition. | Utility decrement: Non-UK general public (HUI 3 Canadian value set) General population norms: UK general public |
| HST8 (2018) [17] | X-linked hypophosphataemia | EQ-5D-5L mapped to 3L | Vignette study | Clinicians | UK general public (UK EQ-5D-3L adult value set) |
| HST11 a (2019) [18] | Retinal dystrophy (RPE65 gene mutations) | Company: EQ-5D-5L mapped to 3L ERG: Disease-specific preference-accompanied measure (Visual Function Questionnaire-25) |
Company: Vignette study ERG: Time trade-off study |
Company: Clinicians ERG: Adults (general public) |
Company: UK general public (UK EQ-5D-3L adult value set) ERG: general public (UK, US, Australia and Canada) |
| HST12 (2019) [19] | Neuronal ceroid lipofuscinosis type 2 | EQ-5D-5L mapped to 3L | Vignette study | Clinicians | UK general public (UK EQ-5D-3L adult value set) |
a The committee stated that the utility values proposed by the committee and ERG were both considered and that ‘true’ utility values were likely somewhere between the two approaches.