Skip to main content

Advertisement

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