In the US healthcare system, patient-centered care has been recognized as a key element of high-quality care. Health technology assessment (HTA) organizations and their associated value frameworks have recently begun to consider how to incorporate the patient perspective or patient-centered outcomes more explicitly into their approaches. Additionally, traditional approaches have not evolved to effectively and quantitatively include the patient perspective in the evaluation of treatment value. Because these nascent efforts build on established methods, they typically treat patient-oriented value assessment as tangential or secondary to the central quantitative assessment of value. If the patient perspective is not incorporated in a meaningful way, stakeholders may undervalue treatments that align with patient preferences and needs.

Although a shift toward including the patient perspective in value assessment has started in the US, the impact on HTA and value assessment has been minimal. Most often, patient engagement is limited, and patient perspectives are not quantified and included in cost-effectiveness analyses. Thus, to incorporate patient perspectives in economic evaluations of medical innovations, HTA bodies will need to utilize methods to elicit treatment value drivers from patients, ascertain the relative importance of the value attributes, and properly weight the attributes in a framework that can account for heterogeneity in patient preferences.

Understanding patient preferences for treatment attributes and components of care from which patients derive value is essential in determining appropriate endpoints for value assessment and for patient-physician shared decision making. Shared decision making that incorporates patient preferences has been found to lead to more engaged patients and better patient outcomes. Elements of value that patients find particularly salient are often not quantitatively or qualitatively included in the assessment of a treatment’s value, which may lead to decisions regarding coverage and pricing that do not reflect patients’ preferences and priorities.

After reviewing the shortcomings of current approaches, this paper outlines the evidence-based methods for quantifying patient preferences and practical steps for including them in value assessment. As a case study to understand how value assessment in the US includes patient perspectives, Avalere—in partnership with Inovalon—conducted qualitative research to understand the perspective of patients with triple-negative breast cancer (TNBC). To better understand the attributes of TNBC and the treatments of the disease that resonate with patients, Avalere conducted focus groups to examine the treatment experiences of patients and elicit their treatment-related preferences. Subsequently, we highlighted aspects of TNBC treatment that are not formally and quantitatively captured in many current value assessment frameworks that, if incorporated, would result in value assessments of TNBC treatments that are more holistic and patient centered.

The white paper Infusing the Patient Perspective into Value Assessment is available for download.

Identifying the Elements of TNBC Treatment That Patients Prefer

The focus groups consistently identified several key factors associated with particular medications or interventions that influence initial and subsequent treatment decision making (Figure 1). For initial treatment decisions, a majority of participants (71%) cited survival or quantity of life as a top priority, while almost half (47%) of participants highly valued quality of life. Limiting the duration of treatment was mentioned by 22% of participants as a factor driving treatment choices. However, patients’ out-of-pocket costs for treatment were not a strong driver of initial treatment decision making, as treatments were largely covered by their health plans—though many participants did experience cost barriers for supportive care (e.g., cold capping). While participants rarely considered the costs of treatment as a driver of subsequent treatment decision making (following initial treatments), participants highlighted several other priorities in their decision making for subsequent treatments that are not consistently incorporated into standard HTA (Figure 1).

Note: The size of the boxes reflects the degree of consensus among focus group participants

How Value Assessment Frameworks in the US Incorporate the Patient Perspective

Existing frameworks do not capture many of the key components of TNBC patients’ decision-making calculus. By excluding key treatment characteristics (e.g., treatment duration, mode of administration, treatment schedule, treatment invasiveness), the frameworks do not consider the patient-centered value some medications offer over others. Additionally, by excluding key supportive components associated with treatment (e.g., cold-capping, physical therapy) and short- and long-term sequelae of TNBC/treatments (e.g., fear of recurrence, fertility, loss of female identity) that focus-group participants identified as value drivers, the patient’s disease and treatment experience are not fully captured. Furthermore, the failure to allow for the weighting of value elements (e.g., survival, adverse events, out-of-pocket costs, mode of administration) according to individual patient value sets leads to an implicit assumption of patient homogeneity. Consequently, these value frameworks may not reflect characteristics of TNBC treatment that resonate with patients. Finally, particular frameworks used in the US—including those developed by the American Society for Clinical Oncology (ASCO), the National Comprehensive Cancer Network, and the Institute for Economic and Clinical Review (ICER)—are implemented in a manner that is agnostic to individual patient preferences. Therefore, the value picture painted by the frameworks may not represent reality for many patients.

Rethinking Value Assessment Frameworks

The increasing attention paid to patient perspectives on the value of healthcare services represents an important start in re-thinking frameworks for value assessment. True patient-oriented value assessment requires quantifying and integrating patient-centered outcomes, treatment preferences, and the full spectrum of patient costs into the quantitative methods used to calculate value. While these methods have emerged recently and continue to become more developed, they are not being implemented by ICER, ASCO, and global HTAs like the National Institute for Health and Care Excellence in their value frameworks.

In order to incorporate patient preferences in a quantitative manner, patient-oriented value assessment has to include some degree of weighting by patient (or other stakeholder) preferences, such as through discrete choice experiments and multi-criteria decision analysis, both to understand what benefits are most desired and to ensure that the definition of “costs” better addresses costs to patients that may not show up in traditional healthcare spending calculations. This type of approach will help ensure that decision making about pricing, coverage, and treatment aligns with what patients value.

Funding for this white paper was provided by Gilead Sciences.  Avalere Health retained full editorial control.

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