Outcomes essential for adopting new­leukemia therapies

December 1, 2010

The outcomes associated with treating patients with chronic myelogenous leukemia improved dramatically when FDA approved imatinab. Subsequently, newer agents in the same therapeutic class have been approved, and early clinical studies suggest that they may become the new standard of care.

Key Points

The outcomes associated with treating patients with chronic myelogenous leukemia (CML) improved dramatically when FDA approved imatinab. Subsequently, newer agents in the same therapeutic class have been approved, and early clinical studies suggest that they may become the new standard of care.

Improved utilization of practice guidelines for the leukemias will be crucial. Coupled with this will be benefit design structures to optimize the value obtained for the anticipated increased costs of these new options. The budgetary impact of these new agents may be mitigated by advances in predictive molecular markers, which could have a dramatic effect on how we can pay for these promising new therapies.

A host of new compounds to treat leukemia-many with novel mechanisms of action, such as the tyrosine kinase inhibitor (TKI)-independent cetaxine class of protein synthesis-inhibitors-are currently in the pipeline. However, their effect on leukemia therapy ultimately is dependent on their ability to demonstrate meaningful, patient-relevant outcomes and to differentiate themselves from the current treatments.

Clearly, the cost trend for novel chemotherapeutics is steeply increasing. Most payers will not intervene in the chemotherapeutic decision process, with 2 exceptions: (1) when comparative effectiveness trials, clinical evidence, or a benchmark such as NCCN guidelines could be used to require one therapy over another, and (2) when molecular markers (genomic, proteomic, or metabolomic) predict response or non-response, justifying access limitations. The expanded use of predictive molecular markers is especially interesting to payers because of the opportunity to deploy the optimal drug therapy first-line. MD Anderson's phase 2 BATTLE trial used an innovative statistical model to match 4 drugs to specific biomarkers in patients with non–small cell lung cancer.1 The trial results showed enhanced disease control at 8 weeks, which recent research has found to be a good indicator of overall survival.

Commonly, cancer drugs are treated as a protected class in terms of benefit design. However, given the number of agents that could become available in the future, it would not be unreasonable to select preferred agents and negotiate rebate contracts with the manufacturers. Furthermore, innovative health plans have developed multi-tiered specialty drug benefits to leverage cost-sharing as a mechanism for driving patient and physician choice. The abundance of drug options could also foster the codification of performance contracts, such as the one negotiated between Johnson & Johnson and the National Health Service for Velcade.2 F

-John Fox, MD, medical director, Priority Health, Grand Rapids, MI

Copyright 2010 by The Medical Pipeline, LLC. All rights reserved. For more information about The Medical Pipeline, please visit http://www.tmp-pipeline.com/.

REFERENCES

1. Kim ES, Herbst RS, Lee JJ, et al. The BATTLE trial (Biomarker-integrated Approaches of Targeted Therapy for Lung Cancer Elimination): Personalizing therapy for lung cancer. Program and abstracts of the AACR 101st Annual Meeting; April 17-21, 2010; Washington, DC; Abstract LB-1. Available at: http://www.abstractsonline.com/Plan/ViewAbstract.aspx?sKey=b21e246a-e09a-4c90-9c59-874f7139d921&cKey=381a62d2-5602-443c-83cc-f9a828f19e1b&mKey=%7b0591FA3B-AFEF-49D2-8E65-55F41EE8117E%7d. Accessed July 11, 2010.

2. Johnson & Johnson Division proposes money-back guarantee for cancer medication in agreement with British National Health Service. Medical News Today: July 18, 2007. Available at: http://www.medicalnewstoday.com/articles/77030.php. Accessed July 15, 2010.