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Background: NYVEPRIA, a pegfilgrastim (a long-acting granulocyte colony-stimulating factor [G-CSF]) biosimilar, was recently recommended for marketing authorization in Europe for decreasing the incidence of febrile neutropenia (FN) in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs. The present study aimed to evaluate the financial impact of introducing a new pegfilgrastim biosimilar from a French healthcare system perspective. Methods: An Excel-based budget impact model was developed to estimate the financial impact by introducing a new pegfilgrastim biosimilar (NYVEPRIA) to France over a 5-year time horizon. Comparators included existing long-acting and short-acting G-CSFs. The burden of FN was obtained from existing literature. Costs (2021 Euros) included drug acquisition and administration, estimated based on drug dosage in both clinical trial and real-world settings. Scenario analyses were conducted to examine the robustness of key model assumptions. Results: In a total French population of 67.19 million, 79,873 patients were estimated to be treated with G-CSFs annually. The annual number of patients to be treated with NYVEPRIA was estimated to be 1593, 3195, 3674, 3782, and 4052 in years 1 to 5, respectively. Using real-world data, NYVEPRIA resulted in total annual cost savings of e8,620, e868,498, e868,498, e814,102, and e958,952 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of e3,518,669. Using data from clinical trials, NYVEPRIA resulted in total annual cost savings of e14,366, e1,447,496, e1,447,496, e1,356,836, and e1,598,253 over years 1 to 5, respectively, leading to a cumulative 5-year cost savings of e5,864,448. Conclusions: The introduction of a new pegfilgrastim biosimilar (NYVEPRIA) is potentially associated with substantial cost savings for the French healthcare system. [ABSTRACT FROM AUTHOR]

Acute myeloid leukemia (AML) is a rapidly progressive hematological malignancy that is difficult to cure. The prognosis is poor and treatment options are limited in case of relapse. A comprehensive assessment of current disease burden and the clinical efficacy of non-intensive therapies in this population are lacking. We conducted two systematic literature reviews (SLRs). The first SLR (disease burden) included observational studies reporting the incidence and economic and humanistic burden of relapsed/refractory (RR) AML. The second SLR (clinical efficacy) included clinical trials (phase II or later) reporting remission rates (complete remission [CR] or CR with incomplete hematologic recovery [CRi]) and median overall survival (mOS) in patients with RR AML or patients with de novo AML who are ineligible for intensive chemotherapy. For both SLRs, MEDLINE ® /Embase ® were searched from January 1, 2008 to January 31, 2020. Clinical trial registries were also searched for the clinical efficacy SLR. After screening, two independent reviewers determined the eligibility for inclusion in the SLRs based on full-text articles. The disease burden SLR identified 130 observational studies. The median cumulative incidence of relapse was 29.4% after stem cell transplant and 46.8% after induction chemotherapy. Total per-patient-per-month costs were $28,148-$29,322; costs and health care resource use were typically higher for RR versus non-RR patients. Patients with RR AML had worse health-related quality of life (HRQoL) scores than patients with de novo AML across multiple instruments, and lower health utility values versus other AML health states (i.e. newly diagnosed, remission, consolidation, and maintenance therapy). The clinical efficacy SLR identified 50 trials (66 total trial arms). CR/CRi rates and mOS have remained relatively stable and low over the last 2 decades. Across all arms, the median rate of CR/CRi was 18.3% and mOS was 6.2 months. In conclusion, a substantial proportion of patients with AML will develop RR AML, which is associated with significant humanistic and economic burden. Existing treatments offer limited efficacy, highlighting the need for more effective non-intensive treatment options., Competing Interests: Esther Oliva has received honoraria from AbbVie, Alexion, Amgen, Apellis, Celgene, and Novartis, and has served on the speakers’ bureau for Celgene and Novartis. Sarah Ronnebaum, Omer Zaidi, and Dipen Patel are employees of OPEN Health, which received funding by Celgene Corporation to conduct the review. Salem Abi Nehme and Clara Chen are employees and stockholders of Bristol Myers Squibb. Salem Abi Nehme was an employee of Celgene Corporation at the time the research was initiated. Antonio Almeida has received honoraria from Bristol Myers Squibb, and has served as a speaker and consultant for AbbVie and Novartis., (AJBR Copyright © 2021.)