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Published in: Current Hematologic Malignancy Reports 3/2019

01-06-2019 | Acute Myeloid Leukemia | Health Economics (N Khera, Section Editor)

Trends in Clinical Benefits and Costs of Novel Therapeutics in AML: at What Price Does Progress Come?

Authors: Jennifer E. Vaughn, Veena Shankaran, Roland B. Walter

Published in: Current Hematologic Malignancy Reports | Issue 3/2019

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Abstract

Purpose of Review

Since 2017, eight novel agents have been approved for the treatment of acute myeloid leukemia (AML) in the USA. Here, we review the clinical benefits and costs associated with these drugs.

Recent Findings

For some of the newly-approved drugs, clinical benefit has been documented in randomized trials. Others received accelerated approval based on surrogate endpoints in early phase trials. All, however, carry significant costs and toxicities. Cost-effectiveness analyses are so far only available for midostaurin, CPX-351, and gemtuzumab ozogamicin.

Summary

Recently approved drugs for AML have varying levels of evidence for clinical effectiveness and because of associated high costs may further increase the overall economic burden of AML care. This issue is complex and whether novel AML drugs will cost-effective will depend on multiple factors, including their ability to improve survival and quality of life while simultaneously reducing the costs of healthcare resource utilization.
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Metadata
Title
Trends in Clinical Benefits and Costs of Novel Therapeutics in AML: at What Price Does Progress Come?
Authors
Jennifer E. Vaughn
Veena Shankaran
Roland B. Walter
Publication date
01-06-2019
Publisher
Springer US
Published in
Current Hematologic Malignancy Reports / Issue 3/2019
Print ISSN: 1558-8211
Electronic ISSN: 1558-822X
DOI
https://doi.org/10.1007/s11899-019-00510-2

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