Table 5 Capitalized cost per approved non-orphan and orphan drug based on sensitivity analysis parameters
From: Estimating the clinical cost of drug development for orphan versus non-orphan drugs
Out-of-pocket costs per approved drug | Capitalized costs per approved drug | |||||
|---|---|---|---|---|---|---|
Parameter | Non-orphan | Orphan | Ratio (orphan/non-orphan) | Non-Orphan | Orphan | Ratio orphan/non-orphan) |
Base case | $291,505,909 | $166,389,643 | 0.57 | $412,404,245 | $291,263,058 | 0.71 |
Number of trials per approved drug – restricted dataset | $217,706,869 | $99,866,706 | 0.46 | $306,399,947 | $172,737,264 | 0.56 |
Average number of subjects per trial – restricted dataset | $302,053,737 | $152,098,329 | 0.50 | $425,244,968 | $271,257,799 | 0.64 |
Multiplier for per patient costs for phase 2 and 3 – 1.5 x | Same as base case | $104,485,324 | 0.36 | $412,404,245 | $185,473,705 | 0.45 |
Multiplier for per patient costs for phase 2 and 3 – 3.5x | Same as base case | $228,293,962 | 0.78 | $412,404,245 | $397,052,411 | 0.96 |
Trial Duration – restricted dataset | $291,505,909 | $166,389,643 | 0.57 | $406,967,750 | $266,003,355 | 0.65 |
Transition Probabilities – DiMasi et al | $255,378,992 | Same as base case | 0.65 | $360,459,277 | Same as base case | 0.81 |
Transition Probabilities, Orphan Drugs- +20% Hay et al | Same as base case | $106,023,300 | 0.36 | Same as base case | $182,841,707 | 0.44 |
Transition Probabilities, Orphan Drugs- -20% Hay et al | Same as base case | $294,848,393 | 1.01 | Same as base case | $526,910,607 | 1.28 |
Discount rate – 3% | Same as base case | $326,048,291 | $202,067,762 | 0.62 | ||
Discount rate – 7% | Same as base case | $372,104,800 | $249,638,586 | 0.67 | ||
Excluding studies that did not report on trial phase | $277,006,845 | $150,354,877 | 0.54 | $389,798,014 | $272,349,673 | 0.70 |
Categorizing phase 1/2 studies as phase 2 and phase 2/3 studies as phase 3 | $286,564,341 | $169,382,779 | 0.59 | $404,141,006 | $293,331,017 | 0.73 |