PB1892 REAL WORLD TREATMENT PERSISTENCE OF NEW ZEALAND IBRUTINIB CHRONIC LYMPHOCYTIC LEUKEMIA PATIENTS IN A NAMED PATIENT PROGRAM

Background: Named Patient Programs (NPPs) provide access to medicines in response to unsolicited requests by physicians for specific patients before they become commercially available. Janssen New Zealand opened a NPP for ibrutinib for patients with relapsed or refractory Chronic Lymphocytic Leukemia (CLL) in September 2014. The program closed for new enrolments in April 2018, but enrolled patients have continued to receive treatment as of 7 th January 2019. Aims: To analyse duration on ibrutinib treatment for patients with relapsed/refractory CLL enrolled in a New Zealand NPP. Methods: A retrospective cohort analysis was conducted using data entered by treating physicians when enrolling patients into the NPP via the Janssen Managed Access Portal (MacWeb). At each resupply patients were provided with 3 months’ supply of ibrutinib. Patient time on treatment was estimated using fulfilled resupply requests submitted by physicians. Patients were considered to be on treatment until the date of last ibrutinib resupply, plus one month. A patient was considered discontinued based on physician declaration, or if no ibrutinib had been resupplied for four months or more. All other patients were censored at the reporting date of 7 th January 2019. This real‐world estimate of treatment duration was compared to time to treatment discontinuation in the ibrutinib arm of the RESONATE trial, as reported in Hillmen et al. EHA; 2016 (abstract 133196) and Tam et al. EHA; 2018 (abstract 214826). Time on treatment was evaluated using Kaplan‐Meier (KM) curves, and statistical testing was conducted using log‐rank testing. Results: 112 ibrutinib‐treated patients with CLL were included in the analysis. 59% of the population were male, 37% had 3 or more prior lines of therapy before commencing therapy, and 32% had refractory disease. 73% of patients were on therapy at 12 months (95% CI: 0.64‐0.8) and median time to discontinuation has not been reached at the time of the analysis. 42% of patients were estimated to have discontinued therapy over the 49‐month period. The comparison between patient time on treatment on the New Zealand NPP and the RESONATE trial were statistically not different (HR 1.01; 95% CI: 0.71‐1.44; p = 0.96). Summary/Conclusion: This real‐world analysis estimates that patient time on NPP treatment in New Zealand closely tracks time on treatment as observed in the RESONATE trial. While there are limitations to the NPP data as it was based on unmonitored physician declarations, and duration on treatment was estimated from resupply data, these findings suggest that duration of treatment in a New Zealand non‐trial setting is similar to that in the Phase 3 clinical trial.