Table 3.
Parameters* | Optimal n | EVSI | ETC | ENG |
---|---|---|---|---|
Base-case | 38 | 26,455,033 | 9,341,061 | 17,113,972 |
High trial costs† | 37 | 26,369,240 | 10,630,102 | 15,739,138 |
Low trial costs‡ | 37 | 26,476,818 | 8,670,621 | 17,806,197 |
Enrollment fraction: 15 % | 31 | 24,788,219 | 9,497,153 | 15,291,066 |
Enrollment fraction: 25 % | 44 | 27,651,540 | 9,205,276 | 18,446,264 |
Time horizon: 15 years | 28 | 15,324,493 | 8,765,786 | 6,558,707 |
Time horizon: 25 years | 47 | 38,125,228 | 9,803,537 | 28,321,692 |
Discount rate: 3 % | 38 | 21,297,611 | 8,200,704 | 18,027,344 |
Discount rate: 5 % | 38 | 16,965,672 | 7,765,750 | 9,199,922 |
*The optimal sample size was 0 for FVIII 75 cent and $1.00 unit costs, indicating that current information is sufficient to favor TP over AP (i.e., no trial needed)
All costs are in U.S. dollars
†High-cost scenario assumes a fixed cost of $2,000,000 and a variable cost of $10,000
‡Low-cost scenario assumes a fixed cost of $500,000 and a variable cost of $2,500
ENG expected net gain; ETC expected total cost; EVSI expected value of sample information