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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 €