TABLE 1.
Characteristics of 56 single patient INDs
| Characteristic |
Frequency (%) Median (range) |
|---|---|
| Type of IND Agent | |
| Small Molecule | 26 (46%) |
| Monoclonal antibody | 9 (16%) |
| Other | 21 (38%) |
| Type of Sponsor (n = 52) | |
| Institutional | 37 (71%) |
| NCI | 3 (6%) |
| Pharmaceutical | 12 (23%) |
| Single patient INDs with emergency filing (n = 37) | 5 (14%) |
| Time from FDA submission to safe to proceed (n = 31), days | 1 (0–12) |
| Time from IRB submission to IRB approval (n = 45), days | 4 (1–16) |
| Biologic justification for therapeutic approach (n = 55) | |
| Genotype‐based | 13 (24%) |
| Histology‐based | 39 (71%) |
| Other | 3 (5%) |
| Regulatory reason for IND therapy (n = 54) | |
| Pediatric formulation not available | 3 (6%) |
| No pediatric clinical trial available | 35 (65%) |
| Clinical trial accepts children, but child unable to travel to study center | 4 (7%) |
| Clinical trial available but patient not eligible | 10 (18%) |
| Other | 2 (4%) |