TRANSLATING LONG-TERM RISK REDUCTION WITH ROPEGINTERFERON ALFA-2B INTO PATIENT AND ECONOMIC IMPACT IN POLYCYTHEMIA VERA
Author(s)
Kyle Downey, PharmD1, Edward Jefford, PhD1, Emi Naslazi, MSc2, Noemi Hummel, PhD3.
1PharmaEssentia USA, Burlington, MA, USA, 2Certara Netherlands, Breda, Netherlands, 3Certara Germany GmbH, Lörrach, Germany.
1PharmaEssentia USA, Burlington, MA, USA, 2Certara Netherlands, Breda, Netherlands, 3Certara Germany GmbH, Lörrach, Germany.
OBJECTIVES: Polycythemia vera (PV) is a chronic myeloproliferative neoplasm characterized by erythrocytosis and increased risk of thromboembolic events (TE), myelofibrosis (MF), and acute myeloid leukemia (AML). Conventional management includes cytoreductive therapies (hydroxyurea (HU) or phlebotomy), while ropeginterferon alfa‑2b has demonstrated disease‑modifying potential. This analysis quantified the absolute risk reduction (ARR) and number needed to treat (NNT) for ropeginterferon alfa‑2b versus HU in preventing TEs, MF, and AML in PV, versus phlebotomy for TEs in low‑risk PV and translated these into potential cost impact.
METHODS: Six-year cumulative event rates for TE, MF, and AML with ropeginterferon alfa‑2b and HU were obtained from long‑term PROUD‑PV, CONTINUATION‑PV, and related publications. For low‑risk PV, ARR and NNT for TE were derived from randomized data of the Low‑PV trial. ARR was calculated as the difference in event rates between the control group and ropeginterferon alfa‑2b; NNT as 1/ARR. Published U.S. cost estimates for PV-related TE, MF, and AML were retrieved to understand potential complication‑related cost avoidance.
RESULTS: Event rates per 100 patient‑years for HU versus ropeginterferon alfa‑2b, were approximately 1.247%vs 1.002% (TE), 0.499% vs 0.200% (MF), and 0.499% vs 0.000% (AML). Corresponding ARRs (95% confidence interval) were 0.245 (-1.149, 1.639), 0.298 (-0.495, 1.092), and 0.499 (-0.191, 1.188), yielding NNTs of 408, 335, and 201, respectively. In low-risk PV, ARR for TE versus phlebotomy was 0.962 (-0.914, 2.837), NNT was 104. Avoided costs per prevented event were approximately USD 45,040 (TE), 105,280 (MF), and 280,236 (AML).
CONCLUSIONS: Ropeginterferon alfa‑2b was associated with long‑term reductions in TE, MF, and AML versus HU and reduced TE risk versus phlebotomy in low-risk PV. When linked to complication costs, these reductions suggest that lower rates of high‑cost events may offset a meaningful proportion of PV complication‑related spending, particularly over extended time horizons, supporting sustained use of ropeginterferon alfa‑2b especially in high‑risk PV populations.
METHODS: Six-year cumulative event rates for TE, MF, and AML with ropeginterferon alfa‑2b and HU were obtained from long‑term PROUD‑PV, CONTINUATION‑PV, and related publications. For low‑risk PV, ARR and NNT for TE were derived from randomized data of the Low‑PV trial. ARR was calculated as the difference in event rates between the control group and ropeginterferon alfa‑2b; NNT as 1/ARR. Published U.S. cost estimates for PV-related TE, MF, and AML were retrieved to understand potential complication‑related cost avoidance.
RESULTS: Event rates per 100 patient‑years for HU versus ropeginterferon alfa‑2b, were approximately 1.247%vs 1.002% (TE), 0.499% vs 0.200% (MF), and 0.499% vs 0.000% (AML). Corresponding ARRs (95% confidence interval) were 0.245 (-1.149, 1.639), 0.298 (-0.495, 1.092), and 0.499 (-0.191, 1.188), yielding NNTs of 408, 335, and 201, respectively. In low-risk PV, ARR for TE versus phlebotomy was 0.962 (-0.914, 2.837), NNT was 104. Avoided costs per prevented event were approximately USD 45,040 (TE), 105,280 (MF), and 280,236 (AML).
CONCLUSIONS: Ropeginterferon alfa‑2b was associated with long‑term reductions in TE, MF, and AML versus HU and reduced TE risk versus phlebotomy in low-risk PV. When linked to complication costs, these reductions suggest that lower rates of high‑cost events may offset a meaningful proportion of PV complication‑related spending, particularly over extended time horizons, supporting sustained use of ropeginterferon alfa‑2b especially in high‑risk PV populations.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
CO77
Topic
Clinical Outcomes
Disease
SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)