ASCO Develops New Framework to Evaluate the Value of Cancer Therapies

Laura Morgan

July 2015, Vol 6, No 6 - ASCO’s Value Framework


Cancer care is one of the fastest growing components of US healthcare costs and is estimated to reach $158 billion by 2020.1 The American Society of Clinical Oncology (ASCO) Value in Cancer Care Task Force attributes the rising cost of cancer care to several factors, including the overall aging of the population, the introduction of costly new drugs and innovative surgery and radiation techniques, and the adoption of more expensive diagnostic tests.1

Although emerging technology continues to revolutionize cancer care, there are cases when the use of expensive novel agents and the latest treatment techniques do not yield sufficient evidence to warrant their use, resulting in increased costs without improved patient outcomes, according to ASCO.1

With more and more individuals feeling the financial impact of cancer treatment through high deductibles and larger copayments, an increasing number of patients would prefer to be more involved in choosing their treatment options. However, the lack of shared decision-making between providers and patients remains a significant barrier toward value-based cancer care.

ASCO’s Proposed Framework

In an effort to contain healthcare costs and to improve the quality of cancer care via shared decision-making, the ASCO Value in Cancer Care Task Force developed a conceptual framework to assess the value of new cancer drugs versus current standard of care treatments in 2 clinical settings: advanced disease and potentially curative disease.1

Co-chaired by Lowell E. Schnipper, MD, Chief, Hematology/Oncology, Beth Israel Deaconess Medical Center, Boston, and Nancy E. Davidson, MD, Director, University of Pittsburgh Cancer Institute, the Value in Cancer Care Task Force was charged with defining the value of cancer treatments using 3 parameters of quality healthcare: clinical benefit, toxicity, and cost. ASCO published its new value framework in June 2015.1

“As policymakers and payers seek ways to assure the best use of limited resources, they are appropriately turning ­­to physician experts for a better understanding—and definition—of value,” wrote Dr Schnipper and colleagues.1

“Value and cost are among the biggest issues in healthcare today, but there are few tools to help doctors and patients objectively assess benefits, side effects, and costs,” ASCO President Julie M. Vose, MD, MBA, Chief, Division of Oncology/Hematology, University of Nebraska Medical Center, Omaha, said in an ASCO press release announcing the publication of the new value framework.2

“Our goal is to help oncologists and their patients weigh potential treatment options based on high-quality scientific evidence and a thoughtful assessment of each patient’s needs and goals. In publishing this initial version of the framework, just the beginning of the process, we hope to drive discussion and debate about a critically important issue,” Dr Vose said.

“It’s critical to distinguish between value and cost,” said Dr Schnipper in the ASCO press release.2 “Sometimes the more valuable treatment will be the more expensive one and sometimes it won’t be. Ultimately, the definition of ‘value’ will be highly personalized for each patient, taking into account an individual’s own preferences and circumstances. For example, in the setting of advanced cancer care, is length of life the most important goal or is quality of life? Is the proposed treatment affordable? That’s why we’re proposing to provide information on net health benefit and costs side-by-side,” Dr Schnipper said.

Clinical Benefit
To assess the clinical benefit of cancer treatments in the advanced disease setting, overall survival (OS) is used as an efficacy measure to compare novel treatments with standard-of-care therapies in prospective clinical trials. If OS data are not available, progression-free survival can be used instead, and if survival data are not reported and/or only noncomparative clinical trials are available, the task force recommends using the response rate as a measure of clinical benefit.

In the advanced disease framework, each efficacy parameter is assigned a categorical score based on the fractional improvement it provides and is weighted based on its importance to value assessment, with OS having the highest weight and response rate having the lowest weight. In addition, bonus points are awarded to regimens that can demonstrate the palliation of symptoms and/or treatment-free intervals (Table 1).

Table 1

In the curative cancer framework, the categorical score for OS is based on the hazard ratio for the new treatment versus the comparator treatment; the hazard ratio for disease-free survival is used if OS data are not reported (Table 2).

Table 2

Dr Schnipper and colleagues acknowledged that other parameters, such as quality of life or patient-reported outcomes, are equally important for evaluating the clinical benefit of cancer treatments, but these parameters are not currently reported in clinical trials with sufficient consistency and reliability to be included in any value assessments.

Toxicity
In both frameworks, the frequency of grades 3 to 5 toxicities is evaluated for each of the treatment options, with points awarded or subtracted depending on how well the new agent is tolerated versus the comparator regimen.

At the end of each framework, the clinical benefit, toxicity assessments, and bonus points (only in the case of advanced disease) are combined to yield the net health benefit (NHB), which is juxtaposed against the cost of treatment to allow patients and providers to view the clinical information as separate from the cost considerations. The NHB is derived from randomized clinical trials, and the study populations are defined by the clinical trial eligibility criteria.

Cost
The direct cost of cancer treatment to the patient is an important factor in treatment decision-making. Overall, 2 cost elements are included in the framework, the drug acquisition cost and the patient cost, the latter of which is highly variable depending on the patient’s insurance benefits. In the advanced treatment framework, the drug acquisition cost and the patient cost are included as monthly costs of the regimen; in the curative framework, the drug acquisition cost and the patient cost represent the total cost of treatment for the standard duration of therapy.

The Need for Dynamic Frameworks to Assess the Value

Dr Schnipper and colleagues agreed that to implement this framework in clinical settings, it must be presented in a user-friendly manner, using software that is compatible on various devices.1

Furthermore, they emphasized that future frameworks need to be dynamic to allow patients to personalize their NHB score and to modify the framework at the point of care based on patient preferences, existing comorbidities, and values.

“In keeping with the patient-specific focus of this approach to assessing value, ASCO anticipates that cost will be interpreted by the patient in the context of the NHB offered by each treatment option,” stated Dr Schnipper and colleagues.1

“ASCO acknowledges that this method of calculating the NHB does not permit assessment of the relative value of regimens that were not directly compared in clinical trials and that the observed improvement in NHB for a new regimen might be influenced by whether the comparator was best supportive care or active treatment. Nevertheless, ASCO believes this method to be one that is well grounded in the available medical evidence and provides the most objective assessment of NHB,” the value task force wrote.

The team concluded that the value of cancer treatments is not static; rather it changes depending on the clinical setting (advanced disease vs adjuvant settings), the use of biomarkers, and the emerging medical information.

“The assessment of the value of any treatment must be dynamic and adapt to new medical information that may better inform its use, mitigate its toxicity, or modify its place in the treatment landscape,” Dr Schnipper and colleagues concluded.1

Dr Schnipper Presents the New Value Framework at ASCO 2015

At the 2015 ASCO meeting, Dr Schnipper discussed this new effort, saying, “Ultimately, we are optimizing a tool that a physician would have at his or her disposal in the office that would help patients understand, for a given clinical indication, what are the possible regimens, what are the ups and downs, and [how to] integrate those with the patient’s personal preferences.”

Practical Metrics

ASCO Task Force on Value in Cancer Care sought feedback from the oncology community, including academia, community practices, payers, and drug manufacturers “to devise a more uniform way of thinking about how best to treat patients for given indications,” he said.

“What you see along the spectrum of care are individuals making decisions that are probably in part, if not in total, predicated on financial issues,” Dr Schnipper. “It’s no surprise that the healthcare system has run amok from a financial perspective.”

Although the task force prioritized “clinical benefit” as the key concept driving value in healthcare, translating this into a practical, everyday value metric was a challenge.

Overall survival was determined to be the best end point for clinically meaningful outcomes. Progression-free survival, although an inferior metric, should also be factored into the value equation, said Dr Schnipper, because it is often all that physicians have to work with.

The palliation of symptoms in the context of advanced disease and treatment-free interval, “because that has to be a surrogate for quality-of-life consideration,” are also important variables, along with toxicity, according to the task force.

“In comparing 2 regimens, we felt it fair to begin to think about toxicities,” said Dr Schnipper. “Is one regimen more or less toxic than the other? The clinical value to a patient of having a less-toxic regimen cannot be overstated in this setting.”

Patient Parameters Are Crucial

Although health economists often use quality-adjusted life-years saved to make policy decisions based on healthcare expenditure, Dr Schnipper underscored the difficulty in providing quality for an individual patient, especially in the setting of advanced disease.

“Patients have very, very particular needs and wishes,” he pointed out. “Some want to avoid toxicity or neuropathy, because they’re violinists; some don’t care about the length of life but prefer to live a high-quality, comfortable life.”

Cost is a crucial factor for many patients’ decisions. “As the cost of care increases, and I’m not talking about the US economy, I’m talking about the patient sitting across from you—people exhaust their savings, so their kids may have to delay going to college. Second mortgages on the house are taken, and adherence to the medication is shown to be reduced,” Dr Schnipper said.

He noted that physicians and patients may have different understandings of the value in care.

“Patients express a great deal of value for bonding and positive relationships with their healthcare provider team…and perhaps are less preoccupied by some of the things that I just discussed as parameters,” he said. “On the other hand, how do you derive a value framework without some concrete scientifically ordained variables that are reproducible from one study to another?”

The task force hopes its framework can provide a user-friendly tool that will be available at the physician–patient interface and potentially affect public policy as well.




References

  1. Schnipper LE, Davidson NE, Wollins DS, et al. American Society of Clinical Oncology statement: a conceptual framework to assess the value of cancer treatment options. J Clin Oncol. 2015 Jun 22. Epub ahead of print.
  2. American Society of Clinical Oncology. ASCO publishes conceptual framework to assess the value of new cancer treatment options: framework to support shared decision-making between doctors and patients; ASCO solicits public comment. Press release. June 22, 2015. www.asco.org/press-center/asco-publishes-conceptual-­framework-assess-value-new-cancer-treatment-options. Accessed July 1, 2015.