Ensuring Greater Acceptance of Minimal Residual Disease Negativity in Health Technology Assessments in Multiple Myeloma: Key Barriers and Opportunities

Author(s)

AIKATERINI FAMELI, MBA, PhD.
Global Oncology Policy Lead, GSK, LONDON, United Kingdom.
OBJECTIVES: In multiple myeloma (MM), extending overall survival (OS) is considered the key outcome, but demonstrating OS is becoming challenging as improved treatments extended time to OS and as cycling through multiple lines dilutes the OS effect of initial therapy. Further, OS does not measure the impact of treatment burden on health-related quality of life. This places importance on complementing OS with alternative endpoints. MRD negativity may support health technology assessments (HTAs) in MM, as some studies show it to be prognostic of OS and as it can be captured within 1 year. But barriers to its acceptance by payers exist. This study aims to identify these barriers and describe actions to support acceptance.
METHODS: Stakeholder perceptions of MRD negativity are assessed via 88 interviews with physicians, patient advocacy groups and former payers in Canada, Japan, UK, France, Germany, Italy, 4 roundtables and a literature review.
RESULTS: Stakeholder feedback confirms OS benefit as the primary value driver in MM, but patients and clinicians express the need for earlier measures of treatment benefit, such as reduction of morbidity and of treatment side effects. MRD negativity is considered to have potential as an OS surrogate and as a standalone measure of delay to progression. But lacking validation of OS correlation and inconsistent measurement methodologies limit payer acceptance. Actions to encourage payer acceptance of MRD negativity include: • Data generation to validate surrogacy through meta-analysis of RCTs • Establishing MRD in clinical decision-making through MRD-adaptive trials • Standardising data collection methodologies
CONCLUSIONS: With increased evidence of surrogacy and standardisation of methodologies, MRD negativity has potential to augment OS as it captures OS benefit earlier and measures broader patient benefits. Cross-stakeholder actions are required to overcome payer concerns over its relevance and validity and to ensure that HTA decisions are based on a holistic view of treatment benefit.

Conference/Value in Health Info

2025-11, ISPOR Europe 2025, Glasgow, Scotland

Value in Health, Volume 28, Issue S2

Code

HTA125

Topic

Health Technology Assessment

Topic Subcategory

Value Frameworks & Dossier Format

Disease

Oncology, Rare & Orphan Diseases

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