TABLE 1.
Estimates and Sources Relevant to CETs
| HTA Body, Methodology, or Technology | Relevant Utility and Cost-Effectiveness Estimates | Implied CET or Incremental Cost-Effectiveness Ratio | Sources |
|---|---|---|---|
| HTA body/government agency | |||
| ICER consensus range | $50K-150K for non-orphans $175k-$500k for ultra-orphans |
$175K-$500K per QALY for ultraorphans | ICER (January 2018; 2017)8,17 |
| Value of a statistical life (U.S. HHS) | Central: $9.9M; range: $4.6M-$15.0M (2014 USD) | $328K per QALY | ASPE (2016)22 |
| Value of a statistical life (U.S. DOT) | Mean: $9.6M; range: $5.4M-$13.4M (2015 USD) | $315K per QALY | Moran and Monje (2016)23 |
| NICE range | £20K-£300K for highly specialized technologies | $390K per QALY | NICE (2017)18 |
| Methodological approaches | |||
| Value of a statistical life (systematic literature review) | Midpoint: $6.5M; $2M to $11.1M per life | $213K per LY | Bosworth et al. (2017)19 |
| Value of a statistical life (systematic review and quantitative analysis) | Included human capital, contingent valuation, and revealed preference studies | $25K-$428K (medians) across study types (1997 USD) | Hirth et al. (2000)4 |
| Welfare economics theory | 2 times per capita GDP | $119K per QALY | Garber and Phelps (1997)11 |
| Opportunity-cost approach | £13K (in relation to U.K. GDP per capita of £39.7K) | $20K per QALY | Claxton et al. (2013)15; Woods et al. (2016)16 |
| Expert consensus | 1-3 times per capita GDP | $60K-$179K per QALY | WHO (2001)12 |
| Rule of rescue for nonmedical identified lives | Thousands and millions | NA | Cookson (2017)26 |
| Specific health technologies | |||
| Hemodialysis for end-stage renal disease | Utility on dialysis: 0.6 1980: Average cost per year: $50K Implied CET: $83K 2016: Average cost per year: $89K Implied CET: $148K |
$148K per QALY | Authors’ calculations; Grosse (2008)6; Wyld (2012)9; U.S. Renal Data System (2017)10 |
| Hemophilia A with bypassing agents | For patients aged <12 years, discounted lifetime costs and QALYs: No prophylaxis: $31M, 20.40 QALYs BPA prophylaxis: $99M, 22.41 QALYs Emicizumab prophylaxis: $21M, 22.79 QALYs |
$39M per QALY Cost-saving; dominant | ICER (April 2018)27 |
| Inherited retinal disease—voretigene neparvovec | Drug wholesale acquisition cost: $855K Average QALY gain: 1.3 (treatment age 12) Icer: $644K per QALY |
$644K per QALY | ICER (February 2018)28 |
| Cystic fibrosis with gating mutation—ivacaftor | Total lifetime drug cost: $7.44M Average QALYs gain: 6.73 Icer: $957K per QALY |
$957K per QALY | ICER (May 2018)29 |
| CAR-T therapy for B-cell acute lymphoblastic leukemia | Tisagenlecleucel (vs. clofarabine) Total discounted lifetime cost: $667K Total discounted QALYs gained: 7.18 Icer: $46K per QALY Axicabtagene ciloleucel (vs. chemotherapy) Total discounted lifetime cost: $617K Total discounted QALYs gained: 3.40 Icer: $136K per QALY |
$46K per QALY $136K per QALY |
ICER (March 2018)32 |
| C1 esterase inhibitors for hereditary angioedema | No prophylaxis: $10.0M, 17.47 QALYs Cinryze: $14.4M, 18.21 QALYs Haegarda: $10.3M, 18.65 QALYS |
Cinryze: $5.9M per QALY Haegarda: $328K per QALY |
ICER (November 2018)30 |
| Nusinersen (Spinraza) for SMA (type 1) | Drug cost: $750K Year 1 and $375K annually thereafter | > $375K per QALY | Medi-Span (2018)31 |
| Organ transplants | Estimated billed charges (2017; 5-year survival):
|
NA | Bentley and Phillips (2017)33 |
ASPE = Office of the Assistant Secretary for Planning and Evaluation; BPA = bypassing agent; CAR-T = chimeric antigen receptor T-cell therapy; CET = cost-effectiveness threshold; DOT = Department of Transportation; GDP = gross domestic product; HHS = Department of Health and Human Services; HTA = health technology assessment; ICER = Institute for Clinical and Economic Review; Icer = incremental cost-effectiveness ratio; LY = life-year; NA = not available; NICE = National Institute for Health and Care Excellence; QALY = quality-adjusted life-year; SMA = spinal muscular atrophy; USD = U.S. dollars; WHO = World Health Organization.