A Strategic Evidence Generation Plan for Enasidenib in Refractory or Relapsed IDH2-Positive Acute Myeloid Leukemia

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Publication Date

7-22-2025

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Presentation: 26:42

Abstract

Enasidenib is an oral, targeted IDH-2 inhibitor indicated for adults with relapsed or refractory acute myeloid leukemia (AML). While this therapy represents a meaningful treatment target, current evidence highlights the persistent unmet need including modest complete response rates, limited survival data, long-term outcomes or quality of life. The goal of this capstone was to assess the current evidence landscape and identify gaps to inform a strategic evidence generation for Enasidenib from a U.S. HEOR perspective. A PICOS-based framework was utilized to guide the literature review where eight relevant studies were identified. The studies demonstrated that most patients were older and unfit for chemotherapy or allogeneic stem cell transplant, with complete response rate approximately 20% and a median survival of 9.3 months. Key literature gaps included limited reporting of health-related quality of life (HRQoL), lack of head-to-head comparative studies and a scarcity of patient reported outcomes studies. To address the limitation, two supportive studies were proposed: a burden of illness study to quantify clinical, economic and humanistic burden, and a healthcare resource utilization (HCRU) study to examine real-world treatment costs and patterns. These studies will complement additional tools such as an AMCP dossier, budget impact model and real-world studies to support payer and stakeholder discussions. A post-launch HEOR framework was created to align with FDA timelines, incorporating tools such as a AMCP dossier pre/post approval, budget impact model and real-world evidence studies on treatment patterns and outcomes. Overall, this the strategic framework was designed to enhance Enasidenib’s value communication, inform payer discussions and ultimately improve outcomes for high-risk patients with R/R IDH-2 mutant AML.

Language

English

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