TABLE 1.
Characteristics of included SMA and CMA cancer indications
| SMA indications (N = 15) | Converted a CMA indications (N = 15) | Unconverted a CMA indications (N = 17) | ||||
|---|---|---|---|---|---|---|
| Pharmacotherapeutic group | ||||||
| Monoclonal antibodies (ATC code L01XC) | 5 | 33% | 5 | 33% | 6 | 35% |
| Protein kinase inhibitors (ATC code L01XE) | 8 | 53% | 7 | 47% | 8 | 47% |
| Other antineoplastic agents (ATC code L01XX) | 2 | 13% | 3 | 20% | 3 | 18% |
| Year of approval | ||||||
| 2004–2008 | 2 | 13% | 4 | 27% | 0 | 0% |
| 2009–2013 | 3 | 20% | 4 | 27% | 4 | 24% |
| 2014–2018 | 10 | 67% | 7 | 47% | 4 | 24% |
| 2019–2020 | 0 | 0% | 0 | 0% | 9 | 53% |
| Indication at initial approval | ||||||
| Solid tumours | 10 | 67% | 11 | 73% | 10 | 59% |
| ‐ Breast cancer | 1 | 7% | 1 | 7% | 0 | 0% |
| ‐ Basal cell cancer | 1 | 7% | 1 | 7% | 0 | 0% |
| ‐ Colorectal cancer | 1 | 7% | 1 | 7% | 0 | 0% |
| ‐ Cutaneous squamous cell cancer | 0 | 0% | 0 | 0% | 1 | 6% |
| ‐ Epithelial ovarian/fallopian tube/peritoneal cancer | 0 | 0% | 0 | 0% | 1 | 6% |
| ‐ Gastrointestinal stromal cancer | 0 | 0% | 1 | 7% | 1 | 6% |
| ‐ Melanoma | 1 | 7% | 0 | 0% | 0 | 0% |
| ‐ Merkel cell cancer | 0 | 0% | 1 | 7% | 0 | 0% |
| ‐ Non‐small cell lung cancer | 4 | 27% | 4 | 27% | 2 | 12% |
| ‐ Renal cell cancer | 2 | 13% | 2 | 13% | 0 | 0% |
| ‐ Soft tissue sarcoma | 0 | 0% | 0 | 0% | 1 | 6% |
| ‐ Thyroid cancer | 0 | 0% | 0 | 0% | 2 | 12% |
| ‐ Tissue agnostic | 0 | 0% | 0 | 0% | 2 | 12% |
| Hematological tumours | 5 | 33% | 4 | 27% | 7 | 41% |
| ‐ Leukaemia | 0 | 0% | 0 | 0% | 1 | 6% |
| ‐ Lymphoma | 4 | 27% | 3 | 20% | 4 | 24% |
| ‐ Multiple myeloma | 1 | 7% | 1 | 7% | 2 | 12% |
| Curative setting | 1 | 7% | 0 | 0% | 0 | 0% |
| Monotherapy | 10 | 67% | 14 | 93% | 14 | 82% |
| First‐line/line‐agnostic treatment | 3 | 20% | 4 | 27% | 6 | 35% |
| Orphan designation at initial approval | 4 | 27% | 6 | 40% | 11 | 65% |
| US FDA approval | ||||||
| Accelerated approval | 4 | 27% | 11 | 73% | 11 | 65% |
| Standard approval | 10 | 67% | 4 | 27% | 6 | 35% |
| Not approved | 1 | 7% | 0 | 0% | 0 | 0% |
| Time (median months, IQR) | ||||||
| To conversion (n = 15) | NA | 32 | 17–48 | NA | ||
| Unconverted (n = 16) | NA | NA | 19 | 7–73 | ||
| To revocation (n = 1) | NA | NA | 32 | NA | ||
| Amended indication after conversion to SMA | NA | 3 | 20% | NA | ||
ATC code, Anatomical Therapeutic Chemical code; CMA, conditional marketing authorization; IQR, interquartile range; NA, not applicable; SMA, standard marketing authorization; US FDA, United States Food and Drug Administration.
a
(Un)converted as at 31 December 2020.