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

  • Jennifer E. VaughnEmail author
  • Veena Shankaran
  • Roland B. Walter
Health Economics (N Khera, Section Editor)
  • 110 Downloads
Part of the following topical collections:
  1. Topical Collection on Health Economics

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.

Keywords

Acute myeloid leukemia Novel therapeutics Clinical benefit Cost-benefit Health economics 

Notes

Acknowledgments

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.

References

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  • Jennifer E. Vaughn
    • 1
    Email author
  • Veena Shankaran
    • 2
    • 3
    • 4
    • 5
    • 6
  • Roland B. Walter
    • 2
    • 7
    • 8
    • 9
  1. 1.Department of MedicineVirginia Tech Carilion School of MedicineRoanokeUSA
  2. 2.Clinical Research DivisionFred Hutchinson Cancer Research CenterSeattleUSA
  3. 3.Public Health Sciences DivisionFred Hutchinson Cancer Research CenterSeattleUSA
  4. 4.Division of Medical Oncology, Department of MedicineUniversity of Washington School of MedicineSeattleUSA
  5. 5.Department of PharmacyUniversity of Washington School of MedicineSeattleUSA
  6. 6.Department of PharmacySeattle Cancer Care AllianceSeattleUSA
  7. 7.Division of Hematology, Department of MedicineUniversity of Washington School of MedicineSeattleUSA
  8. 8.Department of EpidemiologyUniversity of Washington School of Public HealthSeattleUSA
  9. 9.Department of PathologyUniversity of Washington School of MedicineSeattleUSA

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