TABLE 2.
Eculizumab and Ravulizumab Treatment Patterns in the 12-Month Post-Index Period
| Outcome | Eculizumab (n = 34) | Ravulizumab (n = 52) |
|---|---|---|
| New to all C5i therapy on index date, n (%) | 16 (47.2) | 17 (32.7) |
| New to index therapy on index date, n (%) | 16 (47.2) | 52 (100) |
| Discontinuation | ||
| Discontinuation of therapy, n (%)a | 10 (29.4) | 17 (32.7) |
| PNH ICD-10-CM subgroupb: discontinuation of therapy, n (%)a | 4 (17.4) | 13 (27.7) |
| Mean days to discontinuation, mean (SD) | 119.0 (67.9) | 274.9 (100.3) |
| PNH ICD-10-CM subgroupb: mean days to discontinuation, mean (SD) | 154.8 (84.6) | 284.5 (96.6) |
| Dose administered as maintenance therapy (excludes loading dose), n (%) | ||
| Normal (dosing per label recommendation) | 16 (47.0) | 43 (82.7) |
| Higher than standard | 13 (38.2) | 5 (9.6) |
| Lower than standard | 0 (0.0) | 0 (0.0) |
| Unknownc | 5 (14.7) | 4 (7.7) |
| Any dose escalation, 25% increase,d n (%) | ||
| Dose escalation at any point Q2-Q4 after Q1 | 1 (2.9) | 16 (30.8) |
| Dose escalation Q2 | 1 (2.9) | 5 (9.6) |
| Dose escalation Q3 | 0 | 12 (23.1) |
| Dose escalation Q4 | 0 | 13 (25.0) |
| Administration of therapy locations, n (%) | ||
| Hospital outpatient | 5 (14.7) | 21 (40.4) |
| Office | 19 (55.9) | 24 (46.2) |
| ED | 0 (0.0) | 0 (0.0) |
| In-home | 2 (5.9) | 4 (7.7) |
| Other | 8 (23.5) | 3 (5.8) |
a No claims for the index therapy in the 12-month post-index period after the old discontinuation date.
b A sensitivity analysis was performed among the subgroup of members in the sample with confirmed PNH diagnosis based on at least 2 medical claims with an ICD-10-CM code for PNH in the pre-index or post-index period (eculizumab: n = 23; ravulizumab: n = 47).
c Five members in the eculizumab cohort and 4 members in the ravulizumab cohort did not reach maintenance dosing and/or switched therapy.
d Members with a 25% greater- or less-than expected milligrams per day during their maintenance period were defined as not having a standard of care treatment. By normalizing the dose over time, batch billing was accounted for. For members new to therapy, the maintenance dose was established after accounting for the initial loading doses (5 for eculizumab and 1 for ravulizumab). The “number of services” variable in the medical claims data provided the number of units of medication. The daily maintenance dose was determined by dividing the total number of units of medication while on maintenance therapy by the number of days on maintenance therapy.
C5i = C5 inhibitor; ED = emergency department; ICD-10-CM = International Classification of Diseases, Tenth Revision, Clinical Modification; PNH = paroxysmal nocturnal hemoglobinuria; Q = quarter.