Table 2. Question: When can surrogate endpoints be used?
Stakeholder groups | Medical specialty | Pooled mean | ||||||
Regulator | Academia | Industry | P | CR | Other | P | ||
Not enough treatment options | 3.5 (1.2) | 3.6 (1.2) | 4.5 (0.6) | 0.012 | 3.9 (1.1) | 3.4 (1.3) | 0.24 | 3.7 (1.2) |
Large impact on health and well-being | 3.3 (1.4) | 3.4 (1.2) | 4.6 (0.5) | 0.001 | 3.8 (1.2) | 3.2 (1.3) | 0.08 | 3.6 (1.2) |
Scientifically validated | 4.3 (0.7) | 4.3 (1.0) | 4.8 (0.4) | 0.058 | 4.4 (0.9) | 4.2 (1.0) | 0.63 | 4.3 (1.0) |
Hard outcome studies are too costly | 2.1 (0.7) | 2.1 (1.0) | 3.4 (0.9) | <0.001 | 2.4 (1.1) | 2.4 (1.1) | 0.86 | 2.4 (1.1) |
Unmet clinical need | 3.9 (1.1) | 3.4 (1.1) | 4.7 (0.6) | <0.001 | 3.8 (1.2) | 3.7 (1.2) | 0.96 | 3.8 (1.2) |
Cannot be used without hard outcome studies | 2.4 (1.0) | 2.9 (1.1) | 1.6 (0.6) | <0.001 | 2.4 (1.1) | 2.5 (1.1) | 0.55 | 2.4 (1.1) |
Statements on when surrogate endpoints can be used in drug marketing authorization, provided that a post-marketing study with hard outcomes will be conducted. Results from a 5-point Likert response format (strongly disagree – strongly agree) are presented in mean (SD) according to stakeholder group or medical specialty group (cardio-renal vs. no cardio-renal). Statistical differences between groups are mainly driven by differences of industry respondents vs. regulators and academia. Abbreviations: CR, cardiorenal background; P, P value.