Table 3 Clinical evidence considerations of each of the therapies studied
Drug Name (Number of assessments) | Kymriah DLBCL (6) | Kymriah ALL (6) | Yescarta (7) | Luxturna (7) | Strimvelis (1) | Imlygic (3) | Alofisel (5) | Provengea (2) | Glybera (2) | Zolgensma (7) |
|---|---|---|---|---|---|---|---|---|---|---|
Surrogate endpoint used | Yes (5) | Yes (5) | Yes (6) | Yes (6) | Yes (1) | Yes (2) | Yes (4) | No (2) | Yes (1) | Yes (2) |
Rare disease | Yes (3) | Yes (3) | Yes (4) | Yes (7) | Â | No (1) | Yes (4) | No (2) | Yes (2) | Yes (7) |
Serious condition | Yes (5) | Yes (5) | Yes (6) | Yes (6) | Yes (1) | Yes (2) | Yes (4) | Â | Â | Yes (6) |
Single-arm trial | Yes (6) | Yes (6) | Yes (7) | No (7) | Yes (1) | No (3) | No (5) | No (2) | Yes (2) | Yes (7) |
Pediatric population | No (6) | Yes (5) | No (7) | Yes (2) | Yes (1) | No (3) | No (5) | No (2) | No (2) | Yes (7) |
Reporting of adverse consequences and risks | Yes (6) | Yes (6) | Yes (7) | Yes (7) | Yes (1) | Yes (2) | Yes (5) | Yes (2) | Yes (1) | Yes (7) |
Size of clinical trial (number of patients) | 99–167 | 59–97 | 111 | 29–31 | 18 | 436 | 212–289 | 512 | 27 | 15–33 |
Length of clinical trials (months) | 13.9–40.3 | 8.7–30.2 | 8.7–27.1 | 12–48 |  | 48 | 12–24 | 20.6–34 |  < 12 | 12–53 |
Extrapolation to long-term outcomes (months) | 552 |  > 1,000 | 528 |  |  | 360 | 480 | 120 |  | 924 |