Table 2.
Outcomes by country and scenario.
| Countries by scenario | Total life years | Total DALYs | Total cost | |||||||
|---|---|---|---|---|---|---|---|---|---|---|
| GTx | SOC | Life years gained | GTx | SOC | DALYs averted | GTx | SOC | Incremental cost | ||
| Ghana | Pessimistic | 3.99 | 3.97 | 0.02 | 40.11 | 40.21 | 0.10 | $1133 | $1076 | $57 |
| Base case | 4.86 | 3.97 | 0.89 | 38.28 | 40.21 | 1.93 | $2141 | $1076 | $1065 | |
| Optimistic | 14.70 | 3.97 | 10.73 | 18.32 | 40.21 | 21.89 | $13,160 | $1076 | $12,084 | |
| NBS 25% | 5.93 | 4.97 | 0.95 | 35.28 | 37.78 | 2.49 | $2717 | $1341 | $1376 | |
| NBS 50% | 7.26 | 6.23 | 1.03 | 31.54 | 34.74 | 3.20 | $3437 | $1673 | $1765 | |
| SOCM 0% | 4.86 | 3.97 | 0.89 | 38.27 | 40.20 | 1.93 | $2126 | $1062 | $1064 | |
| SOCM 10% | 4.86 | 3.97 | 0.89 | 38.29 | 40.22 | 1.93 | $2156 | $1091 | $1065 | |
| India | Pessimistic | 3.99 | 3.97 | 0.02 | 38.66 | 38.76 | 0.10 | $958 | $918 | $40 |
| Base case | 4.88 | 3.97 | 0.91 | 36.87 | 38.76 | 1.89 | $1669 | $918 | $750 | |
| Optimistic | 15.04 | 3.97 | 11.07 | 17.24 | 38.76 | 21.52 | $9464 | $918 | $8545 | |
| NBS 25% | 5.96 | 4.97 | 0.98 | 33.95 | 36.39 | 2.45 | $2116 | $1145 | $971 | |
| NBS 50% | 7.30 | 6.23 | 1.07 | 30.29 | 33.43 | 3.14 | $2675 | $1427 | $1247 | |
| SOCM 0% | 4.88 | 3.97 | 0.91 | 36.86 | 38.75 | 1.89 | $1656 | $906 | $750 | |
| SOCM 10% | 4.88 | 3.97 | 0.91 | 36.88 | 38.77 | 1.89 | $1682 | $931 | $751 | |
| Kenya | Pessimistic | 3.99 | 3.97 | 0.02 | 40.32 | 40.43 | 0.10 | $1241 | $1191 | $50 |
| Base case | 4.86 | 3.97 | 0.89 | 38.49 | 40.43 | 1.93 | $2125 | $1191 | $933 | |
| Optimistic | 14.65 | 3.97 | 10.68 | 18.50 | 40.43 | 21.92 | $11,779 | $1191 | $10,588 | |
| NBS 25% | 5.92 | 4.97 | 0.95 | 35.49 | 37.98 | 2.50 | $2690 | $1484 | $1206 | |
| NBS 50% | 7.25 | 6.23 | 1.02 | 31.73 | 34.93 | 3.20 | $3397 | $1851 | $1546 | |
| SOCM 0% | 4.86 | 3.97 | 0.89 | 38.48 | 40.42 | 1.93 | $2108 | $1175 | $933 | |
| SOCM 10% | 4.86 | 3.97 | 0.89 | 38.50 | 40.44 | 1.93 | $2141 | £1208 | $934 | |
| Nigeria | Pessimistic | 3.99 | 3.97 | 0.02 | 34.70 | 34.79 | 0.09 | $1039 | $1019 | $20 |
| Base | 4.87 | 3.97 | 0.90 | 33.10 | 34.79 | 1.70 | $1398 | $1019 | $379 | |
| Optimistic | 14.79 | 3.97 | 10.82 | 15.73 | 34.79 | 19.06 | $5269 | $1019 | $4251 | |
| NBS 25% | 5.94 | 4.97 | 0.97 | 30.41 | 32.60 | 2.19 | $1758 | $1270 | $488 | |
| NBS 50% | 7.28 | 6.23 | 1.05 | 27.05 | 29.85 | 2.80 | $2209 | $1583 | $625 | |
| SOCM 0% | 4.87 | 3.97 | 0.90 | 33.09 | 34.78 | 1.70 | $1384 | $1005 | $379 | |
| SOCM 10% | 4.87 | 3.97 | 0.90 | 33.10 | 34.80 | 1.70 | $1412 | $1033 | $379 | |
| South Africa | Pessimistic | 3.98 | 3.97 | 0.01 | 40.08 | 40.18 | 0.10 | $8169 | $7850 | $319 |
| Base case | 4.83 | 3.97 | 0.86 | 38.30 | 40.18 | 1.88 | $13,906 | $7850 | $6056 | |
| Optimistic | 14.37 | 3.97 | 10.40 | 18.85 | 40.18 | 21.32 | $76,687 | $7850 | $68,837 | |
| NBS 25% | 5.88 | 4.97 | 0.91 | 35.33 | 37.74 | 2.41 | $17,570 | $9783 | $7787 | |
| NBS 50% | 7.19 | 6.23 | 0.97 | 31.62 | 34.70 | 3.08 | $22,150 | $12,199 | $9951 | |
| SOCM 0% | 4.83 | 3.97 | 0.86 | 38.29 | 40.17 | 1.88 | $13,799 | $7745 | $6054 | |
| SOCM 10% | 4.83 | 3.97 | 0.86 | 38.31 | 40.19 | 1.88 | $14,017 | $7959 | $6058 | |
| Uganda | Pessimistic | 3.99 | 3.97 | 0.02 | 39.83 | 39.93 | 0.10 | $471 | $459 | $12 |
| Base case | 4.86 | 3.97 | 0.89 | 38.02 | 39.93 | 1.92 | $689 | $459 | $230 | |
| Optimistic | 14.71 | 3.97 | 10.74 | 18.19 | 39.93 | 21.74 | $3068 | $459 | $2609 | |
| NBS 25% | 5.93 | 4.97 | 0.96 | 35.03 | 37.51 | 2.48 | $870 | $572 | $297 | |
| NBS 50% | 7.26 | 6.23 | 1.03 | 31.31 | 34.49 | 3.18 | $1095 | $714 | $381 | |
| SOCM 0% | 4.86 | 3.97 | 0.89 | 38.01 | 39.92 | 1.92 | $683 | $453 | $230 | |
| SOCM 10% | 4.86 | 3.97 | 0.89 | 38.03 | 39.94 | 1.92 | $696 | $466 | $230 | |
| Zambia | Pessimistic | 3.99 | 3.97 | 0.02 | 38.02 | 38.12 | 0.10 | $1047 | $990 | $57 |
| Base case | 4.84 | 3.97 | 0.87 | 36.30 | 38.12 | 1.81 | $2061 | $990 | $1071 | |
| Optimistic | 14.41 | 3.97 | 10.44 | 17.62 | 38.12 | 20.50 | $13,104 | $990 | $12,114 | |
| NBS 25% | 5.90 | 4.97 | 0.93 | 33.44 | 35.77 | 2.33 | $2613 | $1234 | $1379 | |
| NBS 50% | 7.22 | 6.23 | 0.99 | 29.86 | 32.85 | 2.99 | $3303 | $1539 | $1764 | |
| SOCM 0% | 4.84 | 3.97 | 0.87 | 36.29 | 38.11 | 1.81 | $2048 | $977 | $1071 | |
| SOCM 10% | 4.84 | 3.97 | 0.87 | 36.31 | 38.12 | 1.81 | $2075 | $1004 | $1071 | |
| France | Pessimistic | 8.16 | 8.58 | 0.43 | 35.42 | 36.12 | 0.70 | $173,884 | $135,238 | $38,646 |
| Base case | 9.93 | 8.58 | 1.34 | 30.38 | 36.12 | 5.74 | $450,928 | $135,238 | $315,691 | |
| Optimistic | 21.89 | 8.58 | 13.30 | 5.74 | 36.12 | 30.38 | $1,807,035 | $135,238 | $1,671,797 | |
| Germany | Pessimistic | 8.16 | 8.58 | 0.43 | 34.84 | 35.54 | 0.69 | $224,742 | $163,778 | $60,964 |
| Base case | 9.93 | 8.58 | 1.34 | 29.88 | 35.54 | 5.66 | $661,733 | $163,778 | $497,955 | |
| Optimistic | 21.81 | 8.58 | 13.22 | 5.65 | 35.54 | 29.89 | $2,794,945 | $163,778 | $2,631,167 | |
| Italy | Pessimistic | 8.16 | 8.58 | 0.42 | 35.41 | 36.12 | 0.71 | $122,761 | $87,489 | $35,272 |
| Base case | 9.94 | 8.58 | 1.36 | 30.36 | 36.12 | 5.76 | $375,376 | $87,489 | $287,887 | |
| Optimistic | 21.97 | 8.58 | 13.39 | 5.59 | 36.12 | 30.53 | $1,614,089 | $87,489 | $1,526,601 | |
| Spain | Pessimistic | 8.16 | 8.58 | 0.42 | 35.39 | 36.10 | 0.70 | $104,888 | $81,618 | $23,270 |
| Base case | 9.94 | 8.58 | 1.35 | 30.34 | 36.10 | 5.75 | $271,553 | $81,618 | $189,934 | |
| Optimistic | 21.96 | 8.58 | 13.38 | 5.60 | 36.10 | 30.50 | $1,088,512 | $81,618 | $1,006,893 | |
| UK | Pessimistic | 8.16 | 8.58 | 0.43 | 35.07 | 35.77 | 0.70 | $157,245 | $129,848 | $27,397 |
| Base case | 9.93 | 8.58 | 1.34 | 30.08 | 35.77 | 5.69 | $353,749 | $129,848 | $223,900 | |
| Optimistic | 21.84 | 8.58 | 13.26 | 5.68 | 35.77 | 30.08 | $1,314,467 | $129,848 | $1,184,618 | |
| USA | Pessimistic | 8.15 | 8.58 | 0.43 | 34.37 | 35.04 | 0.67 | $396,064 | $328,799 | $67,265 |
| Base case | 9.89 | 8.58 | 1.30 | 29.53 | 35.04 | 5.51 | $879,464 | $328,799 | $550,666 | |
| Optimistic | 21.59 | 8.58 | 13.00 | 5.99 | 35.04 | 29.05 | $3,233,854 | $328,799 | $2,905,056 | |
GTx gene therapy, LMIC low-middle income country, HIC high income country, SOC standard of care, NBS newborn screening, the percentage of the population screened at birth, SOCM standard of care multiplier, the quality of standard of care effect on transition probabilities.
Significant values are in bold.