Table 3.
Exploratory Analysis Incremental Results: Hypothetical Disease-Modifying Treatments vs HD Natural History
| DMT A vs HD natural history | DMT B vs HD natural history | DMT C vs HD natural history | ||||
| Base case | 95% credible range | Base case | 95% credible range | Base case | 95% credible range | |
| Prefunctional decline populationa (discounted) | ||||||
| Incremental costs | −$35,220 | −$142,904 to $20,972 | −$21,808 | −$92,426 to $25,927 | −$14,335 | −$69,357 to $33,443 |
| Standard care | −$9,086 | −$34,024 to $4,748 | −$5,646 | −$22,640 to $6,805 | −$3,651 | −$17,455 to $8,079 |
| Societal cost | −$26,134 | −$117,076 to $18,106 | −$16,161 | −$74,125 to $20,410 | −$10,684 | −$54,983 to $26,386 |
| Incremental QALYs | 1.42 | 0.74 to 2.03 | 0.92 | 0.29 to 1.57 | 0.40 | −0.24 to 1.09 |
| Prefunctional decline | 1.41 | 1.24 to 1.65 | 0.94 | 0.79 to 1.15 | 0.46 | 0.32 to 0.66 |
| Shoulson and Fahn stage 1 | 0.13 | −0.11 to 0.41 | 0.06 | −0.16 to 0.34 | 0.00 | −0.21 to 0.27 |
| Shoulson and Fahn stage 2 | 0.04 | −0.24 to 0.24 | 0.01 | −0.22 to 0.23 | −0.02 | −0.24 to 0.22 |
| Shoulson and Fahn stage 3 | −0.12 | −0.39 to −0.01 | −0.07 | −0.25 to 0.01 | −0.04 | −0.14 to 0.03 |
| Shoulson and Fahn stage 4–5 | −0.04 | −0.09 to −0.01 | −0.02 | −0.06 to −0.01 | −0.01 | −0.04 to 0.00 |
| Incremental life years | 1.48 | 0.47 to 2.29 | 0.97 | 0.10 to 1.79 | 0.40 | −0.49 to 1.30 |
| Shoulson and Fahn stage 1 population (discounted) | ||||||
| Incremental costs | $188,245 | −$34,489 to $395,879 | $115,352 | −$64,427 to $308,235 | $40,184 | −$135,263 to $215,048 |
| Standard care | $52,599 | −$3,679 to $108,738 | $32,723 | −$11,960 to $83,852 | $12,107 | −$33,590 to $58,901 |
| Societal cost | $135,646 | −$38,269 to $313,565 | $82,630 | −$59,226 to $239,628 | $28,077 | −$106,944 to $166,203 |
| Incremental QALYs | 3.13 | 1.61 to 4.61 | 1.96 | 0.53 to 3.54 | 0.82 | −0.62 to 2.43 |
| Shoulson and Fahn stage 1 | 2.70 | 2.11 to 3.55 | 1.75 | 1.23 to 2.54 | 0.82 | 0.32 to 1.48 |
| Shoulson and Fahn stage 2 | 0.40 | −0.27 to 1.07 | 0.20 | −0.41 to 0.91 | 0.02 | −0.59 to 0.72 |
| Shoulson and Fahn stage 3 | 0.05 | −0.41 to 0.30 | 0.02 | −0.28 to 0.29 | −0.02 | −0.32 to 0.27 |
| Shoulson and Fahn stage 4–5 | −0.02 | −0.16 to 0.01 | −0.01 | −0.09 to 0.02 | −0.01 | −0.06 to 0.02 |
| Incremental life years | 4.04 | 1.65 to 6.18 | 2.52 | 0.32 to 4.87 | 1.01 | −1.23 to 3.42 |
| Shoulson and Fahn stage 2 population (discounted) | ||||||
| Incremental costs | $279,724 | −$72,371 to $550,523 | $168,799 | −$72,088 to $403,636 | $62,875 | −$132,939 to $274,364 |
| Standard care | $72,437 | −$21,048 to $171,036 | $44,476 | −$16,205 to $118,729 | $17,327 | −$29,767 to $74,145 |
| Societal cost | $207,287 | −$67,763 to $432,404 | $124,323 | −$67,643 to $317,933 | $45,548 | −$109,101 to $214,394 |
| Incremental QALYs | 2.67 | 1.00 to 3.92 | 1.67 | 0.32 to 2.95 | 0.71 | −0.44 to 1.99 |
| Shoulson and Fahn stage 2 | 2.40 | 1.62 to 3.42 | 1.53 | 0.80 to 2.49 | 0.70 | 0.00 to 1.62 |
| Shoulson and Fahn stage 3 | 0.26 | −0.92 to 0.68 | 0.13 | −0.46 to 0.58 | 0.01 | −0.47 to 0.46 |
| Shoulson and Fahn stage 4–5 | 0.01 | −0.21 to 0.07 | 0.01 | −0.12 to 0.07 | 0.00 | −0.09 to 0.06 |
| Incremental life years | 4.17 | 0.74 to 6.16 | 2.59 | 0.12 to 4.71 | 1.07 | −1.02 to 3.22 |
Abbreviations: QALY = quality-adjusted life year; TFC = total functional score.
a
Prefunctional decline population: age = 40, CAG repeats = 40; results for prefunctional decline individuals with 45 and 50 CAG repeats are available in eAppendix 1.