Table 3 Comparison of pre-clinical and clinical trial outcomes by domain
Outcome domain | Pre-clinical in vivo mouse studies | Phase 3/4 clinical trials [20] | ||
|---|---|---|---|---|
Total number of unique outcomes within domain (verbatim outcomes) | Number of studies reporting one or more outcomes in the domain (%) | Total number of unique outcomes within domain (verbatim outcomes) | Number of studies reporting one or more outcomes in the domain (%) | |
1. Mortality/survival | 2 (7) | 7 (2.5) | 2 (3) | 3 (2.2) |
2. Blood and lymphatic system outcomes | 12 (23) | 13 (4.6) | 14 (19) | 9 (6.5) |
3. Cardiac outcomes | 6 (11) | 8 (2.9) | 11 (56) | 20 (14.5) |
4. Congenital, familial and genetic outcomes | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
5. Endocrine outcomes | 32 (324) | 164 (58.6) | 14 (50) | 31 (22.5) |
6. Ear and labyrinth outcomes | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
7. Eye outcomes | 0 (0) | 0 (0) | 2 (2) | 2 (1.4) |
8. Gastrointestinal outcomes | 7 (9) | 7 (2.5) | 12 (20) | 5 (3.6) |
9. General outcomes | 26 (296) | 171 (61.1) | 40 (146) | 65 (47.1) |
10. Hepatobiliary outcomes | 23 (123) | 70 (25) | 12 (25) | 12 (8.7) |
11. Immune system outcomes | 35 (84) | 26 (9.3) | 32 (73) | 28 (20.3) |
12. Infection and infestation outcomes | 0 (0) | 0 (0) | 7 (8) | 4 (2.9) |
13. Injury and poisoning outcomes | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
14. Metabolism and nutrition outcomes | 317 (1816) | 279 (99.6) | 105 (582) | 121 (87.7) |
15. Musculoskeletal and connective tissue outcomes | 21 (42) | 19 (6.8) | 2 (2) | 2 (1.4) |
16. Outcomes relating to neoplasms: benign, malignant and unspecified (including cysts and polyps) | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
17. Nervous system outcomes | 14 (18) | 7 (2.5) | 16 (16) | 6 (4.3) |
18. Pregnancy, puerperium and perinatal outcomes | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
19. Renal and urinary outcomes | 19 (45) | 30 (10.7) | 53 (76) | 27 (19.6) |
20. Reproductive system and breast outcomes | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
21. Psychiatric outcomes | 2 (3) | 3 (1.1) | 2 (2) | 2 (1.4) |
22. Respiratory, thoracic and mediastinal outcomes | 2 (5) | 4 (1.4) | 3 (11) | 23 (16.7) |
23. Skin and subcutaneous tissue outcomes | 0 (0) | 0 (0) | 1 (1) | 1 (0.7) |
24. Vascular outcomes | 8 (9) | 8 (2.9) | 56 (134) | 52 (37.7) |
25. Physical functioning | 4 (27) | 17 (6.1) | 3 (7) | 5 (3.6) |
26. Social functioning | 0 (0) | 0 (0) | 4 (6) | 5 (3.6) |
27. Role functioning | 0 (0) | 0 (0) | 3 (6) | 3 (2.2) |
28. Emotional functioning/wellbeing | 0 (0) | 0 (0) | 23 (28) | 8 (5.8) |
29. Cognitive functioning | 1 (2) | 2 (0.7) | 16 (22) | 2 (1.4) |
30. Global quality of life | 0 (0) | 0 (0) | 2 (5) | 4 (2.9) |
31. Perceived health status | 0 (0) | 0 (0) | 2 (4) | 4 (2.9) |
32. Delivery of care | 1 (1) | 1 (0.4) | 18 (60) | 30 (21.7) |
33. Personal circumstances | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
34. Economic | 0 (0) | 0 (0) | 6 (6) | 4 (2.9) |
35. Hospital | 0 (0) | 0 (0) | 3 (4) | 3 (2.2) |
36. Need for intervention | 0 (0) | 0 (0) | 7 (24) | 16 (11.6) |
37. Societal/carer burden | 0 (0) | 0 (0) | 0 (0) | 0 (0) |
38. Adverse events/effects | 5 (29) | 20 (7.1) | 4 (46) | 33 (23.9) |