Related Experiment Video
Updated: Nov 11, 2025
An Organotypic High Throughput System for Characterization of Drug Sensitivity of Primary Multiple Myeloma Cells
Published on: July 15, 2015
Cost-effectiveness of Novel Treatment Sequences for Transplant-Ineligible Patients With Multiple Myeloma
Hedwig M Blommestein1, Margreet G Franken2, Chrissy H Y van Beurden-Tan3
1Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands.
Importance:
Although the number of treatments for elderly patients with non-transplant-eligible (NTE) multiple myeloma (MM) has increased substantially, evidence is lacking on the clinical effectiveness and cost-effectiveness of novel treatment sequences.
Objective:
To determine the optimal sequence of treatment for patients with NTE MM from the perspective of the patient, physician, and society.
Design, Setting, And Participants:
Using data from a Dutch observational registry, this economic evaluation combined evidence from network meta-analyses in a patient-level simulation model and modeled time-to-event and types of events from a hospital perspective with a lifetime horizon. Data analysis was performed from June 2019 to September 2020.
Interventions:
Thirty treatment sequences, including up to 3 lines of therapy, were compared with bortezomib-melphalan-prednisone (VMP)-lenalidomide-dexamethasone (LenDex)-pomalidomide-dexamethasone (PomDex).
Main Outcomes And Measures:
The primary outcomes of the model were overall survival (OS), quality-adjusted life-years (QALYs), costs, and cost-effectiveness.
Results:
Sequences starting with daratumumab-VMP (second line: carfilzomib-lenalidomide-dexamethasone or elotuzumab-lenalidomide-dexamethasone) or bortezomib-melphalan-prednisone-thalidomide-maintenance bortezomib-thalidomide (VMPT-VT) (second line: daratumumab-lenalidomide-dexamethasone) had the largest expected OS (7.5 years), which is 3.5 additional life-years compared with VMP-LenDex-PomDex. Total costs per patient for these sequences ranged between $786 024 and $1 085 794. The sequence VMPT-VT-carfilzomib-lenalidomide-dexamethasone-panobinostat-bortezomib-dexamethasone had the most favorable cost-effectiveness ratio ($98 585 per life-year gained and $132 707 per QALY gained vs VMP-LenDex-PomDex).
Conclusions And Relevance:
These findings suggest that sequences including novel treatments were highly effective, but the cost-effectiveness ratios were above currently accepted willingness-to-pay thresholds. Treating MM with novel agents necessitates either a large increase in budget or a substantial reduction of drug costs by price negotiations, and these findings can support these reimbursement decisions and price negotiations.
Insights
Novel treatment sequences for non-transplant-eligible multiple myeloma (NTE MM) significantly improve overall survival but exceed cost-effectiveness thresholds. These findings support budget increase or drug price negotiations for novel agents in NTE MM.
Area of Science:
- Hematology
- Oncology
- Health Economics
Background:
- Elderly patients with non-transplant-eligible multiple myeloma (NTE MM) have increasing treatment options.
- However, evidence on the clinical and cost-effectiveness of novel treatment sequences is limited.
Purpose of the Study:
- To determine the optimal treatment sequence for NTE MM patients.
- Considered perspectives of patients, physicians, and society.
Main Methods:
- Economic evaluation using a Dutch observational registry.
- Patient-level simulation model combined network meta-analyses data.
- Modeled time-to-event and event types over a lifetime horizon.
Main Results:
- Sequences with daratumumab-VMP or VMPT-VT showed the longest overall survival (7.5 years), adding 3.5 years compared to VMP-LenDex-PomDex.
- Total costs ranged from $786,024 to $1,085,794 per patient.
- The VMPT-VT-carfilzomib-lenalidomide-dexamethasone-panobinostat-bortezomib-dexamethasone sequence offered the best cost-effectiveness ratio.
Conclusions:
- Novel treatment sequences for NTE MM are highly effective but costly.
- Cost-effectiveness ratios exceed willingness-to-pay thresholds.
- Findings can inform budget decisions and drug price negotiations for novel MM therapies.
More Related Videos
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
02:56Treatment Resistant Cancers
02:50Combination Therapies and Personalized Medicine
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...