Are we ready to assess the value of cancer drugs?

Cancer drugs have been well known for their high price tag. Cost of newly approved cancer drugs routinely exceeds $10,000 per patient per month. A single new immunotherapy to treat melanoma could cost $1 million per patient per year if used at the higher dose, according to a recent presentation by Leonard Saltz, MD, at the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting.

To drive up the cost of treatment even further, Saltz, also presented the findings of a new study that have shown positive results by combining two different immunotherapies to treat melanoma at a cost of nearly $30,000 per patient per month.

The cost of new cancer drugs is more relevant than ever, as there is an increasing demand for cancer care. Nearly one in three Americans will develop cancer in their lifetime; the National Cancer Institute projects a 30% increase in number of cancer survivors and 26% increase of national expenditure on cancer care, approaching $150 billion by 2020.

The cost of cancer drugs, although not a significant percentage of the total expenditure of cancer care, it is rising to the point that ultimately it will impact patients directly if there is a cost share component of the out-of-pockets. Few studies found that patients experiencing financial distress and hardship are less likely to adhere to the prescribed therapy despite its benefit, according to studies published in the New England Journal of Medicine and Journal of Clinical Oncology.

In addition, a study found that cancer patients are more likely to experience bankruptcy than individuals without cancer, according to a study published in Health Affairs. This is the dilemma cancer patients are challenged with, collectively is what is called, the “financial toxicity” of cancer treatment which is a major concern for patients and their oncologists, according to a study published in Oncology.

Several value assessment tools emerged in response to the escalating drug prices. The focus is not just about the cost, but it’s about the value and quality of cancer care we can afford to deliver to our patients. Defining value in healthcare is complicated and elusive; it depends on the perspectives of the stakeholders. The definition on value of prescription drugs also varied from different organizations (e.g., Institute for Clinical and Economic Review (ICER), ASCO.

Next: The most well-known tools to assess value of cancer drugs

Well-known tools

According to a study published in the New England Journal of Medicine, the most well-known tools to assess value of cancer drugs are the ASCO Value Framework, National Comprehensive Cancer Network (NCCN) Evidence Blocks, Memorial Sloan Kettering Cancer Center (MSKCC) DrugAbacus and European Society of Clinical Oncology (ESMO) Magnitude of Clinical Benefit Scale (MCBS).

The ASCO Value Framework and the NCCN Evidence Blocks have been selected for further analysis because these are the most reputable professional organizations in the oncology community and they are well known for their consensus national guidelines in oncology.

Education on the offerings and limitations of these oncology value tools is the first step to assess value of cancer drugs. It is critical to know when to use the right tool to assess the evidence of a new therapy in order to provide necessary information to make the best treatment decision for individual patient.

The common goal of these tools is to provide healthcare providers and patients the necessary information to make informed decisions on cancer treatment(s). The approach and methodology to assess value of cancer drugs adopted by ASCO and NCCN are different. ASCO Value Framework is a scoring system calculated by the users; while NCCN Evidence Blocks are rated by the NCCN Expert Panels based on their knowledge and experiences, according to an ASCO statement, published in the Journal of Clinical Oncology and NCCN Evidence Blocks FAQ.

Next: Initial version of the value framework

The value framework

ASCO has been very concerned about the rising cost of new cancer therapies for more than a decade.  Part of the ASCO value initiative is to develop a framework that can be used by providers and patients to assess value between treatment options.

ASCO released an initial version of a conceptual value framework in late June 2015; a newly proposed methodology to assess and compare relative clinical benefits, side effects and costs of new cancer treatments that have been studied head-to-head in a randomized clinical trial. Based on these parameters, a health net benefit score (HNB) is generated.

A total point for advanced cancer is 130 points including a bonus point and 100 points in the adjuvant setting. The cost parameter is not part of the total HNB score, according to the framework published in the Journal of Clinical Oncology.

This is ASCO’s first attempt to standardize the value assessment approach to summarize important findings from a comparative randomized clinical trial. The limitation of the framework is that you can only apply the framework for a new cancer drug if it’s compared in a published, randomized clinical trial. The evidence of recently approved breakthrough new cancer drugs is lacking this type of well design randomized trial, particularly in advanced stage cancers and rare cancers.

The NCCN is addressing affordability issue by utilizing visual Evidence Blocks system to compare treatments. The strength of the Evidence Blocks is that in addition to the efficacy, safety and cost elements of regimens used for a specific cancer type, quality and consistency of the evidence are also considered. The users can quickly scan a group of treatment choices accompanied by Evidence Blocks for a cancer type. This requires understanding of the all the elements considered to generate the score.  

By March 2016, eight Evidence Blocks are available for breast, colon, kidney, lung and rectal cancers and multiple myeloma, and chronic myelogenous leukemia (CML) and non-Hodgkin lymphoma (NHL). NCCN plans to complete the entire guideline library by the end of 2017, according to NCCN.

There are opportunities for the organizational pharmacy and therapeutics (P&T) committees to play an important role in utilizing these tools as part of the formulary review process for new cancer drugs.  Application of these tools requires professionals with expertise in oncology to understand the intricacies of the evidence and knowing when to use the right tool based on the evidence. In addition, there are opportunities to implement these tools in the real world setting to test its utility in assisting oncologists and patients in making the best treatment decision choice.

Jennifer T. Chang, PharmD, MPH, is the supervisor of Regional Drug Information Services, Kaiser Permanente, Downey, California. She presented a session entitled “Oncology Value Tools- Are We Ready to Assess Value of Cancer Drugs?” on Wednesday, April 20, at the AMCP Managed Care and Specialty Pharmacy Annual Meeting 2016 in San Francisco.