A New Value Assessment Framework

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We spend 18 percent of our national gross domestic product on health care. As health care spending continues to grow and as we appropriately drive the health care system toward a payment system that rewards value instead of volume, it is imperative that we promote conversations on how to define value. To do this, it is critical that we first answer the question: value to whom?

Value in health care can mean different things to different stakeholders. Payer priorities may not match up with manufacturer concerns, and both may assess value entirely differently than public health entities. However, no matter which of these stakeholders is measuring value, it’s important that value assessments always robustly consider and measure what matters most to the ultimate consumers of health care: patients.

The patient perspective on value is of particular importance now, as patients are responsible for more and more of the costs of their care. Today, more than 1 in 4 Americans report facing challenges paying for their medical bills and about 79 percent of cancer patients report moderate to catastrophic financial burden related to their care. Low-income families often spend more than 20 percent of their after-tax income on out-of-pocket health care spending, even when enrolled in low- or no-deductible plans.

Not surprisingly, a recent Kaiser Family Foundation poll found that two-thirds (67 percent) of Americans, irrespective of political affiliation, feel that lowering out-of-pocket costs for health care should be a top priority for President Donald Trump and Congress. But despite the drive toward value-based health care reimbursement and patients’ ever increasing financial stake in their own health care treatment, many traditional value assessment tools fail to consider value from the patient’s perspective.

Recently, a Health Affairs Blog post from the National Pharmaceutical Council (NPC) outlined the limitations of many new tools for value assessment, positing that we have much more work to do—in particular in considering “what’s important to patients.” The authors critiqued five prominent value assessment models developed by the American College of Cardiology/American Heart Association, the American Society of Clinical Oncology, the Institute for Clinical and Economic Review (ICER), the Memorial Sloan-Kettering Cancer Center (DrugAbacus), and the National Comprehensive Cancer Network.

The NPC makes a number of important recommendations for improvement; however, the piece fails to take into account the progress already being made. In particular, the Avalere-FasterCures Patient-Perspective Value Framework (PPVF) version 1.0—developed in collaboration with a multistakeholder steering committee—has made significant progress in framing a new way to assess value from the patient perspective.

The Patient-Perspective Value Framework

Integrating patient perspectives into value assessment frameworks will not only help patients, but it will also enable pharmaceutical and medical device manufacturers, providers, and payers to develop, deliver, and pay for products that provide a more meaningful benefit to patients and higher value to society. Through a process that included significant public input and collaboration with other value framework developers, Avalere and FasterCures published version 1.0 of the PPVF on May 11, 2017.

Unlike the existing frameworks in which input from patients or other stakeholders is limited, the PPVF process included stakeholders from the start, including a significant number of patient groups. Twenty-three organizations contributed to the framework’s development as members of the PPVF steering committee. In addition, more than 230 individuals and organizations offered feedback to the draft PPVF. Importantly, 80 percent of the feedback came directly from patients, caregivers, and patient advocates.

Furthermore, to ensure the development of a framework that is truly built on the patient perspective, the PPVF started with a list of considerations—or criteria—that are important to patients when making health care decisions. These considerations were then organized into five domains, outlined in Exhibit 1. Each domain includes a set of criteria and outlines a methodology for how those criteria can be assessed through a set of identified metrics. This iterative, collaborative process yielded a framework that is significantly different from any of the other existing frameworks in five key ways.

  1. The value assessment is viewed through the lens of patient preferences.
  2. The PPVF considers outcomes that matter to patients and incorporates real-world data necessary to measure those outcomes, instead of limiting the measures considered to those tracked in randomized clinical trials (RCT).
  3. The PPVF measures the true cost to the patient and family instead of focusing on financial cost to the system alone.
  4. The PPVF acknowledges that different evidence applies to different patients and uses subgroup data where possible.
  5. Usability and transparency are underlying principles that serve as a foundation on which the PPVF value assessment rests.

Exhibit 1

Please visit the Avalere website to download a graphical summary of the criteria and measures as well as a detailed report outlining the PPVF version 1.0 methodology.

Each of the PPVF’s five domains contributes a different type of information to the framework and differentiates the PPVF value assessment in its own way, as described below. Together, the five domains assess the value of individual health care options relative to individual values and preferences.

1. An Assessment Driven By Patient Preferences

Patients in different situations may consider different criteria important. That is why the PPVF uses patient preferences—the assessment of patients’ values, needs, goals, expectations, and openness to financial trade-offs—to drive the value assessment methodology. The assessed patient preferences weight the other PPVF domains, criteria, and measures. This means that, for instance, quality of life may carry twice the weight of efficacy and effectiveness in the ultimate value assessment, if that’s what patients prefer.

If the PPVF is being used by patients and clinicians in the context of shared decision making, the PPVF will elicit patient preferences directly from the patient himself or herself. If it is being used to support value assessments at the population level (for example, by a health plan or policy makers), patient preferences can be assessed through surveying relevant literature, using registries that capture patient perspectives, and—if possible—conducting original research among patients.

The centrality of patient preferences to the PPVF value assessment is a unique feature of the framework. Other value frameworks only consider patient preferences tangentially or as “other considerations.” Recently, the ICER made significant efforts to integrate patient preferences in its 2017 update through a new process for considering its “other benefits or disadvantages” and “contextual considerations” domains. While this effort and similar efforts by other value framework developers to better represent the patient voice in their processes are commendable, none of these other frameworks have clearly placed patient preferences at the center of the decision-making process. The PPVF puts patient preferences front and center as the driver of the value assessment methodology.

2. Outcomes That Matter To Patients

Understanding the clinical benefits and risks of different health care options is central to any value assessment, but too often value assessments use only measures that narrowly focus on clinical benefits, such as those typically used in RCTs. The PPVF is unique in that it includes outcomes that consider the “whole patient,” ranging from a therapy’s effect on functional and cognitive status to a regimen’s complexity. The PPVF steering committee developed the list of patient-centered outcomes criteria through extensive outreach with patients, caregivers, and patient advocates to ensure that no factors important to a patient’s value assessment process were missed. The criteria measured under this domain include quality of life, complexity of regimen, efficacy and effectiveness, treatment-related side effects, adverse events, and complications.

3. The True Cost To Patients And Their Families

Financial constraints often create a burden comparable to more traditional side effects—so much so that many have started using the term “financial toxicity” to describe the condition. Because of this, you cannot assess value without considering costs that accrue to patients and their families. Unlike other value assessment methodologies that consider only cost to the system, the PPVF considers out-of-pocket costs to the patient and family, non-medical costs and burdens, and the impact of a treatment on future costs.

Specifically, the PPVF’s patient and family costs domain measures the financial impact of a patient’s insurance benefit design, such as deductibles, copayments, and the cost of associated supportive care, as well as the non-medical costs and burdens to the patient and family, such as lost productivity, the cost of travel, and the level of burden on family and caregivers.

4. Different Evidence Applies To Different Patients

Throughout the PPVF development process, patients communicated loudly and clearly a desire to understand if the evidence shows that the treatment might work for “people like me” (for example, patients with their specific type of tumor or of their age, race, or gender). While every value assessment must measure the quality of the evidence, the PPVF differs from other value assessments in its consideration of the applicability of evidence to specific subgroups through the use of nontraditional data sources (for example, clinical registries or administrative/electronic medical record repositories, drug/device label information, and cost transparency websites) that can adequately portray heterogeneity.

5. Prioritize Usability And Transparency

Other value frameworks articulate aspirations related to usability and transparency, but these commitments are often made in process documents and are not integrated into the framework. By maintaining usability and transparency as a domain and an integral part of the framework, the PPVF showcases its commitment to ensuring that the framework has a transparent approach and that the information displayed through each application is appropriate for, accessible by, and meaningful to its intended audience.

Patient Perspectives On Value: More Than A Talking Point

The need to incorporate the patient perspective on value has been a key talking point for all types of stakeholders in critiquing value assessment models. It is first on the NPC’s list of “improvements,” and the NPC is far from alone in this perspective. Some framework developers have explicitly articulated different purposes for their frameworks, such as driving payer and policy maker decision making. However, in a health care system in which patients increasingly bear substantial responsibility for the costs of their care, we believe that the patient perspective should be central to any value assessment—no matter the end user of the value determination. It’s time to stop treating this as a lofty, unattainable goal and instead embrace a fully defined framework for measuring value through the patient’s eyes.

Avalere and FasterCures recognize that the current research paradigm does not fully support this approach. However, we can make progress by increasing the focus on measuring patient-centered outcomes in trials and post-market studies, while also developing new tools to create transparency in patient costs. We also should leverage a growing electronic information infrastructure to robustly collect real-world evidence and analyze subgroup data.

The component pieces of the patient perspective on value are increasingly available, and the Avalere-FasterCures PPVF gives us a framework for using them to assess value from the patient’s perspective. It is time for other value framework developers to collaborate with patients and multistakeholder groups such as the PPVF steering committee to better integrate patient perspectives into their own methodologies.

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