Jennifer E Vaughn1, Veena Shankaran2,3,4,5,6, Roland B Walter2,7,8,9. 1. Department of Medicine, Virginia Tech Carilion School of Medicine, 2 Riverside Dr., Roanoke, VA, 24016, USA. jvaughn@vtc.vt.edu. 2. Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA. 3. Public Health Sciences Division, Fred Hutchinson Cancer Research Center, Seattle, WA, USA. 4. Division of Medical Oncology, Department of Medicine, University of Washington School of Medicine, Seattle, WA, USA. 5. Department of Pharmacy, University of Washington School of Medicine, Seattle, WA, USA. 6. Department of Pharmacy, Seattle Cancer Care Alliance, Seattle, WA, USA. 7. Division of Hematology, Department of Medicine, University of Washington School of Medicine, Seattle, WA, USA. 8. Department of Epidemiology, University of Washington School of Public Health, Seattle, WA, USA. 9. Department of Pathology, University of Washington School of Medicine, Seattle, WA, USA.
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. 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.
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. 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.
Authors: Eric J Feldman; Jeffrey E Lancet; Jonathan E Kolitz; Ellen K Ritchie; Gail J Roboz; Alan F List; Steven L Allen; Ekatherine Asatiani; Lawrence D Mayer; Christine Swenson; Arthur C Louie Journal: J Clin Oncol Date: 2011-01-31 Impact factor: 44.544
Authors: A-L Taksin; O Legrand; E Raffoux; T de Revel; X Thomas; N Contentin; R Bouabdallah; C Pautas; P Turlure; O Reman; C Gardin; B Varet; S de Botton; F Pousset; H Farhat; S Chevret; H Dombret; S Castaigne Journal: Leukemia Date: 2006-10-19 Impact factor: 11.528
Authors: Annemieke Leunis; Hedwig M Blommestein; Peter C Huijgens; Nicole M A Blijlevens; Mojca Jongen-Lavrencic; Carin A Uyl-de Groot Journal: Leuk Res Date: 2012-10-12 Impact factor: 3.156
Authors: Stacie B Dusetzina; Aaron N Winn; Gregory A Abel; Haiden A Huskamp; Nancy L Keating Journal: J Clin Oncol Date: 2013-12-23 Impact factor: 44.544
Authors: Alberto Redaelli; Marc F Botteman; Jennifer M Stephens; Suzanne Brandt; Chris L Pashos Journal: Cancer Treat Rev Date: 2004-05 Impact factor: 12.111
Authors: Carla Mamolo; Verna Welch; Roland B Walter; Joseph C Cappelleri; James Brockbank; Matthew Cawson; Chris Knight; Michele Wilson Journal: Pharmacoeconomics Date: 2020-11-25 Impact factor: 4.981
Authors: Naval Daver; Andrew H Wei; Daniel A Pollyea; Amir T Fathi; Paresh Vyas; Courtney D DiNardo Journal: Blood Cancer J Date: 2020-10-30 Impact factor: 11.037