New Tools from NCCN and ASCO Address Value

By Kathy Boltz, PhD

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Editor’s Note: As we went to press with this article, NCCN was just unveiling their first NCCN Evidence Blocks™ for Chronic Myelogenous Leukemia (CML) and Multiple Myeloma (MM) at a press conference on Oct. 16 in San Francisco. OBR will keep you updated on NCCN's new value initiative with daily news and feature articles throughout 2015 and into 2016.  

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As prices for cancer therapies continue to rise, the value offered by a treatment is coming under increasing scrutiny, and professional organizations are responding by including value in their assessments of treatments. Historically, the National Comprehensive Cancer Network (NCCN) has asked its panels not to consider cost when assembling NCCN guidelines; nonetheless the national organization has added the new measure of affordability in their new Evidence Blocks, which are rolling out in October.

Cancer drug prices have escalated by an average of $8,500 a year over the last 15 years, from a cost of each additional year lived of $54,000 in 1995 to a whopping $207,000 in 2013, after adjusting for inflation.1 The median price of a month of therapy is currently running about $10,000, with the costs of some treatments running over $100,000 for a course of treatment.

As costs for cancer care become increasingly stratospheric, financial toxicity is an increasing concern for patients. Along with NCCN’s Evidence Blocks, ASCO has published their Value Framework, which assesses the value of new cancer therapies based on treatment benefits, toxicities, and costs.2 And, overseas, the European Society for Medical Oncology (ESMO) has also published a scale to evaluate the effectiveness of cancer medicines.3

NCCN Evidence Blocks

The NCCN Evidence Blocks represent NCCN’s efforts to better explain why specific recommendations were made in their guidelines, which shape reimbursement policies for many payers. In an interview, Robert Carlson, MD, CEO of NCCN, explained that they came up with five measures that they thought were important to help explain the rationale for a specific recommendation. The five measures are: efficacy, safety, quality, consistency of data that supports the recommendation, and affordability. (Figure 1.)

He reiterated that NCCN panels are specifically told not to consider cost when they are putting the guidelines together.

“The affordability measure is actually an add-on to the guideline. It did not exist before and is not used for the panel to make specific recommendations,” he said.

NCCN is planning to add the affordability measure to all of their guidelines. First will be systemic therapy, starting with chronic myelogenous leukemia and multiple myeloma, and then drugs and biologic agents. After those parts of the guidelines are populated, NCCN will be expanding to include radiation oncology, surgical oncology, and diagnostic tests.

Dr. Carlson explained that the Evidence Blocks add transparency to NCCN’s decision-making process. “It especially adds transparency for patients. While payers know a great deal about what they’re paying for a treatment and most physicians have a general idea of the cost of a treatment, the patients are the people who are really out of the loop,” he said.

New Tools from NCCN and ASCO Address Value (continued)

NCCN’s Perspective on Affordability

The NCCN affordability measure includes the whole package, meaning drug acquisition costs, monitoring costs, administration costs, supportive therapy costs such as antiemetics or growth factors, the site of administration (inpatient or outpatient), and also anticipated costs associated with hospitalization for toxicity. However, it’s a rare patient who will actually be responsible for all of these costs.

The affordability piece is the pharmaceutical industry’s major concern about the NCCN Evidence Blocks, according to Dr. Carlson. Industry’s question is, “Affordable to whom?”

“So, industry’s concern is that the Evidence Blocks will begin to tell us what the cost is to individual patients, but we are extending costs to the entire system, including those that would routinely and normally be covered by a payer. Industry is trying to come up with financial concerns to the individual patient, which would typically include their copay or deductibility. NCCN is looking at system affordability rather than individual patient affordability,” said Dr. Carlson.

Though the NCCN Evidence Blocks describe a system of affordability, which is more than a typical patient’s piece of the costs, if an Evidence Block suggests that a therapy is extremely expensive, Dr. Carlson suggested that that information may be a signal to the patient that they need to better understand what costs they might be responsible for. The patient may want to work with their payer or financial counselor in their oncologist’s office to better understand how much of those payments would be their responsibility.

Dr. Carlson said the Evidence Blocks are meant to aid in framing discussions by providing measures that individual patients and their physicians may wish to consider in their decision making.

ASCO Value Framework: Offers Another Tool for Physician-Patient Discussions

The American Society of Clinical Oncology (ASCO) Value Framework was published earlier this summer.1 (Figure 2.) The Value Framework has been developed “to assist the physician and patient in shared decision making as they work toward defining value and identifying an appropriate intervention for that individual patient.”

The ASCO Value Framework defines value as consisting of clinical benefit, side effects, and cost. Like NCCN, ASCO is now including cost into the discussion of treatment choices.

The Value Framework is built around comparative clinical trials. The Framework considers high-quality evidence, meaning prospective, randomized, controlled trials; this evidence is used to assess benefit (i.e., overall survival, progression-free survival, disease-free survival) and toxicity. Two distinct indications are considered by the Framework: advanced or metastatic disease, and the adjuvant setting, meaning so-called “curative” treatment.

The ASCO Value Framework is standardized information for doctors and patients, but it is not meant to be a ranking or calculator for individual drugs, explained Lowell E. Schnipper, MD, chair of ASCO’s Value in Cancer Care Task Force and Clinical Director of Beth Israel Deaconess Medical Center in Boston. In a press conference, he described the Value Framework as trying to combine complex variables into a single score.

The Value Framework determines a net health benefit, which includes benefit and toxicity separately from cost. The net health benefit is displayed as a score based on clinical benefit and toxicity, with bonus points awarded for palliation of symptoms and treatment-free intervals. (Table 1.) The maximum score for net health benefit is 130 points for advanced cancer treatments and 100 points for adjuvant cancer treatments. The score gives a premium to high-efficacy measures and to improving side effects.

Cost in the Value Framework considers only the cost of a drug. Though there are many other aspects to cost, the Value Framework only focuses on drug acquisition cost and the cost to the patient. Dr. Schnipper explained that quality of life and patient-reported outcomes were not included because of a lack of available data and not because of a lack of importance. Also, the ASCO Value Framework does not address chronic, low-grade toxicity.

The ASCO Value Framework is a tool, and not a substitute for a physician’s judgment. The goal is to help weigh options and not limit options. In the future, ASCO anticipates having software that is curated with many clinical trials to allow patients to compare treatment options.

NCCN Evidence Blocks and ASCO Value Framework are Complementary

“The ASCO Value Framework and our Evidence Blocks initiative are very complementary. They are very different, but they actually provide complementary information,” said Dr. Carlson. The affordability component of the NCCN Evidence Blocks includes the whole package of costs associated with a therapy, while the ASCO Value Framework considers only the cost of acquiring a drug and its cost to the patient.

“The ASCO Value Framework requires that there be a randomized trial, and it compares two therapies in a very objective manner using a fixed methodology and fixed formula for coming up with the comparative benefit number. The NCCN Evidence Blocks do not require the presence of a randomized trial, and so they allow comparisons across multiple different regimens that the [Framework], as currently used, will not allow. Our Evidence Blocks initiative does not have a fixed formula; rather, we provide the individual components and the measures of the evidence, and the patient can develop their own value formula, if you will. So, ours is more of a discussion. The ASCO Value Framework comes up with a specific conclusion,” explained Dr. Carlson.

ESMO Scale: Focusing on Survival and Quality of Life

The ESMO scale aims to highlight treatments that bring substantial improvements to the duration of survival or quality of life of cancer patients.3 Documenting these improvements is critical in the European environment, where single-payer programs require therapies to quantitate their value for approval for reimbursement. ESMO wants the scale to be used for accelerated reimbursement evaluation. In the ESMO scale, cure takes precedence over deferring death and direct endpoints, such as survival and quality of life take precedence over surrogates like progression-free survival or response rate. Also, disease-free survival is a more valid surrogate than progression-free survival or response rate. Further, secondary outcome data may influence how evidence of benefit from surrogate outcomes is interpreted.

Next, Oncologists Speak Out on Cost and Value

Value will continue to be a heated discussion, as professional organizations, the pharmaceutical industry, payers, and patients wrestle to balance cost and benefit, along with risk and reward. The second article in this series will examine the perspectives of oncologists on value as these rising costs are impacting treatment choices. 

References

 

1. Howard DH et al. Pricing in the market for anticancer drugs. J Economic Perspect. 2015;29(1):139-162.

2. Schnipper LE et al. American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options. J Clin Oncol. 2015; 33(23):2563-2577.

3. Cherny NI et al. A standardized, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated form anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Ocol. 2015; 26:1547-1573.

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6. Georgoulias V, Papadakis E, Alexopoulos A, et al: Platinum-based and nonplatinum-based chemotherapy in advanced non-small-cell lung cancer: A randomized multicenter trial. Lancet 357:1478-1484, 2001
 
7. Rosell R, Carcereny E, Gervais R, et al: Erlotinib versus standard chemotherapy as first-line treatment for European patients with advanced EGFR mutation positive non-small-cell lung cancer (EURTAC): A multicenter, open-label, randomized phase 3 trial. Lancet Oncol 13:239-246, 2012
 
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