Value Assessments Are Just One Tool in the Decision-Maker's Toolkit, Panel Says

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In a Monday panel convened at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 23rd Annual International Meeting in Baltimore, Maryland, stakeholders grappled with the role that value assessments play—or should play—in US private payer coverage and formulary decisions.

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In a Monday panel convened at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) 23rd Annual International Meeting in Baltimore, Maryland, stakeholders grappled with the role that value assessments play—or should play—in US private payer coverage and formulary decisions.

The panel was moderated by James D. Chambers, PhD, MPharm, MSc, associate professor of the Center for the Evaluation of Value and Risk in Health at the Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, Massachusetts. Chambers opened the discussion by giving an overview, derived from Tufts’ Specialty Drug Evidence and Coverage Database (SPEC) of how often health technology assessments (HTAs) were cited in coverage decisions by US commercial payers. SPEC’s data, current as of August 2017, included more than 4800 coverage decisions in more than 150 disease states and 200 specialty drugs.

In the more than 27,000 citations, Chambers and colleagues found that information from HTAs were cited in only 4% of cases, and economic evaluations in only 1%. Surprisingly, of the HTAs cited, 46% were set in the United Kingdom, while only 28% were set in the United States. Among the US assessments, the greatest number derived from Blue Cross Blue Shield’s Technology Evaluation Center, and only 2% from the Institute for Clinical and Economic Review (ICER).


“Some payers are going outside of the United States for this information … the takeaway here is that the relatively small percentage of these are issued by ICER given the rise and prominence” of the organization, said Chambers.

Sara Traigle van Geertruyden, JD, the executive director of the Partnership to Improve Patient Care (PIPC), highlighted 1 of the reasons why ICER assessments may not be cited as frequently as might be expected. According to van Geertruyden, assessments that rely on quality-adjusted life years (QALYs) have raised significant ethical questions because they can lead to discrimination against people who have disabilities or long-term health conditions. These patients, says van Geertruyden, will never achieve a state of “perfect” health as assessed by a QALY.

“It’s at odds with what we think of as personalized medicine and the patient-centeredness movement,” she said, adding that both HHS and the Affordable Care Act have restricted the use of QALYs in decision making because its application was deemed discriminatory. However, “[the QALY] seems to be sneaking back.” For the disability community in particular, she said, “that seems like a scary proposition.”

PIPC, which holds that value assessments should include patient-reported outcomes measures, facilitate shared decision making, and avoid a singular focus on cost, pointed to the National Health Council’s Patient-Centered Value Model Rubric and the FasterCures Patient Perspective Value Framework as alternatives to QALY-based value assessments. These models, said van Geertruyden, highlight applicability, usability, and transparency of evidence, and should hopefully assist patients and their families in understanding how treatments will impact their lives.

Susan A. Cantrell, RPh, CAE, CEO of the Academy of Managed Care Pharmacy (AMCP), explained that AMCP’s own framework, which has come to be known in the vernacular as AMCP’s “dossier,” has evolved over time to serve the functions of helping payers gather information, determine that information’s credibility, synthesize relevant information, and make evidence-based coverage and formulary determinations.

According to Cantrell, value assessments have been difficult for stakeholders to adopt, in part because they lack transparency. She referenced ICER reports as an example, saying that, “Economic models used in the development of the frameworks need to be publicly disclosed and available so that they can be evaluated.”

“We like to think that [the dossier] provides a framework for collecting and assessing information,” Cantrell said, but value frameworks are only “a tool in the toolbox,” and they’re not the only tool. Good decision-making should include broad stakeholder perspectives, publicly available methods and models, scientifically valid evidence, understandings of value that are interpretable for relevant audiences (including patients), transparent in terms of who was involved in development, and regularly updated as new information and research becomes available.

Randy Burkholder, vice president of policy and research for the Pharmaceutical Research and Manufacturers of America (PhRMA), agreed with Cantrell that value assessments are only 1 tool at stakeholders’ disposal, saying “I think that’s a fundamental and very important reality.” Burkholder added that he believes the low incidence of citations of ICER assessments in payer determinations may be because “ICER fundamentally purports to be something more than a tool in a toolkit,” and that the QALY as a measurement tool is poorly aligned to the US healthcare system. Payers, he said, are seeking greater nuance and flexibility than such a measure provides.

He went on to argue that cost-effectiveness should not form the cornerstone in thinking about value; a more useful framework will be one that, among other things, supports patient-centered care, upholds continued scientific and medical progress, and takes a system-wide perspective in addressing existing inefficiencies in the healthcare system rather than focusing solely on the cost of new innovations.

Speaking from the audience during the panel’s question-and-answer component was Steven Pearson, MD, MSc, founder and president of ICER. Pearson contended that there would be a higher number of citations for his organization’s reports in the SPEC database had more recent data been included in Chambers’ analysis, and added that “we would never envision” that ICER is the only factor that payers should consider in making decisions. Finally, he cited pricing of Amgen and Novartis’ new migraine therapy, erenumab-aooe (Aimovig), as a reflection of the utility of ICER’s work in helping to rein in drug costs.