Iptacopan / fabhalta for treating paroxysmal nocturnal haemoglobinuria as of September 2024

This page presents a standardized, committee-reviewed assessment of the reimbursement risk associated with Iptacopan / fabhalta for treating paroxysmal nocturnal haemoglobinuria as of September 2024, 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 trials APPOINT-PNH and APPLY-PNH provide strong evidence of iptacopan’s effectiveness in increasing hemoglobin levels and reducing the need for blood transfusions. In APPOINT-PNH, 92.2% of participants achieved a haematological response at 24 weeks, and 97.4% at 48 weeks. In APPLY-PNH, iptacopan showed a significant increase in hemoglobin compared to C5 inhibitors, with 82.3% achieving an increase of 2 g/dl or more at 24 weeks. However, the indirect comparisons with C5 inhibitors introduce some uncertainty, preventing a higher rating.

Cost effectiveness

The cost-effectiveness estimates for iptacopan are within the range that NICE considers acceptable for Healthcare resources. The committee concluded that the ICERs presented were defensible, although they noted high uncertainty regarding the relative effects compared to C5 inhibitors and pegcetacoplan. The economic model was consistent with previous evaluations, supporting its cost-effectiveness.

Quality of life

The evidence suggests that iptacopan improves health-related quality of life due to its oral administration and better haematological response, leading to less fatigue. Patient experts noted that the oral treatment allows for greater independence and fewer hospital visits, which positively impacts daily life. However, the data on HRQoL improvements is based on treatment-dependent utility values, which introduces some uncertainty.

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 Iptacopan / fabhalta for treating paroxysmal nocturnal haemoglobinuria as of September 2024?

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.

Full Legal Disclaimer and Usage Terms

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