Appraisals by Health Technology Assessment Agencies of Economic Evaluations Submitted as Part of Reimbursement Dossiers for Oncology Treatments: Evidence from Canada, the UK, and Australia
Abstract
:1. Introduction
2. Materials and Methods
2.1. Study Data
2.2. Data Abstraction
2.3. Data Analyses
3. Results
3.1. Number of HTA Submissions Reviewed by CADTH between 2019–2020 Matched with Corresponding HTAs from NICE and PBAC
3.2. Manufacturer Economic Submissions’ Characteristics
3.3. HTA Agency Reporting on Economic Model Characteristics Submitted by Manufacturers
3.4. HTA Agency Reporting on Methods Used to Extrapolate Survival Data in Manufacturers’ Cost-Effectiveness Models
3.5. HTA Agency Reporting on Methodological Criticisms of Manufacturer Economic Submissions
3.6. HTA Agency Reporting on Economic Results, HTA Economic Re-Analyses and Funding Recommendations
4. Discussion
4.1. Summary of Findings
4.2. Previous Studies
4.3. Limitations
4.4. Future Directions
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Institutional Review Board Statement
Informed Consent Statement
Data Availability Statement
Conflicts of Interest
References
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Characteristic | n | % |
---|---|---|
HTA agency (n = 108) | ||
pCODR | 36 | 33% |
NICE | 36 | 33% |
PBAC | 36 | 33% |
Data source type (n = 108) | ||
Ph3 | 79 | 67% |
Ph2 (single arm) | 16 | 15% |
Mix of Ph3 and Ph2 | 4 | 3% |
RWE | 0 | 0% |
Mix of Ph2 and RWE | 5 | 6% |
Mix of Ph3 and RWE | 4 | 7% |
Ph4 | 0 | 1% |
Type of cancer studied (n = 108) | ||
Leukemia | 15 | 14% |
Breast | 12 | 11% |
Lung | 27 | 25% |
Genitourinary | 9 | 8% |
Gastrointestinal | 12 | 11% |
Lymphoma | 6 | 6% |
Skin and melanoma | 12 | 11% |
Other | 3 | 3% |
Myeloma | 3 | 3% |
Gynecology | 6 | 6% |
Head and neck | 3 | 3% |
Neurological | 0 | 0% |
Cancer stage (n = 108) | ||
Early/stage I | 12 | 11% |
Stage II/III | 27 | 25% |
Stage IV/metastatic | 69 | 64% |
Reported Characteristic | Number of Studies n (%) | p-Value (χ2) | ||
---|---|---|---|---|
CADTH | NICE | PBAC | ||
Type of analysis | ||||
CUA | 36 (100%) | 36 (100%) | 30 (83%) | 0.013 |
CEA | 0 (0%) | 0 (0%) | 0 (0%) | |
Other (e.g., CMA) | 0 (0%) | 0 (0%) | 6 (17%) | |
QALYs reported (Y/N) | ||||
Yes | 34 (94%) | 36 (100%) | 30 (83%) | 0.023 |
No | 2 (6%) | 0 (0%) | 6 (17%) | |
Utility value method | ||||
EQ5D | 15 (42%) | 33 (92%) | 18 (50%) | 0.001 |
SF36 | 0 (0%) | 0 (0%) | 0 (0%) | |
HUI | 0 (0%) | 0 (0%) | 0 (0%) | |
Other | 1 (3%) | 1 (3%) | 4 (11%) | |
Not reported | 20 (56%) | 2 (6%) | 14 (39%) | |
Model structure | ||||
Partitioned survival | 25 (69%) | 25 (69%) | 24 (67%) | 0.112 |
Markov | 11 (31%) | 10 (28%) | 6 (17%) | |
Not reported | 0 (0%) | 0 (0%) | 6 (17%) | |
Decision tree | 0 (0%) | 0 (0%) | 0 (0%) | |
Combination (decision tree + Markov) | 0 (0%) | 1 (3%) | 0 (0%) | |
Other | 0 (0%) | 0 (0%) | 0 (0%) | |
Number of modeled health states | ||||
Three | 24 (67%) | 29 (81%) | 21 (58%) | 0.516 |
Four | 2 (6%) | 3 (8%) | 4 (11%) | |
Five | 4 (11%) | 2 (6%) | 1 (3%) | |
Six | 1 (3%) | 0 (0%) | 3 (8%) | |
Seven or more | 0 (0%) | 2 (6%) | 0 (0%) | |
Not reported | 5 (14%) | 0 (0%) | 7 (19%) | |
Time horizon (submitted by manufacturer) | ||||
1–5 years | 4 (11%) | 0 (0%) | 3 (8%) | <0.001 |
6–10 years | 14 (39%) | 4 (11%) | 16 (44%) | |
11–20 years | 7 (19%) | 10 (28%) | 3 (8%) | |
21–30 years | 3 (8%) | 7 (19%) | 3 (8%) | |
31–40 years | 2 (6%) | 6 (17%) | 3 (8%) | |
40+ years | 6 (17%) | 8 (22%) | 1 (3%) | |
Not reported | 0 (0%) | 1 (3%) | 7 (19%) | |
Indirect treatment comparison (Y/N) | ||||
Yes | 20 (56%) | 24 (67%) | 20 (56%) | 0.541 |
No | 16 (44%) | 12 (33%) | 16 (44%) | |
Equity issues reported | ||||
Yes | 0 (0%) | 15 (42%) | 0 (0%) | <0.001 |
No | 36 (100%) | 21 (58%) | 36 (100%) | |
Handling of uncertainty | ||||
Deterministic sensitivity analysis | 12 (33%) | 33 (92%) | 9 (25%) | <0.001 |
Probabilistic sensitivity analysis | 11 (31%) | 36 (100%) | 4 (11%) | <0.001 |
Scenario analysis | 13 (36%) | 36 (100%) | 27 (75%) | <0.001 |
Validation (Y/N) | ||||
Yes | 2 (6%) | 35 (97%) | 0 (0%) | <0.001 |
No | 34 (94%) | 1 (3%) | 36 (100%) | |
Reimbursement recommendation | ||||
Reimburse | 28 (78%) | 34 (94%) | 19 (53%) | <0.001 |
Reported Characteristic | Number of Studies n (%) | p-Value (χ2) | ||
---|---|---|---|---|
CADTH | NICE | PBAC | ||
Parametric approach | ||||
Yes | 20 (56%) | 36 (100%) | 28 (78%) | <0.001 |
No | 16 (44%) | 0 (0%) | 8 (22%) | |
Standard parametric distributions tested | N = 20 | N = 36 | N = 28 | |
Yes | 17 (85%) | 36 (100%) | 21 (75%) | 0.008 |
No | 3 (15%) | 0 (0%) | 7 (25%) | |
Curve fitting assessment | N = 20 | N = 36 | N = 28 | |
AIC | 1 (5%) | 2 (6%) | 1 (4%) | <0.001 |
BIC | 1 (5%) | 0 (0%) | 0 (0%) | |
Both AIC and BIC | 6 (30%) | 30 (83%) | 8 (29%) | |
Other | 0 (0%) | 2 (6%) | 1 (4%) | |
Not reported | 28 (60%) | 2 (6%) | 26 (64%) | |
PH assumption tested (if appropriate) | N = 20 | N = 36 | N = 28 | |
Yes | 2 (10%) | 32 (89%) | 9 (32%) | <0.001 |
No | 18 (90%) | 4 (11%) | 19 (68%) | |
Fitted parametric curves | N = 20 | N = 36 | N = 28 | |
Jointly fitted models | 1 (5%) | 20 (56%) | 10 (36%) | <0.001 |
Separately fitted models | 0 (0%) | 11 (31%) | 4 (14%) | |
Not reported | 19 (95%) | 5 (14%) | 14 (50%) | |
Validation of extrapolations | ||||
Yes | 1 (3%) | 35 (97%) | 6 (17%) | <0.001 |
No | 35 (97%) | 1 (3%) | 30 (83%) | |
Scenario analyses of treatment effect | ||||
Yes | 12 (33%) | 19 (53%) | 11 (31%) | 0.109 |
No | 24 (67%) | 17 (47%) | 25 (69%) | |
Use/source of external data justified | ||||
Yes | 1 (3%) | 20 (56%) | 4 (11%) | <0.001 |
No | 35 (97%) | 16 (44%) | 32 (89%) | |
Curves fitted to tail of Kaplan–Meier curves only | ||||
Yes | 1 (3%) | 4 (11%) | 3 (8%) | 0.389 |
No | 35 (97%) | 32 (89%) | 33 (92%) | |
Alternative curve-fitting approaches examined | ||||
Yes | 3 (8%) | 9 (25%) | 1 (3%) | 0.011 |
No | 33 (92%) | 27 (75%) | 35 (97%) |
Selected Parametric Curve Reported | Treatment | Comparator | ||||||||||
---|---|---|---|---|---|---|---|---|---|---|---|---|
CADTH (n = 20) | NICE (n = 36) | PBAC (n = 28) | CADTH (n = 20) | NICE (n = 36) | PBAC (n = 28) | |||||||
PFS | OS | PFS | OS | PFS | OS | PFS | OS | PFS | OS | PFS | OS | |
Weibull | 5% | 10% | 22% | 17% | 11% | 14% | 0% | 5% | 22% | 14% | 11% | 14% |
Exponential | 0% | 5% | 8% | 25% | 25% | 32% | 0% | 5% | 8% | 25% | 25% | 25% |
Log-logistic | 0% | 5% | 17% | 19% | 7% | 11% | 0% | 0% | 17% | 19% | 11% | 14% |
Log-normal | 15% | 5% | 19% | 17% | 32% | 14% | 10% | 5% | 17% | 17% | 25% | 18% |
Gamma | 0% | 0% | 0% | 0% | 0% | 0% | 0% | 0% | 3% | 0% | 0% | 0% |
Generalized gamma | 0% | 0% | 14% | 6% | 11% | 4% | 0% | 5% | 11% | 3% | 11% | 4% |
Gompertz | 5% | 0% | 8% | 6% | 0% | 7% | 0% | 0% | 8% | 6% | 0% | 7% |
Other | 0% | 0% | 0% | 3% | 0% | 0% | 0% | 0% | 0% | 3% | 0% | 0% |
Not reported | 75% | 75% | 11% | 8% | 14% | 18% | 90% | 80% | 14% | 14% | 18% | 18% |
HTA Agency | Incremental QALYs | |||||
---|---|---|---|---|---|---|
Manufacturer: Base Case | Range | Agency Re-Analysis: Base Case | Range | Average Change | ||
CADTH (n = 32) | 1.30 | 0.13 to 4.34 | CADTH (n = 28) | 0.78 | 0.08 to 2.25 | −60.3% |
NICE (n = 21) | 1.17 | 0.07 to 3.44 | NICE (n = 15) | 0.68 | 0.07 to 2.75 | −58.5% |
PBAC (n = 18) | 1.52 | 0.13 to 6.84 | N/A | N/A | N/A | N/A |
HTA Agency | ICER | |||||
Manufacturer: Base Case | Range | Agency Re-Analysis | Range | Average Change | ||
CADTH (n = 32) | $109,581 | $12,242 to $388,172 | CADTH (n = 32) | $200,923 | $41,414 to $983,977 | 183.4% |
NICE (n = 27) | $65,778 | $6631 to $137,200 | NICE (n = 26) | $112,891 | $23,744 to $229,381 | 171.6% |
PBAC (n = 23) | $48,665 | $18,910 to $129,217 | N/A | N/A | N/A | N/A |
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Ball, G.; Levine, M.A.H.; Thabane, L.; Tarride, J.-E. Appraisals by Health Technology Assessment Agencies of Economic Evaluations Submitted as Part of Reimbursement Dossiers for Oncology Treatments: Evidence from Canada, the UK, and Australia. Curr. Oncol. 2022, 29, 7624-7636. https://doi.org/10.3390/curroncol29100602
Ball G, Levine MAH, Thabane L, Tarride J-E. Appraisals by Health Technology Assessment Agencies of Economic Evaluations Submitted as Part of Reimbursement Dossiers for Oncology Treatments: Evidence from Canada, the UK, and Australia. Current Oncology. 2022; 29(10):7624-7636. https://doi.org/10.3390/curroncol29100602
Chicago/Turabian StyleBall, Graeme, Mitchell A. H. Levine, Lehana Thabane, and Jean-Eric Tarride. 2022. "Appraisals by Health Technology Assessment Agencies of Economic Evaluations Submitted as Part of Reimbursement Dossiers for Oncology Treatments: Evidence from Canada, the UK, and Australia" Current Oncology 29, no. 10: 7624-7636. https://doi.org/10.3390/curroncol29100602