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Table 2 Characteristic of rare and non-rare disease clinical trials within the ClinicalTrials.gov registry

From: A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov

Characteristics

Rare disease trials* (n = 2759)

Non-rare disease trials* (n =21329)

Difference in percentage points (rare–non-rare)

95% CI for difference

Year enrolment to protocol began, n (%)

    
 

Prior to 2006

455 (16.7)

2791 (13.2)

3.4

2 to 5

 

2006-2007

871 (31.9)

5121 (24.2)

7.6

6 to 10

 

2008-2009

859 (31.5)

5858 (27.8)

3.7

2 to 6

 

2010-2011

467 (17.1)

5315 (25.2)

−8.1

−7 to −10

 

2012 and after

78 (2.9)

2018 (9.6)

−6.7

−6 to −7

Overall status, n (%)

    
 

Completed

1169 (42.4)

8564 (40.2)

2.2

0 to 4

 

Not yet recruiting

15 (0.5)

1030 (4.8)

−4.3

−4 to −5

 

Recruiting

372 (13.5)

6840 (32.1)

−18.6

−17 to −20

 

Withdrawn

99 (3.6)

332 (1.6)

2.0

1 to 3

 

Active, not recruiting

638 (23.1)

2742 (12.9)

10.3

9 to 12

 

Terminated

379 (13.7)

1332 (6.2)

7.5

6 to 9

 

Suspended

36 (1.3)

154 (0.7)

0.6

0 to 1

 

Enrolling by invitation

51 (1.8)

335 (1.6)

0.3

0 to 1

Gender, n (%)

    
 

Female

116 (4.2)

1949 (9.1)

−4.9

−4 to −6

 

Male

60 (2.2)

1042 (4.9)

−2.7

−2 to −3

 

Both

2583 (93.6)

18338 (86.0)

7.6

7 to 9

 

Includes paediatric (<18), n (%)

    
 

Yes

546 (20.6)

2294 (11.1)

9.5

8 to 11

 

No

2106 (79.4)

18320 (88.9)

−9.5

−8 to −11

 

Includes Elderly (>65), n (%)

    
 

Yes

2291 (86.4)

17157 (83.2)

3.2

2 to 5

 

No

361 (13.6)

3457 (16.8)

−3.2

−2 to −5

Anticipated enrolment‡, n (%)

    
 

0-50

798 (61.7)

4556 (38.2)

23.5

21 to 26

 

51-100

280 (21.6)

2731 (22.9)

−1.3

0 to −4

 

101-500

195 (15.1)

3767 (31.6)

−16.5

−14 to −19

 

500+

21 (1.6)

877 (7.4)

−5.7

−5 to −6

Actual enrolment‡, n (%)

    
 

0-50

955 (71.4)

3570 (43.3)

28.1

25 to 31

 

51-100

211 (15.8)

1607 (19.5)

−3.7

−1 to −6

 

101-500

158 (11.8)

2402 (29.1)

−17.3

−15 to −19

 

500+

14 (1.0)

672 (8.1)

−7.1

−6 to −8

Lead Sponsor, n (%)

    
 

Industry

951 (34.5)

6437 (30.2)

4.3

2 to 6

 

NIH

72 (2.6)

602 (2.8)

−0.2

0.5 to −0.8

 

US Federal

15 (0.5)

472 (2.2)

−1.7

−1 to −2

 

Other

1721 (62.4)

13817 (64.8)

−2.4

−1 to −4

Location, n (%)

    
 

US only

1720 (62.3)

12073 (56.6)

5.7

4 to 8

 

Canada only

83 (3.0)

1387 (6.5)

−3.5

−3 to −4

 

EU only

733 (26.6)

6938 (32.5)

−6.0

−4 to −8

 

US/Canada

84 (3.0)

405 (1.9)

1.1

1 to 2

 

US/EU

93 (3.4)

333 (1.6)

1.8

1 to 3

 

EU/Canada

9 (0.3)

55 (0.3)

0

−0.1 to 0.4

 

US/EU/Canada

37 (1.3)

138 (0.6)

0.7

0.3 to 1

 

Number of countries, n (%)

    
 

Single country

2442 (88.5)

19847 (93.1)

−4.5

−3 to −6

 

2-3 countries

219 (7.9)

1057 (5.0)

3.0

2 to 5

 

≥4 countries

98 (3.6)

425 (2.0)

1.6

1 to 2

  1. *Denominators exclude missing values. Missing data values [Rare (%), Non-Rare(%)] are: Year of enrolment [29(1.1),226(1.1)]; Study duration [361(13.1),2784(13.1)]; Ages [107(3.9),715(3.3)]; Enrolment [127(4.6),1147(5.4)].
  2. ‡Anticipated and Actual enrolment are mutually exclusive in ClinicalTrials.gov (Rare Anticipated [1294] vs. Actual [1338]; Non-Rare Anticipated [11931] vs. Actual [8251]).
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172