1. Eastman, Peggy

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In an era that is shifting from health care based volume to one based on value, more and more attention is being focused on the best frameworks to assess that value. Speakers discussed the promise, challenges, and pitfalls of value assessment tools at a conference sponsored by the National Pharmaceutical Council (NPC) in Washington, D.C. Co-sponsors were the Friends of Cancer Research, the Personalized Medicine Coalition, the National Health Council, the National Black Nurses Association, the National Patient Advocate Association, the Alliance for Aging Research, the Caregiver Action Network, the Global Liver Institute, the Healthcare Leadership Council, and the National Psoriasis Foundation.

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Underlying the theme of the conference was the recognition that rising U.S. health care costs, including co-pays, are unsustainable, and that high value should be a goal and a given in any health care intervention. "Patients should essentially get the benefit that they're paying for," said Lowell Schnipper, MD, Chief of the Division of Hematology/Oncology at Beth Israel Deaconess Medical Center; Theodore W. and Evelyn G. Berenson Professor Emeritus at Harvard Medical School; and Associate Director for Membership and Deputy Associate Director for Clinical Science of the Dana-Farber/Harvard Cancer Center, Boston.


While one standardized value assessment tool for a cancer or for other diseases might be a desire of some, it is highly unlikely, said speakers. "I believe that one framework won't fit all, ever," said Robert W. Dubois, MD, PhD, Executive Vice President & Chief Scientific Officer of the NPC, a nonprofit health policy research organization that receives support from pharmaceutical companies. A case study of four assessment frameworks for multiple myeloma presented at the conference proved that (see below). Dubois said important overall issues in deliberations on health care value assessment frameworks include the following:


* what evidence is used and how it is used;


* the patient perspective and involvement;


* how user preferences are incorporated into a value assessment framework;


* how the reproducibility of findings can be enabled;


* inclusion of cost effectiveness both from a health care and societal standpoint; and


* the budgetary impact of the value assessment framework.



Work in Progress

Dubois emphasized the issue of reproducibility of findings in any value assessment framework. "The byword has to be reproducibility; this is the essence of science," he said.


"Value frameworks are works in progress," noted Clifford Goodman, PhD, Senior Vice President and Director of the Center for Comparative Effectiveness Research at the Lewin Group. Such frameworks "are here to stay and continue to evolve," he said. Goodman noted that current value assessment frameworks have different purposes, which tend to reflect the interests and expertise of the groups that developed them. For example, ASCO has released "A Conceptual Framework to Assess the Value of Cancer Treatment Options (updated in May 2016)," whose goal is to provide a "standardized approach to assist physicians and patients in assessing the value of a new drug treatment for cancer as compared with one or several prevailing standards of care."


The National Comprehensive Cancer Network (NCCN) evidence blocks for value assessment are "intended as a visual representation of five key measures (efficacy, safety, quality of evidence, consistency of evidence, affordability) that provide important information about specific recommendations contained within the "NCCN Clinical Practice Guidelines in Oncology," in order to enable informed choices on systemic therapies by physicians and patients. Goodman noted that NCCN does not do traditional systematic reviews because of the urgency of releasing new information. "They do quick turnarounds because doctors and patients need the evidence," he said.


Robert W. Carlson, MD, Chief Executive Officer of NCCN, confirmed that. "We at NCCN have a whole bunch of people who are devoted to keeping our guidelines up to date," he said. Indeed, Carlson noted, a quick turnaround is one of NCCN's hallmarks.


And Memorial Sloan Kettering Cancer Center's DrugAbacus, created by Peter Bach, MD, is "a first draft of a tool that could be used to determine appropriate prices for cancer drugs based on what experts tend to list as possible components of a drug's value." The tool includes more than 50 drugs and will eventually include other cancer drugs and other indications.


"The user populations are really different across these value frameworks," said Goodman, whose research team evaluated five value frameworks including ASCO, NCCN, and Memorial Sloan Kettering Cancer Center. Goodman noted that even though there may be a target audience of stakeholders, such as oncologists and cancer patients, there are often secondary stakeholders, such as payors and policymakers. He recommended that value assessments be developed with transparency and with input and feedback from stakeholders, including patients (participating from the start) as well as experts, that they be as user friendly as possible, and that those who develop them be aware of potential misinterpretations and/or misuse. If this happens, "those who create the frameworks have to be ready to respond," Goodman stressed.


"I think we should welcome the frameworks; I think they're healthy for the patients and for the country," said Peter J. Neumann, ScD, Director of the Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Professor of Medicine at Tufts University School of Medicine, Boston. "We can always improve our process measures."


Neumann emphasized the need to capture the attributes of values that patients care about, as did Shelley Fuld Nasso, MPP, Chief Executive Officer of the National Coalition for Cancer Survivorship, a patient advocacy group. For example, patients are not only concerned about a treatment's efficacy, but also about side effects, quality of life, and whether they can continue working.


Case Study

The case study of value assessment in treatment of multiple myeloma, presented by Goodman, showed how four value assessment frameworks-ASCO's, NCCN's, the DrugAbacus, and one from the Institute for Clinical and Economic Review (ICER)-arrived at different conclusions. Goodman said he was not surprised by these different conclusions in value assessment for one disease, because "when you look at these frameworks, they don't look a lot alike."


Goodman noted that while there is some overlap in clinical outcomes of interest, they are not all the same. Also, he said, when it comes to cost/affordability outcomes, "there is a lot of divergence there." Goodman explained that value assessment frameworks can have methodological differences as well as differences in clinical outcomes across networks. And, he said, "Attempting to align these networks may be difficult and not useful."


While total uniformity of alignment may not be possible in different value assessment networks even for one disease, what should be possible is some expert expression of consensus, said Roy Beveridge, MD, Senior Vice President and Chief Medical Officer of Humana. "What payors are looking for is a uniformity of opinion," he said, noting that multiple myeloma is one of the most expensive diseases to treat. "For certain diseases, is there a unanimity of opinion? That's the standardization that we're looking for." Beveridge said that Humana uses several value assessment frameworks, including NCCN's and ICER reports. He added that today "the world is changing," and medicine is moving into alignment on what constitutes value between provider and payor. "They're not antagonistic," he said.


Beveridge stressed that payors are comfortable paying for high-value drugs "that have great efficacy and great utility. That isn't the problem." But what gives payors pause is "marginal drugs that have high toxicity," he explained.


Ongoing Conversations

"I am so happy that we're having this discussion," said Stephen J. Noga, MD, PhD, Senior Medical Director for Global Medical Affairs at Takeda Oncology and a Clinical Professor of Medicine at the University of Maryland. He agreed with Dubois that value assessment frameworks should prioritize the focus on the patient, be user friendly, be based on solid data, and be open and transparent. But, he cautioned, value assessment frameworks must not be used to pigeon-hole physicians in clinical practice. Also, he said, there is "an enormous amount of unmet need" in multiple myeloma and other disease areas, so it is important that value assessment tools not be used to stifle innovation.


The PMC has taken the position that budget impact assessments should be separate from value assessments in health care, because they are not the same. Also, the group stresses, budget impact statements should examine all aspects of the health care system, not just medications, since prescription drugs have an impact on the use of other health services (such as increased laboratory tests and decreased hospital costs).


Goodman pointed out that pharmaceutical companies have been dealing with value assessment frameworks globally for some time, and as an example cited the National Institute for Health and Care Excellence (NICE) in Great Britain, which sets guidance for medical professionals based on quality standards. So in the U.S., "Let them be used, however imperfect, and let people improve them," he said of value assessment tools. "If we don't start using some of these frameworks, it's going to be too late."


And, he concluded "I'm optimistic that market forces are making providers think much more seriously about value frameworks."


Peggy Eastman is a contributing writer.