- Cutaneous T-cell lymphoma: 68-year-old man started romidepsin September 22, 2010
- Received 108 doses through May 2016. Total cost to date~ $652,970.
The rate of FDA approval for oncology drugs has been accelerating rapidly in the past 15 years. Sequential therapies beyond second-line therapy are common as more agents become available. Table 2 shows FDA approval for all cancer drugs by decade.
As researchers continue to better understand the many pathways involved with the development and progression of cancer, they are beginning to combine multiple targeted agents to augment response rates, prolong survival, and reduce the potential for resistance. Recent combination regimens approved by the FDA include dabrafenib plus trametinib (January 2014), and ipilimumab plus nivolumab (October 2015), both for the treatment of melanoma. In November 2015, ixazomib was FDA approved to be used in combination with lenalidomide for multiple myeloma. Many more combination regimens are currently in clinical trials, and more combinations are expected to receive FDA approval. It is easy to see how the combination of multiple expensive agents with the prospect of prolonged therapy has the potential to increase the cost of many regimens to well over $100,000 per year.
Maintenance therapy is used to prolong PFS for patients receiving an excellent response to primary therapy. For example, VA costs for maintenance regimens include lenalidomide 10 mg daily: $8,314 for 28 days equals $216,177 for 2 years; bortezomib 1.3 mg/m2 (2.6 mg) q: 2 weeks equals $60,730 for 2 years (includes waste as bortezomib 3.5-mg vials do not a contain preservative and must be discarded within 8 hours of preparation); and rituximab 800 mg q: 2 months equals $47,635 for 2 years.
Until recently, immunotherapy for cancer was limited to melanoma and renal cell carcinoma using interleukin-2 (aldesleukin) and interferon alfa. However, the immergence of new immunotherapies, such as anti-PD-1 and anti-CTLA-4 monoclonal antibodies, have expanded the role of immunotherapy to many other, more common, malignancies, such as lung cancer, breast cancer, prostate cancer, head and neck cancer, and many more.
Most randomized clinical trials study drugs as second- or occasionally third-line therapy. However, many patients continue to be treated beyond the third-line setting, often without evidence-based data to support potential benefit. Patients often place value on treatments unlikely to work so as not to give up hope. These “hopeful gambles,” even with the potential of significant toxicity and decreased quality of life (QOL), are common in cancer treatment.6 In addition, oncologists often overestimate the clinical benefit when considering additional therapy in this setting.7
Influx of New Patients
Outside the VHA setting, the financial burden of cancer treatment has led to an influx of new patients transferring care to the VHA to reduce out-of-pocket expenses. Because private insurance copays for oral agents are increasing, many reaching 20% to 30%, out-of-pocket expenses for medications can reach several thousand dollars per month. Patients often change insurance plans due to changing jobs or to decrease cost, or employers may change plans to save money, which may significantly alter or discontinue coverage. Patients often request that the VA provide medication while continuing to see only their private oncologist. This practice should be discouraged because the VA, without clinical involvement, may supply drugs for inappropriate indications. In addition, VA providers writing prescriptions for medications without personally following patients may be liable for poor outcomes.
VA PBM Services
Prior to 1995, the VA was a much criticized and poorly performing health care system that had experienced significant budget cuts, forcing many veterans to seek care outside the VA. Then beginning in 1995, a remarkable transformation occurred, which modernized and improved the VA into a system that consistently outperforms the private sector in quality of care, patient safety, patient satisfaction, all at a lower cost.8 The story of the VA’s transformation has been well chronicled by Phillip Longman.9
Under the direction of VA Under Secretary for Health Kenneth Kizer, MD, MPH, VA established PBM Clinical Services to develop and maintain the National Drug Formulary, create clinical guidance documents, and manage drug costs and utilization. A recent article by Heron and Geraci examined the functions and role of the VA PBM in controlling oncology drug costs.10 The following is a brief review of several documents and VA PBM responsibilities as reviewed by Heron and Geraci.
VA National Formulary
Prior to the establishment of the VA National Formulary in 1995, each VA maintained its own formulary, which led to extreme variability in drug access across the country. When a patient accessed care at different VAMCs, it was common for the patient’s medications to be changed based on the specific facility formulary. This practice led to many potential problems, such as lack of clinical benefit and potentially increased or new toxicities, and led to extra hospital visits for monitoring and adjustment of medications.
