Health Technology Assessment: Evidence Generation Methods For Patient-Driven Values

Editor’s Note

This article is part of a Health Affairs Forefront short series, “Enhancing Value By Evaluating Health Care Services.” The series discusses ways to extend the use of tools for clinical and economic evaluation beyond medical technologies to the services and procedures that account for the bulk of health care spending; the goal is to create a more robust evidence base for the effectiveness and value of health care services. The articles in the series were completed with support for the authors from the Research Consortium for Health Care Value Assessment, a partnership between Altarum and VBID Health, through a grant from the Pharmaceutical Research and Manufacturers of America (PhRMA). PhRMA extended complete independence to Altarum to select researchers and specific topics. Health Affairs retained review and editing rights.

Innovations in health technology often represent important advances in improving health outcomes, but at what cost, and to whom? Health technology assessment (HTA) hinges on the perspective used for evaluation. While HTA is traditionally anchored on the payer’s perspective, incorporating the patient experience is gaining traction globally. Methods to capture the patient perspective exist, but their adaptation to HTA is still emerging. Furthermore, uncertainty remains as to which value elements are important to patients and how best to incorporate the patient’s perspective into an HTA evaluation.

The Patient-driven Values in Health care Evaluation (PAVE) Center at the University of Maryland School of Pharmacy was established in 2018 with funding from the PhRMA Foundation as a Center of Excellence in Value Assessment. The PAVE Center’s mission is to bring together patients and other stakeholders with the goal to co-produce reliable and meaningful value assessments to support patient-centered health care decision-making. Central to achieving this goal will be addressing a key gap in HTA methods that is applicable to all health care products and services: incorporating the elements of health care that patients value most into value assessments.

The PAVE Center’s patient-informed value elements, which encompass attributes of treatment effects, access, cost, and life and social impacts, were conceptualized from the patient perspective with continuous engagement of patient stakeholders. The PAVE Center is building off these elements and integrating two key lines of research—stated preferences and economic evaluation—with the primary goal of testing novel methods to generate evidence of patient values in health care services.

Stated Preferences

Stated preferences methods have been used to study preferences and values for health care services, treatments, and outcomes. The theoretical premise is that individuals consider jointly different attributes of a health care service and make a choice depending upon their preferences and utility for one attribute over another. These methods are well-suited to quantify the patient perspective, which is essential for translation to HTA.

Economic Evaluations

Economic evaluations for HTA rely on modeling methods to estimate the costs and outcomes of treatment, with a focus on the value domains of quality and quantity of life, often measured by quality-adjusted life-years (QALYs). Incorporating additional value domains, such as patient preferences for novel value elements, may be achieved through several approaches including quantifying the domains and harmonizing them into model inputs. Since stated preferences approaches are based on the premise that people generally choose what is most important to them—in other words, what they value—this information may 1) inform economic evaluation model inputs so they reflect the most- and least-preferred intervention attributes, 2) be used to investigate trade-offs between different attributes, and 3)be applied to health state utilities.

The PAVE Center Application Of Emerging Methods For Patient Perspectives

A common application of stated preference methods is discrete choice experiments (DCE). These experiments elicit preferences by allowing individuals to choose between hypothetical, mutually exclusive scenarios. DCE methods are especially relevant for obtaining a quantifiable measure of the relative importance (i.e., importance scores) of health care intervention attributes that may be included in economic evaluations. The PAVE Center is applying DCE methods to elicit and quantify patient preferences for attributes of health care interventions and is investigating methods for translating this information into economic evaluations to inform a patient-centered value assessment. The PAVE Center patient-informed value elements are central to this work. A multi-phase process with continuous stakeholder engagement is essential to navigating the stages of elicitation, quantification, and translation of the patient-informed value elements to a patient-centered value assessment. While the PAVE Center patient-informed value elements are disease agnostic, the application of the value elements generally is condition-specific.

Elicitation of the attributes of a health care service or intervention that are important to individuals begins with the conceptual model of patient-informed value elements. Using qualitative methods, we narrow the value elements within each of the five domains (i.e., treatment effects, treatment access, treatment costs, and life and social impacts) to those most important to individuals. Iterative feedback from the patient stakeholders and persons living with the target condition contextualizes the general value element to an attribute of a health care intervention or service that influences decision-making. Patient input guides the selection of five to seven key attributes and the language refinement to mimic a real-life decision-making scenario.

Quantification of preferences for the attributes elicited from patients is accomplished through a DCE. We display anywhere from five to seven attributes in a profile and, for each question, provide up to three different profiles to select from. The DCE questions are incorporated into a survey, and results are analyzed using a standard regression model. The analysis results generate a mean score for each attribute, which can then be ranked in order of relative importance.

Translation of the preference utility betas into an economic evaluation has been investigated with several approaches. Lancsar et al. (2011) used DCE to calculate distributional weights for QALYs. While an oft-cited concern of expanding value elements is the double-counting of outcomes, more recent evidence illustrates how to overcome this. Reed et al. (2021) used DCE to quantify the value of hope, where this construct was isolated from gains in quality and quantity of life, thereby avoiding the potential issue of double-counting benefits through both QALYs and the individual novel value elements themselves. These studies provide a basis for applying the quantified preferences for the PAVE value elements, currently being tested in the two clinical areas described here.

Applied Case Studies From The PAVE Center Patient-Driven Value Elements

Case Study 1: Chronic Obstructive Pulmonary Disease

The goals of this project were to: 1) prioritize PAVE patient-informed value elements among individuals living with chronic obstructive pulmonary disease (COPD); 2) compare patients’ priorities to model inputs and parameters commonly used in economic evaluations of COPD; and 3) quantify the most important COPD elements to translate these into future evaluations. The prioritization of these elements revealed that COPD patients prioritized impacts on their physical abilities, as well as attributes of treatment such as medication frequency and side effects.

Despite this, previous models for COPD HTA do not necessarily address side effects or medication frequency and are operationalized mainly through exacerbations and lung function. Lung function impacts physical abilities, a domain typically measured through quality-of-life instruments. However, we do not have the means to understand the importance of these elements in relation to each other. Current efforts are focused on developing an instrument to quantify these elements, along with other important factors for COPD, such as out-of-pocket costs and access to providers. Our team completed a pre-test of the DCE alongside a clinical questionnaire.

Ultimately, the goal is to quantify preferences by defining patient subtypes, potentially characterized by not only their preferences but also their clinical severity and symptom burden. These results will guide translation into economic inputs, subgroups, and other approaches for augmented cost-effectiveness analysis.

Case Study 2: Major Depressive Disorder

The project on patient preferences for attributes of treatment for adults with major depressive disorder (MDD) is a collaboration between the PAVE Center and the Innovation and Value Initiative. The goal of this project is to determine how patient-informed value elements can be incorporated into the value assessment of treatment options for MDD. The specific aims are: 1) To identify through a stakeholder-engaged process the PAVE patient-informed value elements that are important to individuals living with MDD; 2) To elicit patient priorities for treatment attributes to manage MDD; and 3) To identify the patient-informed treatment attributes that can be incorporated in an economic model for MDD.

The study enrolled adults age 18 and older with MDD who did not have a co-existing bipolar disorder and who did not have post-partum depression. Adults with MDD and a diverse stakeholder advisory group were engaged in an iterative manner to elicit, using qualitative research methods, the most important elements influencing their treatment decisions. From the elicited elements, we derive attributes for inclusion in a DCE, which is included in a survey to collect information about treatment preferences, depression symptoms, and sociodemographic characteristics. This project will generate empirical evidence of the trade-offs made between attributes, e.g., feeling hopeful, being productive, feeling treatment effects, and paying out-of-pocket, and will inform inputs for a patient-centered economic evaluation.

Future Applications Of Patient Perspectives In Health Care Value Assessment

Applying patient-centered methods to economic evaluations or other types of HTA across all types of health care services will provide important tools to drive value and efficiency. For example, an important area in need of patient-centered value assessment is health services delivery technologies, such as telehealth, digital health, and other related telecommunication applications. During the COVID-19 pandemic, the need for telehealth care changed the way patients interact with the health care system and the policies governing reimbursement for remote patient care (e.g., temporary expansion of services covered and reimbursement to allow for audio-only consultations). Although telehealth utilization decreased as lockdowns and other restrictions eased, patients express willingness to continue using telehealth services, and it remains to be seen if expanded reimbursement for these services will continue after the pandemic.

Assessing the value of telehealth from the patients’ perspective will be an important consideration as we emerge from the pandemic and face decisions about the value of continuing to reimburse for remote care. For this purpose, the PAVE patient-informed value elements could be integrated as model inputs into technology-oriented assessments of telehealth services to determine their impact on health care. Integrating the value elements is also intended to distinguish for whom, for which conditions, and under what types of modalities (e.g., live video, mobile health, or store and forward) low or high-value telehealth care is provided. For example, we could determine how patients with major depression treated with telepsychiatry value this type of care based on elements related to treatment access (e.g., available treatment or appropriateness of care) or treatment cost (e.g., affordability or reimbursed care). Furthermore, integrating the value elements into telehealth assessments potentially would address existing information needs for generating recommendations and making decisions on more permanent telehealth reimbursement policies. 

Currently, researchers at the PAVE Center are in the early stages of this research. Building upon conceptual models of patient engagement in telehealth care services, the PAVE patient-informed value elements will be applied to generate empirical data that can support future patient-driven value assessment of telehealth care.

Impact Of This Work In Patient-Driven Value Assessment

We hope the PAVE Center’s work will have a downstream impact on health care policies that facilitate access to and reimbursement for health care services that are important to patients and improve health outcomes. The patient-centered approach for evidence generation of patient-informed health care values can be adopted by researchers, value framework developers, payers, and policymakers. Our goal is to embed patient-centered value evidence in methods that the HTA field relies on, increasing the ease of application and uptake of our approach.

Authors’ Note

Drs. dosReis, Slejko, and Amill-Rosario are affiliated with the PAVE Center, which is funded by the PhRMA Foundation.