Table 2. Postmarket Safety Events at 10 Years and Associations Between These Events and Characteristics of Novel Therapeutics Approved by the US Food and Drug Administration Between 2001 and 2010.
| Characteristic | Proportion Affected by Postmarket Safety Event at 10 y, % (95% CI) | Bivariate Analysis | Multivariable Analysis | ||
|---|---|---|---|---|---|
| Difference in Proportion Affected by Postmarket Safety Event at 10 y, % (95% CI) | P Valuea | Incidence Rate Ratio (95% CI)b | P Value | ||
| Drug class | |||||
| Pharmaceutical | 29.7 (23.5 to 37.0) | 0 [Reference] | 1 [Reference] | ||
| Biologic | 36.1 (23.2 to 53.3) | 6.4 (−10.1 to 23.0) | .45 | 1.93 (1.06 to 3.52) | .03 |
| Therapeutic area | |||||
| Cancer and hematology | 21.4 (12.1 to 36.1) | 0 [Reference] | 1 [Reference] | ||
| Autoimmune, musculoskeletal, and dermatology | 42.5 (22.8 to 69.4) | 21.1 (−5.8 to 48.0) | .12 | 1.93 (0.87 to 4.28) | .11 |
| Cardiovascular, diabetes, and hyperlipidemia | 27.9 (14.3 to 50.0) | 6.5 (−14.8 to 27.8) | .55 | 1.46 (0.66 to 3.21) | .35 |
| Genitourinary and renal | 23.5 (9.6 to 51.2) | 2.1 (−21.2 to 25.5) | .86 | 1.23 (0.47 to 3.28) | .67 |
| Infectious disease | 27.4 (15.8 to 45.0) | 6.0 (−12.6 to 24.7) | .53 | 1.17 (0.57 to 2.41) | .67 |
| Neurology | 41.2 (22.2 to 67.5) | 19.8 (−6.4 to 46.0) | .14 | 1.53 (0.61 to 3.82) | .36 |
| Psychiatry | 60.0 (37.2 to 83.5) | 38.6 (11.2 to 66.1) | .006 | 3.78 (1.77 to 8.06) | <.001 |
| Other | 29.4 (18.2 to 45.4) | 8.0 (−9.9 to 26.0) | .38 | 1.75 (0.91 to 3.37) | .10 |
| Priority vs standard review | |||||
| Standard review | 32.3 (25.2 to 40.7) | 0 [Reference] | 1 [Reference] | ||
| Priority review | 27.8 (19.1 to 39.5) | 4.4 (−8.3 to 17.2) | .50 | 1.52 (0.87 to 2.65) | .14 |
| Accelerated vs not accelerated approval | |||||
| Not accelerated | 29.7 (23.7 to 36.8) | 0 [Reference] | 1 [Reference] | ||
| Accelerated | 39.3 (24.0 to 59.6) | 9.6 (−9.6 to 28.9) | .33 | 2.20 (1.15 to 4.21) | .02 |
| Orphan vs not orphan status | |||||
| Not orphan | 33.0 (26.2 to 41.0) | 0 [Reference] | 1 [Reference] | ||
| Orphan | 25.0 (15.9 to 38.2) | 8.0 (−5.3 to 21.3) | .24 | 0.60 (0.35 to 1.02) | .06 |
| Near–regulatory deadline vs regular approval | |||||
| Regular | 28.7 (22.5 to 36.2) | 0 [Reference] | 1 [Reference] | ||
| Near–regulatory deadline | 38.8 (26.6 to 54.2) | 10.1 (−5.4 to 25.6) | .20 | 1.90 (1.19 to 3.05) | .008 |
| Regulatory review time, d | |||||
| <200 | 24.7 (15.1 to 38.8) | 9.7 (−5.7 to 25.2) | .22 | 0.46 (0.24 to 0.87) | .02 |
| 200-399 | 34.4 (25.4 to 45.5) | 0 [Reference] | 1 [Reference] | ||
| ≥400 | 31.7 (22.5 to 43.4) | 2.7 (−11.8 to 17.3) | .71 | 1.27 (0.81 to 1.99) | .30 |
a
P values calculated using a Z test for differences in proportions. See text for full details.
b
Incidence rate ratios were derived from Poisson regression analysis using a composite of safety events as the dependent variable and 7 categories of novel therapeutic characteristics as independent variables. See text for full details.