Ivosidenib for untreated acute myeloid leukaemia with an IDH1 R132 mutation

As of June 2024, MARA’s assessment finds Ivosidenib’s reimbursement risk concentrated in uncertainty, sensitivity, and broader impacts, with care pathway integration a strength; the current MARA Rating and full rationale are available in the report.

The assessment also weighs comparator selection: whether the drug was tested against the treatment payers actually fund today; a benefit shown against the wrong comparator carries little weight in a reimbursement decision. The strength recorded in care pathway integration carries weight because that domain asks how the drug fits into the way care is organised today; a treatment that demands new infrastructure or displaces an established pathway faces extra scrutiny.

Oncology

This rating sits within MARA’s Oncology coverage, alongside 153 other independently assessed treatments in the same area.

What payers will ask

Is the clinical benefit the kind payers reward? — Clinical effectiveness

The AGILE trial demonstrated significant improvements in event-free survival and overall survival for ivosidenib plus azacitidine compared to azacitidine plus placebo, with hazard ratios of 0.33 and 0.42 respectively. However, the lack of direct comparison with the standard of care (venetoclax plus azacitidine) limits the strength of the evidence.

Does the economic case hold at the expected price? — Cost effectiveness

The cost-effectiveness estimates for ivosidenib plus azacitidine are within the acceptable range for Healthcare resources, with an ICER under £30,000 per QALY gained, indicating marginal cost-effectiveness.

Is there quality-of-life evidence payers weigh? — Quality of life

While the document discusses the potential benefits of ivosidenib plus azacitidine, it does not provide specific data on HRQoL improvements or validated tools measuring these outcomes, leading to a conclusion of minimal or mixed impact.

Does the safety profile hold up for payers? — Safety and Adverse Effects

The safety profile of ivosidenib plus azacitidine is considered very good, with manageable adverse events reported in the AGILE trial, which is comparable to existing therapies.

Was the drug compared against what payers expect? — Comparator Selection

The primary comparator, venetoclax plus azacitidine, was not directly tested against ivosidenib plus azacitidine, leading to reliance on indirect comparisons which introduces uncertainty.

Is the population defined the way payers need it? — Patient Population and Subgroups

The trial population is representative of the intended patient population with untreated AML and IDH1 mutations, although there are some limitations in subgroup analyses.

Does the drug fit how care is delivered and paid for? — Care Pathway Integration

Ivosidenib plus azacitidine can be integrated into existing treatment pathways with minor adjustments, as it is an oral treatment option that is preferable to intravenous therapies.

Are the wider system costs understood? — Resource Use and Cost Implications

The economic model indicates manageable budget impact and resource use, with the potential for cost savings due to reduced hospital stays and transfusion needs.

Would the evidence survive payer scrutiny? — Evidence Quality and Robustness

The evidence is based on a Phase 3 RCT (AGILE trial) with a robust design, although there are some concerns regarding the indirect comparisons and the potential for bias.

How exposed is the case to uncertainty? — Uncertainty, Sensitivity, and Broader Impacts

There are significant uncertainties regarding the long-term outcomes and the assumptions made in the economic model, particularly concerning the cure assumption and the generalizability of results.

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Related Reimbursement Risk Assessments

Public record

The entries below are official decisions and assessments concerning this drug and indication, listed with their dates as a matter of record. The assessment on this page reflects the evidence available as of its date.

20 August 2026 — Germany (G-BA), benefit assessment (orphan reassessment): no additional benefit proven. official record

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