Skip to main content

Table 1 CDR assessment results for 55 submissions by prevalence of treated disorder

From: Health technology assessment of new drugs for rare disorders in Canada: impact of disease prevalence and cost

 

Prevalence of treated disorder (per 100,000)

p-value

<50 to >10

≤10 to >1

≤1

n = 19

n = 23

n = 13

Median assessment time (range)

203 (164–251)

195 (126–392)

232 (153–439)

0.409

Median daily cost (range)a

$55 ($13–$252)

$186 ($26–$1800)

$796 ($181–$1460)

0.0001

Median number of RCTs (range)

2 (1–6)

2 (1–4)

1 (0–4)

0.100

Median number of patients in RCTs (range)

120 (15–555)

118 (12–742)

58 (11–177)

0.020

No. with a negative recommendation

5 (26.3%)

10 (43.5%)

10 (76.9%)

0.006

No. with acceptable clinical efficacy evidence

14 (73.7%)

11 (47.8%)

2 (15.4%)

0.001

No. with concern about cost-effectiveness analysis

9 (47.4%)

18 (78.3%)

12 (92.3%)

0.004

No. with high cost or need for price reduction stated in the recommendation

3 (15.8%)

10 (43.5%)

6 (46.2%)

0.057

No. with high cost noted elsewhere in the report

0 (0.0%)

1 (4.3%)

2 (15.4%)

0.067

No. with high cost or need for price reduction noted anywhere in the report

3 (15.8%)

11 (47.8%)

8 (61.5%)

0.007

  1. CDR Common Drug Review, RCT Randomized clinical trial
  2. aCalculated for drugs used daily from prices available in the CDR reports