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Table 1 Characteristics of the published and unpublished studies included in the analysis (n = 62)

From: Italian program for independent research on drugs: 10 year follow-up of funded studies in the area of rare diseases

  Published Unpublished p(chi-square) Total
  N % N %   N %
Studies funded and started 39 62.9 23 37.1   62 100.0
Clinical area      0.604   
 Oncology/hematology 11 28.2 8 34.8   19 30.6
 Congenital and genetic disorders 13 33.3 4 17.4   17 27.4
 Immune system disorders 4 10.3 3 13.0   7 11.3
 Other 11 28.2 8 34.8   19 30.6
Prevalence of rare disease      0.381   
  > 1/10,000 14 35.9 7 30.4   21 33.9
 1–9/100,000 12 30.8 11 47.8   23 37.1
 1–9/1,000,000 13 33.3 5 21.8   18 29.0
Study characteristics        
Design      0.055   
 Randomized clinical trials (RCT) 21 53.8 18 78.3   39 62.9
 Uncontrolled clinical trials (CT) 18 46.2 5 21.7   23 37.1
Type of control group (n = 39) 21 100.0 18 100.0 0.584 39 100.0
 Active control 11 52.4 11 61.1   22 56.4
 Placebo control/no treatment 10 47.6 7 38.9   17 43.6
Planned sample size (tertiles)      0.901   
  < =46 patients 13 33.3 8 34.8   21 33.9
 47-100 patients 14 35.9 7 30.4   21 33.9
  > 100 patients 12 30.8 8 34.8   20 32.3
 Mean 98   107    101  
 Median 60   80    60  
 Q1-Q3 30–120   29–177    30–124  
Special populations      0.307   
 Only pediatrics 12 30.8 6 26.1   18 29.0
 Also pediatrics 4 10.3 0 0.0   4 6.5
 Only adults 15 38.5 9 39.1   24 38.7
 Adults and elderly 8 20.5 8 34.8   16 25.8