Table 4. . Pharmaceutical Benefits Advisory Committee single-arm trial oncology submissions by external control data source and external control manufacturer's justification, patient and disease characteristics and final decision-making.
| EC source | Total PBAC SAT submissions, n (%) | SAT submissions with a biomarker-defined patient population | SAT submissions in an orphan disease indication | Final HTA recommendations | Any EC justification described in the SAT submission | |
|---|---|---|---|---|---|---|
| Positive | Negative | |||||
| Published, aggregate data derived from an RCT or a SAT only | 7 (36.8%) | 5 (26.3%) | – | 5 (26.3%) | 2 (10.5%) | 2 (10.5%) |
| IPD data derived from an RCT or a SAT only | – | – | – | – | – | – |
| Published, aggregate data derived from observational studies only | 5 (26.3%) | – | – | 2 (10.5%) | 1 (5.3%) | 3 (15.8%) |
| IPD data derived from RWD only | – | – | – | 2 (10.5%) | 1 (5.3%) | |
| Concurrent internal controls only | – | – | – | – | – | – |
| Multiple sources (combination of the above) | 7 (36.8%) | 4 (15.8%) | – | 7 (36.8%) | – | 2 (10.5%) |
EC: External control; HTA: Health technology assessment; IPD: Individual patient data; PBAC: Pharmaceutical Benefits Advisory Committee; RCT: Randomized controlled trial; RWD: Real-world data; SAT: Single-arm trial.