The management of complex oncology drugs in pharmacy and in medical benefits presents unique challenges for all parties who seek cost-effective, positive clinical outcomes for patients with cancer. New therapies are offering the exciting prospect of improved outcomes, prolonged life, and, in some cases, a cure for specific diseases. Targeted oncolytics and pharmacogenomics, which carry the promise of improved likelihood of successful treatment, have become welcome additions to the current standards of care. The concept of cancer as a chronic disease is becoming accepted in pharmacy oncology management. Targeted therapies are now standard treatments for multiple myeloma (MM), non-Hodgkin lymphoma (NHL), and chronic myelogenous leukemia (CML). Recent trials in MM have used time to progression and progression-free survival as primary end points (as recommended by the International Myeloma Workshop Consensus Panel 1). The use of different end points in clinical trials for the same disease, however, has complicated the analysis and evaluation process for the pharmacy and therapeutics (P & T) committees of health plans in comparing competing therapies to select the most efficacious and cost-effective treatment options for their members. The ability to diagnose cancer earlier in the disease process and to begin life-saving treatments sooner place additional strain on pharmacy managers to maximize the value from all therapies in the treatment algorithm of a particular cancer type. The tyrosine kinase inhibitors (TKIs) have revolutionized the treatment of CML by extending survival. It is now possible to achieve long-term success with these agents if treatment is initiated early enough in the course of disease, and a positive early response can be a good predictor of long-term survival. As clinical studies are able to demonstrate clinically meaningful differentiations between first- and second- generation TKIs, health plans can consider promoting specific treatment pathways to maximize cost-savings and outcomes. NHL affects a diverse population of patients with various disease subtypes that require careful diagnosis to be matched with specific drug treatment protocols. The International Working Group has attempted to define appropriate clinical trial end points and response criteria to effectively differentiate treatments. This process is critical for managed care plans to manage specific therapies effectively and control costs in the treatment of NHL and other cancer types. It is clear that in the near-term, overall survival will remain the gold standard for P & T committees in their assessment of drug efficacy. However, other end points will continue to be assessed as clinical trial results become available. The development of pharmaceuticals with biomarkers can increasingly give providers and health plans the confidence that patients will have a greater likelihood of response. This new trend also has the potential to decrease waste and the risk of untoward side effects in patients who are not good candidates for a particular treatment. As health plans progress in the management of oncology drugs, the ability to target patients, predict outcomes, and reduce costs will drive the most successful programs.
