From: Determinants of orphan drugs prices in France: a regression analysis
Categorical variables | |||||
Mean | Median | 95% CI | Min-Max | p-value | |
Severity | |||||
Severe | 96,385 (174,479) | 25,069 | [−9052; 201,821] | [1474; 509,600] | p = 1.00 |
Not severe | 96,549 (170,493) | 38,482 | [50,459 ; 142,640] | [1524; 912,600] | |
Availability of alternative treatments | |||||
None | 131,253 (211,910) | 53,746 | [37,297; 225,209] | [2080; 912,600] | p = 0.02 |
One | 70,280 (71,467) | 40,746 | [19,155; 121,404] | [1474; 198,640] | |
Several | 45,572 (92,292) | 18,695 | [11,109; 80,034] | [1500; 481,581] | |
Non-pharmacological | 267,615 (287,063) | 205,962 | [−33,638; 568,869] | [3163; 676,260] | |
ATC class | |||||
L | 44,473 (60,908) | 29,525 | [22,513; 66,432] | [1474; 347,100] | p = 0.01 |
C | 36,044 (8004) | 37,108 | [23,309; 48,780] | [23,309; 48,780] | |
A | 280,878 (277,242) | 197,886 | [120,803; 440,953] | [1524; 912,600] | |
J | 11,278 (4622) | 13,236 | [−203; 22,760] | [6000; 14,600] | |
N | 10,682 (10,669) | 7688 | [−2565; 23,930] | [2637; 29,200] | |
H | 186,122 (237,122) | 52,560 | [−402,923; 775,166] | [45,905; 459,900] | |
B | 45,349 (15,033) | 37,799 | [8006; 82,692] | [35,588; 62,660] | |
V, R, G | 70,488 (110,625) | 22,614 | [−105,542; 246,517] | [2080; 234,642] | |
Treatment line, | |||||
First | 140,828 (210,443) | 44,142 | [73,525; 208,131] | [1474; 912,600] | p = 0.13 |
Subsequent | 33,218 (25,149) | 25,655 | [23,466; 42,969] | [2637; 95,265] | |
Age of targeted population | |||||
Adults | 44,554 (46,449) | 34,219 | [28,347; 60,761] | [1500; 198,640] | p = 0.53 |
Paediatric | 262,492 (292,292) | 192,477 | [100,626; 424,359] | [1474; 912,600] | |
Adults and paediatric | 66,384 (100,464) | 33,566 | [8378; 124,390] | [1524; 347,100] | |
Elderly | 36,321 (12,527) | 44,604 | [20,767; 51,875] | [20,440; 46,888] | |
MA date | |||||
2202–2003 | 122,186 (166,320) | 33,941 | [−16,860; 261,233] | [4623; 481,581] | p = 0.66 |
2004–2005 | 102,040 (182,695) | 37,800 | [−66,924; 271,005] | [1500; 509,600] | |
2006–2007 | 160,681 (276,804) | 38,482 | [7393; 313,971] | [1524; 912,600] | |
2008–2009 | 65,466 (111,874) | 43,680 | [3512; 127,420] | [5451; 459,900] | |
2010–2011 | 63,544 (80,581) | 32,394 | [−21,021; 148,108] | [2637; 219,889] | |
2012–2013 | 88,466 (116,624) | 46,888 | [10,117; 166,815] | [3163; 382,200] | |
2014–2015 | 20,806 (16,447) | 20,775 | [3546; 38,066] | [1474; 41,366] | |
IAB score | |||||
I | 101,666 (186,914) | 33,941 | [−94,488; 297,820] | [4745; 481,581] | p = 0.20 |
II | 148,647 (206,028) | 62,660 | [63,603; 233,691] | [12,093; 912,600] | |
III | 111,831 (199,604) | 49,724 | [−3417; 227,080] | [2080; 676,260] | |
IV | 16,724 (16,359) | 9265 | [7664; 25,783] | [1474; 45,905] | |
V | 50,807 (83,243) | 19,758 | [−36,551; 138,165] | [6000; 219,889] | |
Type of study | |||||
Phase II | 48,782 (49,992) | 40,038 | [19,917; 77,646] | [1500; 198,640] | p = 0.40 |
Phase III | 116,757 (197,406) | 38,483 | [56,004; 177,510] | [2080; 912,600] | |
Other | 78,156 (145,794) | 14,600 | [−19,790; 176,102] | [1474; 481,581] | |
Comparator | |||||
No | 90,783 (153,970) | 33,566 | [18,723; 162,844] | [1500; 481,581] | p = 0.50 |
Placebo | 128,831 (226,137) | 35,588 | [39,374; 218,288] | [2637; 912,600] | |
Active | 68,347 (73,068) | 44,142 | [32,012; 104,683] | [32,012; 104,683] | |
Endpoint | |||||
Surrogate | 110,012 (187,794) | 41,366 | [53,593; 166,431] | [1500; 912,600] | p = 0.50 |
Hard | 71,632 (113,019) | 32,190 | [−177; 143,441] | [2080; 382,200] | |
Continuous variables | |||||
Pearson Correlation | p-value | Spearman Correlation | p-value | ||
Prevalence | −0.32472 | p = 0.01 | −0.28433 | p = 0.02 | |
Delay between HTA and commercialisation | 0.04208 | p = 0.73 | −0.19956 | p = 0.10 |