Clinical trial experience | Real-world experience | |||
Pooled phase III trials# [12] | Integrated trials¶ [20] | EAP [21] | PASSPORT [22] | |
Patients n | 623 | 1299 | 1620 | 1009 |
Duration of treatment years+ | 1.0 (0–2.3) | 1.7 (0–9.9) | 0.45 (0–0.91) | 1.0 (0–2.4) |
Cumulative total exposure PEY | 735.7 | 3159.9 | 707.9 | 415 |
AE type | TEAE | TEAE | ADR | ADR |
TEAEs or ADRs per 100 PEY§ | ||||
Total | 418.8 | 790.2 | 419.6 | 67.0 |
Nausea | 38.6 | 26.0 | 62.1 | 20.4 |
Rash | 33.3 | 16.2 | 19.3 | 12.7 |
Diarrhoea | 26.0 | 20.1 | 27.6 | 10.6 |
Fatigue | 21.2 | 16.0 | 52.4 | 17.9 |
Dyspepsia | 16.3 | 9.5 | 16.4 | 4.6 |
Anorexia | 10.2 | 7.1 | 16.8 | NA |
Dizziness | 9.9 | 12.7 | 14.4 | 6.5 |
Gastro-oesophageal reflux disease | 7.1 | 6.6 | 12.7 | 1.9 |
Decreased appetite | 6.8 | 5.1 | 13.7 | 15.8 |
Decreased weight | 3.4 | 7.3 | 6.6 | 15.6 |
ADR/TEAE resulting in death | 2.2 | 18.5ƒ | NA | 1.4 |
ADR/TEAE leading to discontinuation | 14.6 | 29.7 | 68.0 |
EAP: expanded access programme; PEY: patient-exposure years; AE: adverse event; TEAE: treatment-emergent adverse event; ADR: adverse drug reaction; NA: not available. #: CAPACITY (004/006) and ASCEND (016); ¶: phase III trials+RECAP+PIPF-002, including two patients in the PIPF-002 study with a diagnosis of “pulmonary fibrosis”; +: data presented as median (range); §: one PEY is the equivalent of one patient exposed to a study drug for 1 year, total PEY is the sum of the PEY for each patient, adjusted rate per 100 PEY=(total number of events/total years of exposure)×100; ƒ: combined data for death and discontinuation.