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Table 2 Results of the univariate analysis: association between the annual treatment costs and the covariates

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
  1. ATC class: A Alimentary tract and metabolism; B Blood and blood forming organs; C Cardiovascular system; G Genito-urinary system and sex hormones; H Systemic hormonal preparations, excluding sex hormones and insulins; J Antiinfectives for systemic use; L Antineoplastic and immunomodulating agents; N Nervous system; R Respiratory system; V Various
  2. Costs are given in €