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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