Midostaurin / rydapt for treating advanced systemic mastocytosis as of September 2021

This page presents a standardized, committee-reviewed assessment of the reimbursement risk associated with Midostaurin / rydapt for treating advanced systemic mastocytosis as of September 2021, calibrated against historical HTA and payer outcomes across the US, EU4, UK, and Japan using a structured 10-domain evaluation framework.

Updates & Coverage Scope

MARA Ratings reflect the evidence and payer context available as of the stated date. Where material new evidence or payer decisions emerge, MARA may issue a rating action under the Rating Actions & Surveillance Policy.

For market-specific extensions or updated cut dates, request a revised assessment.

What the Complimentary MARA Rating Includes

The Full MARA Assessment provides full 10-domain scoring, weighted determinants, benchmarking against comparable assets, and historical context calibrated to observed HTA outcomes.

Reimbursement Risk Positioning

This assessment positions the asset relative to reimbursement thresholds observed across major HTA bodies. It distinguishes structural drivers of reimbursement risk from variables that are typically sensitive to evidence maturity or price expectations.

The Full MARA Assessment provides weighted scoring across all 10 domains and benchmarks this positioning against historical reimbursement outcomes for comparable evidence profiles.

Selected Risk Drivers

Below is a snapshot of domains that materially influence the MARA Rating. The Full MARA Assessment provides complete 10-domain scoring and the evidence-linked rationale for each domain outcome.

Clinical effectiveness

The clinical effectiveness evidence for midostaurin is derived from two single-arm trials (D2201 and A2213), which do not provide direct comparisons with current treatment options. While the evidence suggests midostaurin may be more effective than existing treatments, the lack of direct comparative data and the uncertainty surrounding the results limit the strength of the evidence. The committee acknowledged that conducting a Phase 3 trial would be challenging due to the rarity of the condition, which further complicates the assessment of clinical effectiveness.

Cost effectiveness

The cost-effectiveness estimates for midostaurin are below the NICE threshold of £50,000 per QALY gained, which is considered acceptable. The committee noted that midostaurin meets the criteria for a life-extending treatment at the end of life, allowing for higher cost-effectiveness estimates. Despite some uncertainties in the clinical evidence, the overall economic model was deemed broadly acceptable for decision making.

Quality of life

Patient and clinical experts reported substantial improvements in quality of life following treatment with midostaurin, with some patients experiencing significant benefits shortly after starting treatment. However, the utility values used in the economic model were derived from the single-arm trials and did not include data from current clinical management, which may not fully capture the quality-of-life benefits. Despite this limitation, the evidence suggests a moderate improvement in HRQoL.

The Full MARA Reimbursement Risk Assessment expands this analysis with weighted scoring, benchmarking depth, and ranking versus hundreds of comparable assets.

Benchmarking & Historical Calibration

This asset is benchmarked against therapeutic-area comparators and a historical dataset of previously assessed products. MARA calibrates rating bands against observed reimbursement outcomes across major HTA bodies.

Scope of the Full MARA Assessment

Designed to support governance and diligence before major commitments in pricing, evidence generation, partnering, or launch planning.

What is the reimbursement risk of Midostaurin / rydapt for treating advanced systemic mastocytosis as of September 2021?

The MARA Rating provides a standardized, committee-reviewed assessment of the reimbursement risk associated with this drug–indication pair, calibrated against historical HTA and payer outcomes across the US, EU4, UK, and Japan.

The licensed report includes structured scoring across 10 payer-relevant domains, evidence-linked rationale, benchmarking against comparable assets, and identification of variables that may influence rating migration.

The complimentary summary provides the rating band, market access score, confidence level, and high-level context. Detailed domain scoring, benchmarking analysis, and full rationale are available in the licensed report.

Related Reimbursement Risk Assessments

Surveillance Status

This rating is subject to defined surveillance under the Rating Actions & Surveillance Policy. Where material new evidence, payer decisions, or policy changes occur, MARA may issue a rating action (affirmation, upgrade, downgrade, or under review) with an as-of date and rationale.

This aligns every report page with your governance system.

Professional Use Notice

MARA Rating is an independent analytical opinion intended for professional use in market access, HTA, and investment contexts. It does not constitute medical, legal, or investment advice.

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