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The Talk in Medicine is of Cultivating Value and Cost-Effectiveness, But Is Neurology Getting It?

ARTICLE IN BRIEF

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AS COSTLY NEW treatments for neurologic emerge, clinicians will need a way to determine whether they are worth the money compared to less expensive options, health services researchers said.

In the first article in a new series on cost-effectiveness in neurology, leaders in health outcomes research discuss the challenges of defining value in neurology.

The health care value movement is gathering steam, with most payers vowing that value-based payments will soon become the norm.

The Centers for Medicare & Medicaid Services (CMS) intends to have 80 percent of payments flow through value-based arrangements by 2018, and the Health Care Transformation Task Force, made up of America's largest insurers and health systems, says that 75 percent of their business will be in value-based contracts by 2020.

So what constitutes value in neurologic care? That is not yet clear, according to some experts engaged in the issue, and they say the question is begging for an answer.

“Despite the fact that many of the products we have in multiple sclerosis [MS] don't really work very well, they are wildly expensive,” said Annette Langer-Gould, MD, PhD, a regional physician multiple sclerosis champion at Kaiser Permanente Southern California.

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DR. NABILA DAHODWALA: “How do you extrapolate benefit data from the clinical trials? Its challenging when we use different outcome measures across different trials, and there has not been much focus on patient-centered outcomes until more recently.”

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DR. CLAIBORNE JOHNSTON: “Theres so much opportunity to just eliminate waste, that if we just take care of that, then we will have made substantial progress. To be short-sighted and not to recognize that youre harming your patients by wasting resources, I think is naïve.”

As costly new treatments for other neurologic conditions emerge, she said, clinicians are going to need a way to determine whether they are worth the money compared to less expensive options.

“We're going to get to that point in stroke really quickly, and we're going to get there in the next 10 years for dementia medications as well,” she added. “So MS is just the beginning.”

VALUE DEFINED

Value in health care is generally defined as outcomes relative to costs. But measuring value is difficult because the terms “outcomes” and “costs” can justifiably be defined in many different ways.

Although “value” and “cost-effectiveness” are often used interchangeably, the latter term has a more precise meaning, according to Peter Neumann, ScD, director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center in Boston.

“That often means someone has actually analyzed the cost and benefits and is looking at the cost per unit of health gained from using an intervention,” he said. “The word ‘value’ is a very broad term that typically means whether an intervention is worth the money, whether or not there has been an analysis that has quantified it.”

The question of value in neurology is arising because of the rapid advances in neuroscience. “We are beginning to have treatments for neurological conditions that we've never had before and so we have issues around how we can optimally use those treatments in terms of costs and health benefits,” said Mitchell S. Elkind, MD, FAAN, director of the Neurology Clinical Outcomes Research and Population Sciences Group at Columbia University.

Policymakers and payers — both government and private — are focusing broadly on how to improve the value of health care, and much of their attention is on avoiding inappropriate hospital use. Medical societies, however, are most concerned with the relative value of treatment options.

Physician and hospital costs make up larger chunks of the nation's health care tab than prescription drugs, which account for between 10 and 15 percent of overall health care spending. But evaluating a drug is generally more straightforward than many other elements of health care, Dr. Neumann said.

“Drugs are an inviting target in a way because the prices have been going up rapidly and it begs questions about whether there's value or not,” he said. “So it's understandable and maybe convenient and appropriate, but it's not the only place you'd want to look.”

WHO DETERMINES VALUE?

In 2013, the AAN joined other medical societies in the Choosing Wisely campaign, one of the first attempts to identify and curtail low-value health care practices. Like the other societies, the Academy published a list of five common, high-cost practices that clinicians and patients should question because they may not be worthwhile.

The use of those recommendations has not been studied to determine if, in fact, they are having the intended effect of reducing unnecessary health care spending. But Dr. Langer-Gould, co-chair of the Academy's Choosing Wisely committee, said publishing the recommendations has raised awareness of the concept of value.

“They have certainly served the purpose of getting neurologists to think about us leading the way — instead of always feeling like the payers are leading the way — and helping us get a voice at the table for making some of these decisions,” she said.

Physicians in some medical specialties have assumed responsibility for promoting high-value products and highlighting their low-value alternatives. For example, leaders in oncology and ophthalmology have called out drugmakers that charge high prices for biosimilars that provide no benefits over existing options, Dr. Langer-Gould said.

She hopes neurologists will become bold in advocating for high-value therapies.

“If professional societies don't take the lead on making some rational recommendations, the payers will make their own because some drugs are not really affordable,” she said. “That is where we are in MS care — unfortunately, payers are classifying some treatments as high-tiered specialty pharmaceuticals and increasing the out-of-pocket expenses for the patient.”

Neurologist Claiborne Johnston, MD, PhD, is dean and vice president for medical affairs at the new University of Texas-Austin Dell Medical School, which is focused on creating new models of care based on value and improving health at the population level. In his view, neurologists should not cede responsibility for determining value in neurology care to other stakeholders.

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DR. PETER NEUMANN noted that ASCO came up with a rating system that, to an outside observer, could be considered arbitrary. The ACC/AHA does measure cost-effectiveness in terms of QALYs, but ignores a drugs overall budget impact. Thus, a therapy that might be deemed to be high-value for an individual patient might overwhelm Medicare or a private payer if that drug were prescribed to every patient that would benefit, he said.

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DR. MITCHELL S. ELKIND: “We are beginning to have treatments for neurological conditions that weve never had before and so we have issues around how we can optimally use those treatments in terms of costs and health benefits.”

“The most appropriate plans for how to manage our patients that are aligned with the patients' best interests are going to be created by experts who understand those conditions the best,” he said. “We need to have neurologists stepping up to lead, not just to reluctantly participate.”

DIFFERENT VIEWS ON VALUE

There is no consensus on the neurologists' responsibility to address the value conundrum, however. Of course, most physicians put their patients first, and some neurologists say their patients are focused solely on getting help, not the cost of treatment. Others report a different experience.

“Most of us have been forced to deal with this issue for many years,” Dr. Elkind, a stroke specialist at New York-Presbyterian Hospital, said. “I have patients who tell me that they can't take the medication I've prescribed because they can't afford it, or perhaps they're taking a half of the pill each day instead of a full pill because that way they can stretch it out longer.”

Dr. Langer-Gould was introduced to the bigger picture of health care costs when she moved from an academic practice at Stanford Medicine to Kaiser Permanente, which receives a fixed per-capita amount to pay for all heath care services provided to its population of patients.

“For the first time in my career I was asked by my colleagues to consider the cost of care of all of our members, not just those with MS, and to think more seriously about whether the care that we are providing for our members with MS is both high-quality care and affordable care,” she said.

Dr. Johnston also thinks neurologists should consider value from the perspective of their individual patients and society at large.If a patient's out-of-pocket responsibility is not considered before making a treatment recommendation, he said, the patient may be non-adherent, leading to adverse outcomes.

“Failure to incorporate the full insights of a patient about the impact of a decision on them represents a failure of the neurologist in reaching out to them and understanding their situation, and what's likely to be effective,” he said.

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DR. ANNETTE LANGER-GOULD: “If professional societies dont take the lead on making some rational recommendations, the payers will make their own because some drugs are not really affordable. That is where we are in MS care — unfortunately, payers are classifying some treatments as high-tiered specialty pharmaceuticals and increasing the out-of-pocket expenses for the patient.”

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MODELS FROM TWO MEDICAL SOCIETIES FOR ASSESSING VALUE AND COST-EFFECTIVENESS

Beyond that, former CMS Administrator Don Berwick, MD, and a colleague estimated in a 2012 paper in the Journal of the American Medical Association that as much as one-third of US health care spending is wasteful.

Dr. Johnston, former director of the Center for Healthcare Value at the University of California, San Francisco, believes physicians have a responsibility to tackle that by, for example, avoiding unnecessary imaging that does not change treatment decisions.

“There's so much opportunity to just eliminate waste, that if we just take care of that, then we will have made substantial progress,” he said. “To be short-sighted and not to recognize that you're harming your patients by wasting resources, I think is naïve.”

WAYS TO ASSESS VALUE

Some medical societies are trying to tackle the value conundrum. For example, the American Society of Clinical Oncology (ASCO) proposed a value framework for considering chemotherapy options last year, and the American College of Cardiology (ACC) and the American Heart Association (AHA) developed a cost/value methodology for use in developing guidelines and performance measures in cardiology.

The two approaches vary considerably, demonstrating that there is not yet a consensus about how to evaluate treatment options. (See the sidebar, “Models from Two Medical Societies for Assessing Value and Cost-Effectiveness.”)

“This is hard to do, and we need groups like this thinking about and issuing statements about measurement of value. So I welcome and applaud their efforts,” Dr. Neumann said. “Having said that, there are a lot of challenges here, and I think we might see these as evolving frameworks that are probably in need of refinement and experience.”

Likewise, the neurologists interviewed for this article appreciated the idea of a framework to quantify the relative value of therapies and diagnostics — although neither approach would lend itself easily to neurology care.

For example, ASCO's approach — comparing the relative efficacy, toxicity, and cost of alternative treatments — works well for cancer care because survival is a primary outcome measure for most diagnoses, Dr. Elkind said. Not so for neurology.

“Neurologists are not only concerned about people dying — we are concerned about people's functional ability to look after themselves,” he said. “We are concerned about dementia and gait disorders and incontinence and a whole host of other quality-of-life-related issues, so that complicates things.”

Even if there were consensus about the outcomes that should be used to assess value, obtaining data about those outcomes is its own challenge, said Nabila Dahodwala, MD, a neurologist at Penn Medicine in Philadelphia and a senior fellow at the Leonard Davis Institute of Health Economics.

“How do you extrapolate benefit data from the clinical trials?” asked Dr. Dahodwala, an assistant professor of neurology at Pennsylvania Hospital. “It's challenging when we use different outcome measures across different trials, and there has not been much focus on patient-centered outcomes until more recently.”

While ASCO's dimensions of value — clinical benefit, toxicity, and cost — are reasonable, the framework does not consider other dimensions, such as novelty of a treatment and cost-effectiveness as measured in cost per quality-adjusted life year (QALY).

“Then the question is: If you agree on the dimensions of value, how do you weight those different dimensions?” Dr. Neumann said.

ASCO came up with a rating system that, to an outside observer, could be considered arbitrary, he pointed out.The ACC/AHA does measure cost-effectiveness in terms of QALYs, but ignores a drug's overall budget impact. Thus, a therapy that might be deemed to be high-value for an individual patient might overwhelm Medicare or a private payer if that drug were prescribed to every patient that would benefit.

“The main point is there are analytic challenges here, and I think there's a need for ongoing work on this,” Dr. Neumann said.

Dr. Dahodwala likes the scoring approach. But, as the authors of the ASCO framework point out, it does not incorporate individual patient preferences and, thus, does not address a patient's own values.

“And I think that's where we need to get to if we're really going to have a personalized approach to how we counsel patients,” she said. “We need to understand, as part of patients' cost, how they value different health states.”

Meanwhile, Dr. Johnston agrees that a consensus about the importance of assessing value and making recommendations for neurologists is critical, but perhaps not in the scoring system such as that used by ASCO and ACC/AHA.

“Do we need something like that in neurology? I'm not convinced,” he said.

Practice guidelines developed by the AAN and other professional societies are based on the scientific evidence about treatment options, so they address the quality component of value. What's missing is the cost component of the value equation.

“We need to come together to develop that consensus and then incorporate that into our guidelines processes,” he said.

This article is the first in a series, “Dollars and Sense,” that will explore neurology's efforts to deliver high-value, cost-effective care. In the next article, we'll look at how physicians are discussing costs with their patients so the patients can help determine value from their own perspective.

LINK UP FOR MORE INFORMATION:

•. Berwick DM, Hackbarth AD. Eliminating waste in US health care http://www.ncbi.nlm.nih.gov/pubmed/22419800. JAMA 2012; 307(14):1513–1516.
    •. 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 http://www.ncbi.nlm.nih.gov/pubmed/26101248. J Clin Oncol 2015:33(23): 2563–2577.
      •. Anderson JL, Heidenreich PA, Barnett PG, et al. ACC/AHA statement on cost/value measures: A Report of the American College of Cardiology/American Heart Association on performance measures and task force onpractice guidelines http://www.ncbi.nlm.nih.gov/pubmed/24681044. J Am Coll Cardiol 2014;63(21):2304–2322.
        •. Neumann PJ, Cohen JT. Measuring the value of prescription drugs http://www.ncbi.nlm.nih.gov/pubmed/26580666. N Engl J Med 2015; 373(27): 2595–2597.
          •. Roh JA, Cohen JT, Veenstra DL, et al. Development of a cost-effectiveness analysis framework for modeling treatment of Alzheimer's disease and mild cognitive impairment http://www.ncbi.nlm.nih.gov/pubmed/26533910. Value Health 2015:18(7): A698–699.