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Table 3 Distribution of positive recommendations for all new submissions of DRDs

From: Factors associated with positive and negative recommendations for cancer and non-cancer drugs for rare diseases in Canada

  All new submissions Non-Cancer drugs Cancer drugs
Factors n Positive recommendations % Positive p-value* n Positive recommendations % Positive p-value* n Positive recommendations % Positive p-value*
All 92 71 77.2   35 27 77.1   57 44 77.2  
Type of condition     0.996         
 Cancer 57 44 77.2          
 Non-cancer 35 27 77.1          
Submission characteristics
Presence of RCTs     0.083     0.033     0.727
 No 22 14 63.6   7 3 42.9   15 11 73.3  
 Yes 70 57 81.4   28 24 85.7   42 33 78.6  
Therapeutic class of drug     0.714     0.418   NA   
 Alimentary tract & metabolism 10 9 90.0   10 9 90.0      
 Antineoplastic & immunomodulating 68 51 75.0   11 7 63.6      
 Others 14 11 78.6   14 11 78.6      
Characteristics of disease
Prevalence     1.000     1.000     1.000
 Ultra- orphan 14 11 78.6   8 6 75.0   6 5 83.3  
 Orphan 78 60 96.9   27 21 77.8   51 39 76.5  
Clinical need     0.051     0.419     0.070
 No/ not stated 28 18 64.3   20 14 70.0   8 4 50.0  
 Yes 64 53 82.8   15 13 86.7   49 40 81.6  
Clinical safety/ efficacy
Safety issues     0.021     0.226     0.102
 Yes 33 21 63.6   11 7 63.6   22 14 63.6  
 No 59 50 84.7   24 20 83.3   35 30 85.7  
Improvements in biomarker/surrogate outcomes     0.316     0.228     0.006
 No/ inconsistent/ not measured 27 19 70.4   17 15 88.2   10 4 40.0  
 Yes 65 52 80.0   18 12 66.7   47 40 85.1  
Improvements in clinical outcomes     0.005     0.073     0.084
 No/ inconsistent/ not measured 66 46 69.7   25 17 68.0   41 29 70.7  
 Yes 26 25 96.1   10 10 100.0   16 15 93.7  
Improvements in PRO     0.010     0.299     0.042
 No/ inconsistent/ not measured 67 47 70.1   29 21 72.4   38 26 68.4  
 Yes 25 24 96.0   6 6 100.0   19 18 94.7  
Quality of evidence
Availability of comparative data     0.427     1.000     0.510
 No 33 27 81.8   14 11 78.6   19 16 84.2  
 Yes 59 44 74.6   21 16 76.2   38 28 73.7  
Consistency between population in trials and indications     0.130     0.431     0.345
 No 48 34 70.8   21 15 71.4   27 19 70.4  
 Yes 44 37 84.1   14 12 85.7   30 25 83.3  
Bias in outcome measures     0.503     0.216     1.000
 Yes 54 43 79.6   12 11 91.7   42 32 76.2  
 No 38 28 73.7   23 16 69.6   15 12 80.0  
Long term data     0.186     0.390     0.346
 No 62 45 72.6   25 18 72.0   37 27 73.0  
 Yes 30 26 86.7   10 9 90.0   20 17 85.0  
Other study design issues     0.202     1.000     0.044
 Yes 58 42 72.4   19 15 78.9   39 27 69.2  
 No 34 29 85.3   16 12 75.0   18 17 94.4  
Cost/ cost-effectiveness
Daily treatment cost     1.000     0.298     0.258
  ≤ 150 19 15 78.9   13 12 92.3   6 3 50.0  
 150–500 52 40 76.9   8 5 62.5   44 35 79.6  
  > 500 21 16 76.2   14 10 71.4   7 6 85.7  
ICER in $CDN/QALYs a     0.647     1.000     0.194
  ≤ 100,000 12 11 91.7   2 2 100.0   10 9 90.0  
 100,000-500,000 48 37 77.1   7 6 85.7   41 31 75.6  
  > 500,000 16 13 81.2   12 11 91.7   4 2 50.0  
  1. DRDs Drugs for rare diseases, ICER Incremental cost-effectiveness ratio, NA Not applicable, PRO Patient-reported outcomes, RCT Randomized controlled trial
  2. *p-values based on Pearson’s chi-square statistic or Fisher’s exact test
  3. aData on ICER was only available for 76 recommendations
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172