Quantifying the Value of Introducing an Oral Drug Delivery Option for Edaravone: A Review of Analyses Evaluating the Economic Impact of Oral versus Intravenous Formulations

Abstract

Background

Drug formulation and route of administration can have an impact on not only patients’ quality of life and disease outcomes but also costs of care. It is essential for decision makers to use appropriate economic modeling methods to guide drug coverage policies and to support patients’ decision-making.

Purpose

To illustrate key cost considerations for decision makers in economic evaluation of innovative oral formulations as alternatives to intravenous medication.

Materials and Methods

A structured literature review was conducted using the PubMed database to examine methods used for quantifying the economic impact of introducing a new oral pharmaceutical formulation as an alternative to intravenous medication. To illustrate the methods described in this review, a cost-minimization analysis was conducted to quantify the impact of introducing an oral formulation of a medication originally developed as an intravenous treatment for amyotrophic lateral sclerosis.

Results

We identified 14 published evaluations of oral and intravenous formulations from 10 countries across a variety of disease areas. The identified studies used cost-effectiveness (n=10), cost-minimization (n=2), and cost-calculation (n=2) modeling approaches. All but one (13/14) reported outcomes from payers’ perspective, while societal perspectives were also incorporated in 3 of the reviewed evaluations. One study estimated costs from a public hospital’s perspective. Only a subset of the identified studies accounted for the effects of safety (n=6) or efficacy (n=8) differences on treatment costs when estimating the costs of a formulation choice. Many studies that omitted these aspects did not include rationales for their decisions.

Conclusion

We found significant design variations in published models that estimated the impact of an additional formulation option on the treatment costs to payers and the society. Models need to be accompanied with clear descriptions on rationales for their time horizons and assumptions on how different formulations may affect healthcare costs from the selected perspectives.

Keywords:

• cost-effectiveness analysis
• cost-minimization
• decision makers
• formulation comparison
• amyotrophic lateral sclerosis

Abbreviations

5-FU, 5-fluorouracil; AE, adverse event; ALS, amyotrophic lateral sclerosis; BIA, budget-impact analysis; CEA, cost-effectiveness analysis; CMA, cost-minimization analysis; FDA, Food and Drug Administration; GI, gastrointestinal; HCRU, healthcare resource use; ICER, incremental cost-effectiveness ratio; IM, intramuscular; IV, intravenous; LV, leucovorin; MRSA, methicillin-resistant staphylococcus aureus; ND-CKD, non-dialysis chronic kidney disease; NHS, National Health Service; PICC, peripherally inserted central catheter; PPI, proton pump inhibitor; PRISMA, Preferred Reporting Items for Systematic Reviews and Meta-Analyses; SC, subcutaneous; US, United States; UK, United Kingdom.

Data Sharing Statement

Only published data were used in this study; all references reviewed are presented in Table 1. All sources for model inputs are reported in Supplementary Table S-1.

Acknowledgments

The authors wish to thank Steven Apple of Mitsubishi Tanabe Pharma America for providing expert knowledge in IV edaravone administrations and John Forbes of RTI Health Solutions for providing medical editing support.

Author Contributions

All authors made substantial contributions to conception and design, acquisition of data, or analysis and interpretation of data; took part in drafting the article or revising it critically for important intellectual content; agreed to submit to the current journal; gave final approval of the version to be published; and agree to be accountable for all aspects of the work.

Disclosure

Melissa Hagan and Malgorzata Ciepielewska have received personal compensation for serving as full-time employees of Mitsubishi Tanabe Pharma America, Inc. Naoko Ronquest, Kyle Paret, and Aaron Lucas are employees of RTI Health Solutions, which received consultancy fees from Mitsubishi Tanabe Pharma America, Inc. to conduct this study. The authors report no other conflicts of interest in this work.

Additional information

Funding

This manuscript was supported by consultancy fees from Mitsubishi Tanabe Pharma America, Inc.