Cost-effectiveness and drug wastage of immunotherapeutic agents for hematologic malignancies: a systematic review

ABSTRACT

Introduction: Novel immunotherapeutic agents (e.g. monoclonal antibodies, antibody-drug conjugates, bispecific T-cell engagers) as treatment options for hematologic malignancies continue to emerge. These agents have been used as the standard of care in specific disease states and are associated with high costs. Value assessment of these therapies is of critical importance for coverage and reimbursement decision-making.

Areas covered: We identified 15 immunotherapeutic agents through the U.S. FDA approvals for hematologic malignancies until 2018 and systematically reviewed related cost-effectiveness studies. Additionally, we examined whether drug wastage was accounted for in these studies.

Expert opinion: We reviewed 51 studies for 14 identified immunotherapeutic agents that met the inclusion criteria for this systematic review. Three studies were observational-based, one study was model-based and incorporated observational data. The remaining studies were model-based with the majority of the model parameters extracted from randomized control trials (RCTs). Among 43 model-based economic evaluations, 13 studies accounted for drug wastage. Most of the studies showed favorable incremental cost-effectiveness ratios of immunotherapeutic agents-containing regimens when compared with no immunotherapeutic agents-containing regimens. Alemtuzumab, brentuximab vedotin, and daratumumab were not considered cost-effective across all the studies. Further investigations are warranted to establish the value of recent immunotherapeutic agents for hematologic malignancies.

KEYWORDS:

• Hematologic malignancy
• cost-effectiveness
• immunotherapeutic agents
• drug wastage

Article highlights

• More than half of the cost-effectiveness studies (n=33, 65%) were pharmaceutical industry-funded.

• As the first approved immunotherapeutic agents, we found more than half of the studies evaluated the cost-effectiveness of rituximab-included regiments. Studies of cost-effectiveness analyses of rituximab-included regiments were heterogeneous, with different combinations with other medications in the treatment of a wide range of diseases.

• Results of cost-effectiveness analyses of agents other than rituximab were homogeneous. However, the number of studies was limited.

• Observational-based cost-effectiveness analyses using real-world data provide an opportunity to understand the treatment strategy in clinical practice and the value beyond a single regimen, which contributes to better decision-making from a real-world perspective.

• Among 48 model-based economic evaluations, 13 studies accounted for drug wastage, 2 studies made the assumption of no wastage in their analyses. The remaining studies did not mention drug wastage in their studies.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewers disclosure

Peer reviewers on this manuscript have no relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.