Introduction
Methods
Systematic review
Data extraction
Descriptive analyses
Statistical analyses
Results
Systematic review
Descriptive analyses
Distribution of sample means and medians
Distribution of samples by health condition and valuation method
General health: 501
CHU9D (27.4) EQ-5D-Y VAS (24.4) EQ-5D-Y (10.6) |
Chapter 1 Infectious and parasitic diseases: 195
VAS (24.1) EQ-5D (15.9) TTO (15.4) |
Chapter 2 Cancer: 442
HUI2 (52.0) HUI3 (37.1) VAS (2.94) |
Chapter 3 Diseases of blood: 85 SG (43.5) EQ-5D (24.7) VAS (12.9) | Chapter 4 Endocrine, nutritional and metabolic disorders: 411 SG (14.1) HUI3 (12.9) EQ-5D-Y VAS (12.4) | Chapter 5 Mental and behavioural disorders: 698 EQ-5D (20.2) HUI3 (18.9) EQ-5D VAS (16.6) |
Chapter 6 Nervous system disorders: 114 HUI3 (43.0) SF-6D (9.65) EQ-5D (7.89) | Chapter 7 Diseases of the eye: 61 TTO (42.6) Chained Gamble (42.6) HUI3 (14.8) | Chapter 8 Diseases of the ear: 158 HUI3 (58.2) VAS (13.3) HUI2 (6.96) |
Chapter 9 Circulatory system disorders: 16 VAS (31.3) HUI3 (31.3) HUI2 (18.8) | Chapter 10 Respiratory system disorders: 192 PAHOM (35.8) EQ-5D VAS (16.8) EQ-5D (12.1) | Chapter 11 Digestive system disorders: 60 16D/17D (20.0) EQ-5D-Y VAS (18.3) CHU9D (16.7) |
Chapter 12 Diseases of the skin: 26 VAS (38.5) TTO (26.9) EQ-5D (15.4) | Chapter 13 Musculoskeletal system disorders: 114 EQ-5D (37.7) EQ-5D VAS (17.5) VAS (14.9) | Chapter 14 Genitourinary system disorders: 56 VAS (30.4) TTO (26.8) HUI3 (16.1) |
Chapter 16 Conditions originating in perinatal period: 210 HUI3 (38.1) SG (33.3) HUI2 (15.7) | Chapter 17 Congenital malformations: 155 HUI3 (33.6) VAS (23.2) TTO (18.1) | Chapter 19 Injury, poisoning and other consequences of external causes: 297 QWB (40.7) EQ-5D (29.0) EQ-5D VAS (13.1) |
Chapter 21 Contact with health services: 80 HUI3 (48.8) HUI2 (36.3) 15D/16D/17D (8.75) | Combined chronic diseases: 103 HUI3 (40.8) EQ-5D-Y VAS (23.3) EQ-5D VAS (12.6) |
Distribution of samples by methodological factors, age and geographical setting
Number of samples by respondent type | |||||
Self-assessment: 1498 (37.7)
| |||||
Self-assessment by children: 1498 | |||||
Proxy assessment—joint assessment by children and proxy: 456 (11.5) | |||||
Assessment by children and parents: 391 | Assessment by children and caregivers: 65 | ||||
Proxy assessment: 2020 (50.8) | |||||
Proxy assessment by parents: 1091 | Proxy assessment by caregivers: 435 | Proxy assessment by physicians: 256 | |||
Proxy assessment by physicians and caregivers: 47 | Proxy assessment by nurses: 76 | Proxy assessment by the general public: 24 | |||
Proxy assessment by parents from the general public: 77 | Proxy assessment by adult patients: 7 | Proxy assessment by parents, adult patients and the general public: 7 | |||
Number of samples by administration mode | |||||
Self-administration by respondents: 2464 (62.0) | |||||
Non-postal survey: 1276 | Postal survey: 864 | Online survey: 319 | |||
Delphi process: 5 | |||||
Interview-administration: 1478 (37.2) | |||||
Face-to-face interview: 1198 | Telephone interview: 265 | Face-to-face or telephone: 15 | |||
Mode of administration not specified: 32 (0.8) | |||||
Number of samples by age of children in sample | |||||
Pre-adolescent children (age < 12 years) in sample: 2086 (52.5) | |||||
Minimum age of 0: 238 | Minimum age of 2: 247 | Minimum age of 5: 560 | |||
Minimum age of 8: 1041 | |||||
No pre-adolescent children in sample: 1315 (33.1) | |||||
Minimum age of 12: 776 | Minimum age of 15: 498 | Minimum age of 18: 41 | |||
Age unspecified: 573 (14.4) | |||||
Number of samples by geography | |||||
High-income countries in Europea: 1522 (38.3) | |||||
Austria: 12 | Belgium: 5 | Denmark: 10 | Finland: 114 | France: 4 | Germany: 49 |
Hungary: 3 | Italy: 24 | Netherlands: 445 | Norway: 2 | Portugal: 2 | Spain: 34 |
Sweden: 184 | UK: 634 | ||||
North America: 1644 (41.4) | |||||
Canada: 674 | US: 970 | ||||
Other high-income countries: 473 (11.9) | |||||
Australia:263 | New Zealand: 14 | Singapore: 44 | South Korea: 11 | Developed countries: 141 | |
Central and South America: 79 (2.0) | |||||
Argentina: 2 | Brazil: 5 | Colombia: 22 | Honduras: 8 | Uruguay: 12 | South America: 30 |
Africa: 89 (2.2) | |||||
Kenya: 30 | Sierra Leon: 4 | South Africa: 47 | Uganda: 4 | Zimbabwe: 4 | |
Other low- and middle-income countriesa: 156 (3.9) | |||||
Bulgaria: 3 | China: 36 | Cuba: 16 | India: 2 | Iran: 14 | Russia: 8 |
Thailand: 71 | Turkey: 6 | ||||
Other: 11 (0.3) | |||||
Developed and developing: 2 | Not specified: 9 |
MAUIs and tariff application
MAUI | Target age range | Tariff valuation population and method | Number of samples (%) |
---|---|---|---|
Childhood-specific MAUIs | |||
16D | 12–15 | Finnish schoolchildren aged 12–16 (n = 213) using VAS [18] | 68 (93.2) |
No information or reference on tariff | 5 (6.8) | ||
17D | 8–11 (Proxy for < 8) | Finnish parents of children aged 8–11 (n = 115) using VAS [19] | 39 (100.0) |
AQoL-6D | Adolescents | 1. Australian adolescents (n = 2790) using TTO [69] | 36 (72.0) |
2. Australian general adult public (n = 411) using TTO [70] | 14 (28.0) | ||
CHU9D | 7–17 | 1. Australian adolescents aged 11–17 (n = 590) using best-worst scale discrete-choice experiment [71] | 91 (39.4) |
2. Australian adolescents aged 11–17 (n = 1982) using best-worst scale discrete-choice experiment [72] | 16 (6.9) | ||
3. UK general adult public (n = 300) using SG [73] | 122 (52.8) | ||
No information or reference on tariff | 2 (0.9) | ||
EQ-5D-Y | 8–15 (Proxy for 4–7) | 1. Canadian elementary schoolchildren aged 10–11 (n = 4485) using VAS [74] | 1 (0.9) |
2. UK general adult public (n = 3395) using TTO [75] | 25 (23.1) | ||
3. Dutch general adult public (n = 309) using TTO [76] | 9 (8.3) | ||
4. Dutch general adult public (n = 303) using TTO [77] | 5 (4.6) | ||
5. Australian general adult public (n = 417) using TTO [78] | 14 (13.0) | ||
6. French general adult public (n = 452) using TTO [79] | 3 (2.8) | ||
7. Swedish general adult public (n = 45,000) using TTO and VAS [80] | 4 (3.7) | ||
8. General adult public from Finland, Germany, Netherlands, Spain, Sweden and UK (n = 6870) using VAS [81] | 2 (1.9) | ||
9. US general adult public using TTO [82] | 23 (21.3) | ||
No information or reference on tariff | 22 (20.4) | ||
PAHOM | 7–13 | 1. Asthmatic children aged 6–12 (n = 261) using SG [83] | 60 (87.0) |
2. US general adult public (n = 94) using VAS [44] | 4 (5.8) | ||
3. US general adult public (n = 101) using SG [44] | 4 (5.8) | ||
No information or reference on tariff | 1 (1.4) | ||
CH-6D | 16–18 | No information or reference on tariff | 3 (100.0) |
MAUIs compatible with childhood and adult populations | |||
HUI2 | ≥ 5 (Proxy for 5–8) | 1. Canadian parents of schoolchildren (n = 194) using VAS and SG [84] | 357 (74.1) |
2. Canadian parents of normal children (n = 300) using VAS and SG [85] | 22 (4.6) | ||
3. Singaporean parents of paediatric cancer patients (n = 59) using VAS and SG [86] | 10 (2.1) | ||
4. Singaporean general public parents of children (n = 194) using VAS and SG [86] | 10 (2.1) | ||
5. UK general adult public (n = 201) using VAS and SG [87] | 58 (12.0) | ||
No information or reference on tariff | 25 (5.2) | ||
HUI3 | ≥ 5 (Proxy for 5–8) | Canadian general adult public (n = 504) using VAS and SG [88] | 753 (91.6) |
No information or reference on tariff | 69 (8.4) | ||
N/A | 1. Canadian parents of schoolchildren (n = 194) using VAS and SG [84] | 7 (87.5) | |
2. Canadian general adult public (n = 504) using VAS and SG [88] | 1 (12.5) | ||
QWB | N/A | US general public (n = 800) using VAS [89] | 224 (100.0) |
ABC-UI | N/A (Proxy completed) | UK general adult public (n = 349) using TTO [90] | 1 (100.0) |
Statistical analyses
Linear trend in utility studies and samples and PEDE CUAs
Association between PEDE CUAs and utility studies and samples
Periodic change in composition of utility studies and samples
Category | Number (%) of studies/samples | Test of proportion | ||||
---|---|---|---|---|---|---|
1990–June 2017 | 1990–2008 | 2009–June 2017 | Direction of change | P value | ||
Study design | Patient case seriesa | 47 (14.6) | 24 (21.4) | 25 (11.2) |
_
|
0.013
|
Cross-sectional surveyb | 171 (51.0) | 57 (50.9) | 114 (51.1) | + | 0.972 | |
Longitudinal studies and RCTs without CUAc | 47 (14.0) | 14 (12.5) | 33 (14.8) | + | 0.568 | |
Longitudinal studies and RCTs with CUA and decision models with primary utility assessmentd | 68 (20.3) | 17 (15.2) | 51 (22.9) | + | 0.099 | |
Total | 335 | 112 | 223 | |||
Health condition | General health | 501 (12.6) | 41 (2.9) | 460 (18.0) | + | < 0.001 |
ICD-10 chapter 1: Infectious and parasitic diseases | 195 (4.9) | 143 (10.0) | 52 (2.0) |
_
| < 0.001 | |
ICD-10 chapter 2: Cancer | 442 (11.1) | 329 (23.1) | 113 (4.4) |
_
| < 0.001 | |
ICD-10 chapter 3: Diseases of the blood and immune system | 85 (2.1) | 49 (3.4) | 36 (1.4) | _ | < 0.001 | |
ICD-10 chapter 4: Endocrine, nutritional and metabolic disorders | 411 (10.3) | 77 (5.4) | 334 (13.1) | + | < 0.001 | |
ICD-10 chapter 5: Mental and behavioural disorders | 698 (17.6) | 140 (9.8) | 558 (21.9) | + | < 0.001 | |
ICD-10 chapter 6: Nervous system disorders | 114 (2.9) | 18 (1.3) | 96 (3.8) | + | < 0.001 | |
ICD-10 chapter 7: Diseases of the eye | 61 (1.5) | 51 (3.6) | 10 (0.4) | _ | < 0.001 | |
ICD-10 chapter 8: Diseases of the ear | 158 (4.0) | 70 (4.9) | 88 (3.4) | _ |
0.020
| |
ICD-10 chapter 9: Circulatory system disorders | 16 (0.4) | 10 (0.7) | 6 (0.2) | _ |
0.014
| |
ICD-10 chapter 10: Respiratory system disorders | 192 (4.8) | 37 (2.6) | 155 (6.1) | + | < 0.001 | |
ICD-10 chapter 11: Digestive system disorders | 60 (1.5) | 5 (0.4) | 55 (2.2) | + | < 0.001 | |
ICD-10 chapter 12: Diseases of the skin | 26 (0.7) | 19 (1.3) | 7 (0.3) | _ | < 0.001 | |
ICD-10 chapter 13: Musculoskeletal system disorders | 114 (2.9) | 42 (3.0) | 72 (2.8) | _ | 0.717 | |
ICD-10 chapter 14: Genitourinary system disorders | 56 (1.4) | 7 (0.5) | 49 (1.9) | + | < 0.001 | |
ICD-10 chapter 16: Conditions originating in the perinatal period | 210 (5.3) | 121 (8.5) | 89 (3.5) | _ | < 0.001 | |
ICD-10 chapter 17: Congenital malformations | 155 (3.9) | 16 (1.1) | 139 (5.4) | + | < 0.001 | |
ICD-10 chapter 19: Injury, poisoning and other consequences of external causes | 297 (7.5) | 211 (14.8) | 86 (3.4) | _ | < 0.001 | |
ICD-10 chapter 21: Contact with health services | 80 (2.0) | 10 (0.7) | 70 (2.7) | + | < 0.001 | |
Combined chronic diseases | 103 (2.6) | 27 (1.9) | 76 (3.0) | + |
0.037
| |
Total | 3974 | 1423 | 2551 | |||
Valuation method | Visual analogue scalese | 832 (20.9) | 230 (16.2) | 602 (23.6) | + | < 0.001 |
Trade-off-based direct valuation methodsf | 541 (13.6) | 337 (23.7) | 204 (8.0) | _ | < 0.001 | |
Adult-specific MAUIsg | 476 (12.0) | 130 (9.1) | 346 (13.6) | + | < 0.001 | |
MAUIs compatible with childhood and adult populationsh | 1537 (38.7) | 660 (46.4) | 877 (34.4) | _ | < 0.001 | |
Childhood-specific MAUIsi | 573 (14.4) | 63 (4.4) | 510 (20.0) | + | < 0.001 | |
Utility from non-preference-based methodsj | 15 (0.4) | 3 (0.2) | 12 (0.5) | + | 0.147 | |
Total | 3974 | 1423 | 2551 | |||
Respondent type | Self-assessment by children | 1498 (37.7) | 318 (22.3) | 1180 (46.3) | + | < 0.001 |
Proxy assessment (includes joint assessment by proxies and children) | 2476 (62.3) | 1105 (77.7) | 1371 (53.7) | _ | ||
Total | 3974 | 1423 | 2551 | |||
Administration mode | Self-administered surveys | 2464 (62.0) | 609 (42.8) | 1855 (72.7) | + | < 0.001 |
Interview-administered surveys | 1510 (38.0) | 814 (57.2) | 696 (27.3) | _ | ||
Total | 3974 | 1423 | 2551 | |||
Valuation of hypothetical state | Experienced health state | 3247 (81.7) | 1009 (70.9) | 2238 (87.7) | + | < 0.001 |
Hypothetical health state | 727 (18.3) | 414 (29.1) | 313 (12.3) | _ | ||
Total | 3974 | 1423 | 2551 | |||
Age of target population | Sample contains pre-adolescents (mean or median age below 12 or minimum age below 12 if mean/median age not reported) | 2086 (52.5) | 708 (49.8) | 1378 (54.0) | + |
0.011
|
Sample does not contain pre-adolescents | 1315 (33.1) | 445 (31.3) | 870 (34.1) | + | 0.072 | |
Sample age not specified | 573 (14.4) | 270 (19.0) | 303 (11.9) | _ | < 0.001 | |
Total | 3974 | 1423 | 2551 |
Influence of HTA guidance on primary samples
Category | Valuation method | Number of samples (%) | Test of proportion | ||
---|---|---|---|---|---|
1990–2012 | 2013–June 2017 | Direction of change | P value | ||
Sample contains pre-adolescentsa | |||||
Reference for adult population | EQ-5D | 23 (11.1) | 48 (32.4) | + | < 0.001 |
Reference for childhood population | EQ-5D-Y | 8 (3.9) | 25 (16.9) | + | < 0.001 |
MAUIs compatible with childhood populations | HUI2 | 50 (24.2) | 0 (0.0) | ||
HUI3 | 106 (51.2) | 0 (0.0) | |||
Total | 156 (75.4) | 0 (0.0) | − | < 0.001 | |
Childhood-specific MAUIs | CHU9D | 0 (0.0) | 51 (34.5) | + | < 0.001 |
VAS | 8 (3.9) | 24 (16.2) | + | < 0.001 | |
Trade-off direct valuation methods | 12 (5.8) | 0 (0.0) | − |
0.003
| |
Sub-total (% of total) | 207 (45.0) | 148 (85.1) | + | < 0.001 | |
Sample does not contain pre-adolescents | |||||
Reference for adult population | EQ-5D | 12 (6.6) | 9 (34.6) | + | < 0.001 |
Reference for childhood population | EQ-5D-Y | 15 (8.3) | 4 (15.4) | + | 0.241 |
Adult-specific MAUIs | SF-6D | 0 (0.0) | 2 (7.7) | + | < 0.001 |
MAUIs compatible with childhood populations | HUI2 | 11 (6.1) | 2 (7.7) | ||
HUI3 | 58 (32.0) | 6 (23.1) | |||
Total | 69 (38.1) | 8 (30.8) | − | 0.471 | |
VAS | 38 (21.0) | 3 (11.5) | − | 0.256 | |
Trade-off direct valuation methods | 47 (26.0) | 0 (0.0) | − |
0.003
| |
Sub-total (% of total) | 181 (39.3) | 26 (14.9) | − | < 0.001 | |
Sample did not report age of target population | |||||
Sub-total (% of total) | 72 (15.7) | 0 (0.0) | − | < 0.001 | |
Total | 460 | 174 |
Associations between methodological factors
Visual analogue scales | Trade-off-based direct valuation methods | Adult-specific MAUIs | MAUIs compatible with childhood and adult populations | Childhood-specific MAUIs | Utility from non-preference-based methods | Total | Chi-square test statistic (P value) | |
---|---|---|---|---|---|---|---|---|
(1) Age of sample | ||||||||
Sample contains pre-adolescents* | 486**
(471.7)
| 152 (231.2) | 250 (284.0) |
751
(740.3)
|
443
(349.6)
| 4 (9.2) | 2086 |
154.4
(< 0.001) |
Sample does not contain pre-adolescents | 283 (297.3) |
225
(145.8)
|
213
(179.0)
| 456 (466.7) | 127 (220.4) |
11
(5.8)
| 1315 | 5 degrees of freedom |
Total | 769 | 377 | 463 | 1207 | 570 | 15 | 3401 | |
(2) Respondent type | ||||||||
Self-assessment by children |
353
(313.6)
| 140 (203.9) |
220
(179.4)
| 337 (579.4) |
439
(216.0)
|
9
(5.7)
| 1498 |
590.3
(< 0.001) |
Proxy assessment*** | 479 (518.4) |
401
(337.1)
| 256 (296.6) |
1200
(957.6)
| 134 (357.0) | 6 (9.3) | 2476 | 5 degrees of freedom |
Total | 832 | 541 | 476 | 1537 | 573 | 15 | 3974 | |
(3) Administration mode | ||||||||
Self-administered surveys |
580
(515.9)
| 80 (335.4) |
321
(295.1)
|
1036
(953.0)
|
438
(355.3)
| 9 (9.3) | 2464 |
608.6
(< 0.001) |
Interview-administered surveys | 252 (316.1) |
461
(205.6)
| 155 (180.9) | 501 (584.0) | 135 (217.7) |
6
(5.7)
| 1510 | 5 degrees of freedom |
Total | 832 | 541 | 476 | 1537 | 573 | 15 | 3974 | |
(4) Valuation of hypothetical states | ||||||||
Experienced health states | 582 (679.8) | 152 (442.0) |
450
(388.9)
|
1513
(1255.8)
|
535
(468.2)
|
15
(12.3)
| 3247 | > 1500.0 (< 0.001) |
Hypothetical health states |
250
(152.2)
|
389
(99.0)
| 26 (87.1) | 24 (281.2) | 38 (104.8) | 0 (2.7) | 727 | 5 degrees of freedom |
Total | 832 | 541 | 476 | 1537 | 573 | 15 | 3974 |