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Table 2 Comparison of study variables in DURDs and DORDs submissions

From: Characteristics of drugs for ultra-rare diseases versus drugs for other rare diseases in HTA submissions made to the CADTH CDR

Variable

DURD N = 14

DORD N = 46

Effect size (95% CI)

P value

Molecular structure

 Biologic, n (%)

11 (78.6)

17 (37.0)

OR = 6.06 (1.25 to 38.58)

0.012

 Small molecule, n (%)

3 (21.4)

29 (63.0)

  

 Missing data, n (%)

0 (0)

0 (0)

  

Number of clinical studies considered, median (range)

3 (3)

2 (13)

Median difference = 0 (0 to 1)

0.305

 Missing data, n (%)

1 (7.1)

1 (2.2)

  

Level of evidence considered

 At least one double blinded RCT, n (%)

8 (57.1)

42 (91.3)

OR = 0.13 (0.02 to 0.70)

0.007

 At least one open label RCT, n (%)

0 (0)

3 (6.5)

  

 At least one non-randomized uncontrolled trial, n (%)

6 (42.9)

1 (2.2)

  

 Missing data, n (%)

0 (0)

0 (0)

  

Size of largest study, median, n of patients (range)

59 (156)

167 (1134)

Median difference = −108 (−234 to −50)

0.0001

 Missing data, n (%)

0 (0)

5 (10.9)

  

Clinical study comparator

 No control, n (%)

5 (35.7)

1 (2.2)

OR = 23.11 (2.23 to 1207.19)

0.0019

 Historical control, n (%)

1 (7.1)

0 (0)

  

 placebo, n (%)

7 (50.0)

36 (78.3)

  

 Active control, n (%)

1 (7.1)

9 (19.6)

  

 Missing data, n (%)

0 (0)

0 (0)

  

Annual treatment cost per patient, median (range)

CAN$330,395 (CAN$934,000)

CAN$52,596 (CAN$429,858.5)

Median difference = CAN$243,787.75 (83,396 to 329,050)

P < 0.0001

 Missing data, n (%)

1 (7.1)

17 (37.0)

  

Incremental cost per quality adjusted life-year, median (range)

CAN$2,680,000 (CAN$560,000)

CAN$ 165,923.5 (CAN$4,574,241)

NA

NA

 Missing data, n (%)

12 (85.7)

24 (52.2)

  

Recommendation

 Positive recommendation, n (%)

5 (35.7)

33 (71.7)

OR = 0.22 (0.05 to 0.91)

0.025

 Negative recommendation, n (%)

9 (64.3)

13 (28.3)

  

 Missing data, n (%)

0 (0)

0 (0)

  
  1. *Bold P value indicates statistical significance