Trends in Clinical Benefits and Costs of Novel Therapeutics in AML: at What Price Does Progress Come?
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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.
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.
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.
KeywordsAcute myeloid leukemia Novel therapeutics Clinical benefit Cost-benefit Health economics
R.B.W. is a Leukemia & Lymphoma Society Scholar in Clinical Research.
Compliance with Ethical Standards
Conflict of Interest
The authors declare that they have no conflict of interest.
Human and Animal Rights and Informed Consent
This article does not contain any studies with human or animal subjects performed by any of the authors.
Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance
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