Challenges of Identifying Health Utility Data for Patients With Penta-Refractory Multiple Myeloma to Inform HTA Reimbursement Discussion for Newer Treatment Options

Author(s)

Hibbs R1, Bianco M2, Noble-Longster J1, Stainer L3, Cooper C4, Strickson AJ1
1Tolley Health Economics Ltd., Buxton, Derbyshire, UK, 2Menarini Stemline, Pomezia, RM, Italy, 3Tolley Health Economics Ltd., Buxton, DBY, UK, 4Independent Researcher, London, UK

OBJECTIVES: Selinexor plus dexamethasone (Sd) is the first regimen licensed for penta-refractory multiple myeloma (PR-MM), seeking reimbursement in Europe through Health Technology Assessment (HTA). To inform cost-effectiveness analyses (CEA) for HTA, robust data in a relevant population is required, including health state utility values (HSUVs) representative of health-related quality of life (HRQoL) outcomes of interventions versus standard of care.

METHODS: A systematic review was conducted to identify economic evaluations, costs and resource use, and health utility data, in patients with relapsed and/or refractory MM (RRMM), including PR-MM.

RESULTS: Bibliographic database searching with additional handsearching identified 62 records reporting utility values mostly in earlier lines of RRMM (2L/3L/4L). Additional studies were identified reporting HRQoL data from measures mappable to EQ-5D, however, there were no published studies reporting utility values for a PR-MM population. Two records were identified from the phase 2b STORM trial of Sd, in a penta-exposed population (PE-MM) with ≥50% PR-MM, reporting HRQoL data using FACT-G/MM/MM-TOI for which mapping to EQ-5D-3L/5L is required to elicit utility values. As a potential proxy to PR-MM, a CEA of belantamab mafodotin (belamaf) versus Sd in a 5L+ triple-class refractory (TCR) population applied HSUVs from DREAMM-2 (4L+ triple-class exposed [TCE]) obtained by mapping EORTC-QLQ-C30/MY-20 data to EQ-5D-3L. These mapped utilities were referenced in a second belamaf CEA (5L+ TCE). A third CEA of treatments in heavily pre-treated RRMM applied published HSUVs from MM-003 in double-class refractory participants who had received a median of five prior lines. The pre-progression HSUV reported across all three records was 0.73.

CONCLUSIONS: Although robust data is required for CEA informing HTA of newer interventions, studies only reported HRQoL data specifically for patients with PE-MM and nothing specifically was for PR-MM. Furthermore, reported HRQoL data were derived from tools other than EQ-5D, necessitating mapping exercises to obtain HSUVs for CEA.

Conference/Value in Health Info

2023-11, ISPOR Europe 2023, Copenhagen, Denmark

Value in Health, Volume 26, Issue 11, S2 (December 2023)

Code

HTA18

Topic

Patient-Centered Research, Study Approaches

Topic Subcategory

Health State Utilities, Literature Review & Synthesis, Patient-reported Outcomes & Quality of Life Outcomes

Disease

No Additional Disease & Conditions/Specialized Treatment Areas, Oncology

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