Value Tools Are Still Missing a Few Pieces

Constantine Mantz, MD

Assessing the value of treatment options has become much more complex over the past several years, according to Constantine Mantz, MD, chief medical officer and radiation oncologist at 21st Century Oncology in Fort Myers, Florida. Many of his patients face high out-of-pocket costs and multiple therapeutic options—some of which cost several-fold more than pre-existing therapies and yield minimal clinical benefits. “Previously, physicians would make decisions based on the diagnosis and what, in their best knowledge and judgment, represented the right selection of therapies to address that disease,” said Mantz. “Those decisions were fairly uncomplicated compared with what we have to face today.”

Many community oncologists also report that for them and their patients it is difficult to decide whether a few extra weeks of life with a new therapy is worth the now-common five-figure price tag. To help facilitate patient—provider discussions about the value of a therapy based on its clinical benefit, toxicity, and affordability, the NCCN and ASCO introduced the Evidence Blocks and Value Framework, respectively, in 2015. However, Mantz and Kenneth Adler, MD, a hematologist-oncologist at Summit Medical Group, MD Anderson Cancer Center in Morristown, New Jersey, stated that the clinical usefulness of these value frameworks is limited by the type of data used, the subjectivity of the rating process, and the lack of consensus on which of these value tools is best. Both suggested that continued improvement and development of such tools could provide a more useful platform for discussion of the cost versus clinical benefit for multiple regimens.

Although the cost of the cancer drug is an obvious source of financial burden, one advantage of the NCCN Evidence Blocks is that they are based on the recognition that multiple other associated costs must be included when assessing treatment cost, and some of these costs are incorporated into the model. The panel members who create the Evidence Blocks rate affordability of treatment on a scale of 1 to 5 and consider costs of acquisition and administration of the antineoplastic drug, point-of-care treatment, supportive drugs, tests for monitoring toxicity, and further interventions such as antibiotic therapy or hospitalization. “The affordability is a metric of cost or expense to the healthcare system,” said Robert Carlson, MD, CEO of the NCCN. “We still think [this metric] is useful to the individual patient, because if the patient learns that a regimen is unaffordable, that may precipitate a conversation with the healthcare provider, financial counselor, or insurance company to figure out what the individual cost or expense is likely to be.”

Carlson described a case in his own practice in which he considered prescribing an effective-but-expensive aromatase inhibitor for a patient with metastatic, hormone receptor-positive breast cancer. After she discovered that she would not be able to afford her insurance co-pay, she was referred to a financial counselor, who found her a co-pay assistance program to help with the remaining balance. “The reason that this whole sequence of events unfolded was because there was an indication in the decision making that this was a very expensive treatment,” said Carlson, suggesting that this conversation may not have happened without the use of a value tool to identify efficacy and cost.

However, Mantz noted that with the Evidence Blocks, expert panel members rate each of the treatments based on what they think will be most or least expensive: they don’t use objective cost data, which he stated makes the Evidence Blocks too subjective for clinical purposes.

The cost metric for the ASCO Value Framework focuses on just the estimated costs of the antineoplastic and supportive care agents because these are usually the most immediate and relevant costs to the patient and provider, according to Lowell Schnipper, MD, chair of ASCO’s Value in Cancer Care Task Force. He acknowledged that some experts have criticized the model for failing to include other variable costs, such as time away from work due to repeated infusions or hospitalizations required for certain agents. However, he said the framework is designed to promote the patient-provider dialogue. “We really wanted to focus on the medical oncology interaction and, specifically, the financial vulnerabilities that the patients are facing,” he said. “We tried to narrow it to the doctor—patient dialogue because that is what the patient comes to the doctor for,” he said.

Although Mantz supports the focus on high-quality studies to assess cost and net clinical benefit, he noted that excluding well-performed prospective studies that are not large randomized controlled trials could limit the usefulness of the tool, particularly for less-common cancers that do not have a large population of patients to recruit for clinical trials. “Allowing a little more latitude to permit other high-quality data to enter into the tool so that more diseases can be assessed and compared across different regimens would be helpful,” said Mantz.

Overall, Adler said, the lack of consensus on an optimal cost-valuation framework will make widespread adoption slow for any particular framework. “People come up with different tools, but which one should they follow? There’s no right answer, so how do you promote one?” Many community and academic oncologists surveyed at the 2016 ASCO Annual Meeting are in agreement on this point, as 46% reported not using value frameworks to in uence their treatment decisions and about 20% said they consider using them only to provide guidance on cost.¹ The lack of consensus on which framework to use was evident in the wide variety of models considered by survey respondents, including the ASCO Value Framework, Evidence Blocks, clinical pathways, self-created frameworks, and patient-centered models.

Other notable frameworks include the DrugAbacus, which compares actual drug costs with what its makers consider reasonable prices based on efficacy, safety, toxicity, and novelty, and other factors. The ICER Value Assessment Framework assesses the societal value of a therapeutic regimen. However, Mantz noted that the clinical utility of the DrugAbacus is limited, as it serves as a cost-comparison tool for specific cancer drugs and doesn’t factor in costs of supportive care; and Carlson said that the ICER framework is targeted toward the payer, rather than patients and physicians. The European Society of Medical Oncology (ESMO) also published a Magnitude of Clinical Benefit Scale in June 2015, which rates new antineoplastic drugs according to their anticipated benefits (in terms of overall and/or progression-free survival) over the standard treatments. Although this tool does not include a cost factor, the authors of the paper stated that assessing the incremental clinical benefit supports value-based decision making and appropriate use of resources for affordable cancer care.²

However, both Mantz and Adler acknowledged that value frameworks could help facilitate the increasingly complex dialogue about financial cost versus clinical benefit with the multiple new agents available. Mantz and his colleagues require that all new patients meet with a financial counselor to clarify out-of-pocket responsibilities before starting therapy, which helps physicians choose an affordable treatment plan and increases transparency of healthcare costs to patients and their families. He stated that a value framework could be useful during these initial cost discussions. “We would like to have a very robust tool that would allow value comparisons across different regimens,” said Mantz. Similarly, Adler and other oncologists at his practice assess predicted costs of therapy and out-of-pocket responsibilities before starting treatment. The complexity of this process has led them to rely on administrators and staff to initiate the cost talk with patients.

The Future of Value Frameworks

Adler stated that value frameworks may be valuable for demonstrating that the marginal improvements in survival afforded by some new agents may not be worth the greater financial cost. “We have to determine the efficacy [of potential agents] and whether their expense is worth putting someone on them,” said Adler.In the updated ASCO Value Framework,³ Schnipper stated that the ultimate goal of the ASCO Value Framework, when fully operational, is to deliver effective, affordable care that considers the patient’s priorities for treatment. “Patients are spending huge sums of money that exceed the capacity of their budget to get care, but the treatment programs may not be better than less costly ones.”

Schnipper indicated that ASCO plans to employ information technology developers to put the detailed framework into a software vehicle, which would allow the physician to look up the net health benefit and cost for multiple regimens. This information would, in turn, help patients make a treatment decision based on their personal assessment of value, which in the case of a musician might have more to do with preserving manual dexterity than the dollar cost of therapy. “A patient might say, ‘Gee, I’m a cello player. I really don’t want to have neuropathy, and I see that a lot of these regimens have this side effect,’” said Schnipper. “On the other hand, a patient may say ‘this one looks a tiny bit better but is 10 times more costly—is it really worth it?’ This is what we envision with our tool.” Second-line therapy for non—small-cell lung cancer, according to the experts and guidelines, can range from Taxotere to Alimta to Opdivo. The cost differences among those options are in the thousands of dollars. Is CMS trying to deter us from using the better and safer yet much more expensive agents to achieve a benchmark that will reward us for a Good boy! performance? The OCM seems based on the assumption that oncologists chose more expensive drugs for a better profit margin, but even if that is true, most of the agents chosen are more effective, cause fewer side effects, and provide better quality of life. We really have to think about this more deeply. The taxpayers’ money is there to serve our fellow citizens, especially the most vulnerable ones—patients with cancer.

One area that the OCM model has miserably failed to address is clinical trials participation. There are pros and cons to inclusion of clinical trial patients. The pros are better medicine for the patients because they are treated with futuristic drugs. Second, studies have shown that patients on clinical trials live longer and have better quality of life regardless of whether trials are successful or not.

But patients who get enrolled in clinical trials are the cream of the crop, they are generally healthier than average, they are motivated and want to live longer, and they fight one step more than the average patient with the same disease and stage. Theoretically, practices that attract patients seeking clinical trials are potentially the ones whose patients will live longer and receive more lines of therapy. This can and will make such practices look more costly when compared with those that don’t participate in meaningful phase I, II, and III clinical trials. They will be penalized and unfairly so.

Speaking of the “fighter patients,” who would refuse to leave any stone unturned—even outside clinical trials—such patients will never quit as long as there is any potential that they can live another few days or weeks to see a daughter getting married or witness the first communion of a grandson. But when, finally, there is nothing else that can be done, such patients agree to go for hospice care, where the survival is numbered in days, not weeks or months, and this affects the OCM penalty, because the days a patient spends in hospice are a part of the equation. The longer the hospice time, the better boy you are, because it is assumed that earlier referral to hospice results in longer hospice days, a presumed fact that is very much in dispute.

The hospice length of stay is vastly variable depending on the practice and philosophy of a particular hospice. We at Gabrail Cancer Center have done an analysis of that. Generally, privately owned for-profit hospices keep the patient alive for a significantly longer period of time compared with the not-for-profit, which is usually hospital-owned. That variation between centers can have an impact on the OCM program evaluation and this disparity is totally out of the control of the treating oncologist.

The most formidable challenge still rests on hospitalization. I need to come back to this topic because it is as convoluted and political as it gets. When an increasing number of primary care doctors decided to relinquish their hospital privileges, hospitals got nervous about who would take care of the hospitalized patients and, more critically, how they would keep hospital beds full. The hospitalist program was the answer. Now, hospitals employ or contract with ED groups and an undeclared admissions quota is put in place, complemented by the hospitalist program, which serves both the owners of the hospitalists program and the hospitals. Of course, it is even more convenient if the hospital employs the hospitalists. It is very hard for the oncologists to control that scenario when their patients show up at the ED with a cough or sore throat. The ED looks for a reason to admit, and the hospitalist is happy to take on the task.

The OCM program is a good start on a path that is as rough as it gets in our healthcare system that needs reform, which can’t come from legislation such as Obamacare. The change has to come through gradual reform that is trial and error and that would work as long as the intent is honest and pure.

References

1. Lederman L. Value tools at ASCO 2016: building a framework for prime time. OBR Green. obroncology.com/obrgreen/article/Value-Tools-at-ASCO-2016- Building-a-Framework-for-Prime-Time. Accessed September 12, 2016.
2. Cherny NI, Sullivan R, Dafni U, et al. A standardised, generic, validated approach to stratify the magnitude of clinical benefit that can be anticipated from anti-cancer therapies: the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS). Ann Oncol. 2015; 26(8):1547-1573. doi: 10.1093/annonc/mdv249. Accessed September 12, 2016.
3. Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology Value Framework: revisions and reflections in response to comments received. J Clin Onc. 2016;34(24):2925-2934. doi: 10.1200/ wJCO.2016.68.2518. Accessed September 12, 2016.