As healthcare innovation accelerates, it’s important to have scientific methods and begin to move towards a more inclusive and transparent approach to measuring value—one that acknowledges diversity among patients’ characteristics, preferences, and related treatment effects.
This article was co-authored by Jennifer Bright, executive director, and Mark Linthicum, director of scientific communications, Innovation and Value Initiative.
The private sector is known for driving innovation in healthcare. From scientific advancements to advanced analytics, industry leadership moves next-generation concepts from ideas to practice quickly, in the name of improved health for all.
How each of us understands and defines the value of that innovation differs based on a multitude of factors, including our role (patient, clinician, or payer), health status, demographics, geography, and economic situation. Decision making in this regard is localized based on the above demographic factors in the US healthcare system.
It’s tempting to think that the explosion of initiatives striving for “value-based care” are a move in the right direction. Broadly, value-based care means care where the clinical benefits outweigh the risks and the cost. The challenge is an individual’s perception of “value” isn’t based on a universal definition, though. Clinical benefits, risks, and costs will vary across patients. Further, patient preferences may vary over these outcomes: some may prefer a more efficacious treatment despite more side effects, while others may prefer a less efficacious treatment that is associated with fewer side effects. Although we lack a more personalized assessment of value that pays attention to this heterogeneity, “value” efforts continue to charge ahead.
For example, pharmacy benefit manager (PBM) CVS Caremark recently announced it is introducing a new prescription drug cost management program for payers that would exclude from coverage certain new treatments that do not meet cost effectiveness benchmarks, specifically those used by the Institute for Clinical and Economic Review (ICER). Moreover, the health insurance lobby organization, America’s Health Insurance Plans, as well as the PBM lobby, the Pharmaceutical Care Management Association, each recommended in comment letters on the Trump Administration’s blueprint to lower drug prices that the federal government rely solely on ICER to assess value.
It’s premature to conclude that we should have a single arbiter of value in our healthcare system.
While it makes sense for value assessment to be woven into healthcare decision making, relying on a single method to measure value and using this metric to restrict treatment access is shortsighted. ICER typically calculates value based on costs and benefits for the “average” patient with a given condition. But it raises a critical question about how value is used in decision making: is information on value being used to identify the most valuable treatment for a given context and a given patient, or is it simply a tool for reducing costs? ICER’s approach may yield a convenient conclusion about cost-effectiveness that contributes to far-reaching decisions about access (formulary design) and price, but it fails to provide the most important insight: how to evaluate value that applies to localized decision-making.
Patient characteristics and factors that affect their care choices are highly relevant to the outcomes following a prescribed treatment, and yet such information is often missing from value assessment models because such data are not always recorded in clinical trials. Thus, the neat “answer” regarding the value of a treatment often overlooks vital information about sub-populations (eg, ethnicities, age, gender, or disease sub-type). Further, the benefits of expanded patient choice are not considered when high-level models—such as ICER’s—only measure clinical benefits, risks, and costs for the average patient. Ultimately, perspectives about how a therapy may work “in the real world” are often relegated to narratives or side-bar discussions rather than being directly factored into the value calculation.
One step in the right direction is to better incorporate the patient voice into measures of value. In fact, HHS Secretary Alex Azar and FDA Commissioner Scott Gottlieb, MD, have repeatedly advocated for the incorporation of patient viewpoints in research, development, approval, and clinical application of health technologies. It’s encouraging that government actors recognize the importance of balancing clinical evidence with patient perspective. Private sector decision makers should join federal officials and champion the same principles in value assessment.
Our collective focus in value assessment should be to evaluate evidence from both trials and real-world settings to assist all stakeholders in optimizing treatment choices. Achieving this will require innovation in our methods, even more transparency in data sharing and modeling, and a common commitment to synthesizing data to support localized decisions. We believe in engaging all stakeholders to contribute in an open-source environment that emphasizes building next-generation value assessment tools to meet the modern need for multiple perspectives.
As healthcare innovation accelerates, it’s important to have scientific methods and begin to move towards a more inclusive and transparent approach to measuring value—one that acknowledges diversity among patients’ characteristics, preferences, and related treatment effects. While it may be convenient to promote a one-size-fits-all answer, it’s not good enough for every patient, every time. That fact should cause all stakeholders to pause and to come together to seek and find a better approach.