In an effort to allocate health care resources most efficiently to the highest value health interventions, payers have begun to adopt formalized health technology assessments (HTAs). An HTA estimates the value of health interventions by estimating their net clinical and economic benefits. Publicly reported and replicable HTA approaches have therefore been gaining a rightful role as one way to increase efficiency in health care expenditures.
However, current standards for the conduct of HTAs may not accurately represent the value to patients of specific interventions because such standards do not measure benefits and costs from the patient perspective. A core pillar of patient-centered research and care is the appropriate selection and use of patient input—for example, around which clinical and economic outcomes to elevate—in designing studies that generate outcomes that matter to patients. Many research teams and disciplines have begun to adopt patient-important outcomes and to account for patient perspectives in decisions about treatment options.
This strategy has the potential to bring the most important decision-making dyad in medicine—patients and their doctors—to the research table. If researchers adopt outcomes that patients say are important, HTA evidence may be considered more useful decision makers across the health care ecosystem, including among payers, employers, policy makers, clinicians, and—importantly—patients.
In recent years, we and others have started to explore ways to incorporate patient input into HTA methods and processes. We developed the Patient-Engaged Healthcare Valuation strategy to elicit patient-important treatment goals in a format that could be synthesized for population-level insights. As proof of concept, RAND and the Innovation and Value Initiative developed a survey instrument for ascertaining and prioritizing goals for people living with rheumatoid arthritis (RA), the output of which can be used for deliberative assessment of both individual treatments and sequences of treatments.
Obtaining patient input is particularly relevant for a disease such as RA, where there is discrepancy between, on one hand, the outcomes included in clinical trials and value assessments, and on the other hand, outcomes that patients most care about. For example, routine blood monitoring is widely used for assessment of disease activity in RA, but many patients report symptoms such as fatigue and functional limits that are not captured in laboratory results—so called “subclinical” manifestations of disease. To date, the best way to understand and evaluate what is most important to patients is to ask patients directly about their own personal goals for care.
Goal Attainment Scaling
The Patient-Engaged Healthcare Valuation strategy draws on methods of goal attainment scaling (GAS), a method validated across multiple disease states, to identify and quantify patient-important outcomes for use in subsequent care and research. GAS was developed to guide clinical decision making through discussion and completion of treatment goals by the patient along with a clinician. Using a survey method to collect goals directly from patients is a way to scale this approach to the population level, yielding outcomes to use in HTA. Goals can be efficiently “crowd-sourced” from a larger patient population survey, with the option for open-ended contribution of goals in addition to rating the importance of a defined set of goals.
Patient goals can be collected by patient representatives within a given community and incorporated with the help of the patient representatives in the next steps of deliberation. By collecting patient-generated goals and understanding their relative importance, the inputs for deliberative methods such as multi-criteria decision analysis can be informed directly by a large number of patients, increasing the representativeness of those inputs.
We tested some of these ideas by working with two RA patient groups—AiArthritis, and the Arthritis Foundation—to co-develop a menu of patient-important goals, supporting their discussion with a review of systematically identified relevant studies, and then by surveying patients about the relative importance of items on the list of goals. Of the 47 respondents we surveyed, the majority were women (93 percent), White (87 percent), and college-educated (72 percent). Most respondents were in their 40s or 50s (range: 28–72 years). Results of this initial survey may apply only to similar populations with like sex, age, education, and race/ethnicity characteristics. Collecting data on population characteristics, disease duration, and disease severity is central to understanding the population to which results may apply. We incorporated input on the approach, project plan, and results from a multistakeholder steering committee throughout the effort.
All of the patient-identified items on the menu were confirmed as important or very important by more than half of patients surveyed (exhibit 1). Several additional goals were recommended by participants, including trying to find the right specialist who would listen to their symptoms and concerns. About half also discussed goals related to their emotional health, including addressing the loneliness or isolation they had experienced, and wanting to find support from others with RA. Some described their goal of wanting to help others with RA. Some respondents wrote in goals that specified aspirational activities, such as lifting weights, practicing yoga, or running.
Exhibit 1: A ranked menu of patient-identified goals in rheumatoid arthritis
Source: Author-analyzed surveys conducted in conjunction with AiArthritis and the Arthritis Foundation.
More than half of respondents further expanded on the financial issues they or others had experienced, including the additional costs associated with living with RA. One discussed the additional time required for daily tasks while living with RA. Others reiterated that dealing with their pain was their most important goal. Several respondents hypothesized that goals would vary with age or time with the disease.
In short, there is much to be learned from goal-based outcomes elicitation from patients. With this work, we established the feasibility of collecting individualized patient goals at scale. The method for goals collection used partnerships with patient organizations for online-only contact and data collection of treatment goals, expanding the goals-based approach from an in-clinic discussion between patient and clinician. With wider “crowd-sourced” data collection using various platforms—such as web-based interfaces and mobile applications—it is possible to include broader input from patient communities in health care valuation, including HTA of specific interventions or treatment sequences.
This work follows on a substantial history of outcomes assessment in RA, including recommendations of the European Alliance of Associations for Rheumatology, Outcome Measures in Rheumatology, and the Common Fund’s Patient-Reported Outcomes Measurement Information System. The method described above, however, focuses in particular on patients’ own care goals and the representation of goals that can be used in a GAS framework. It also enables collection from large samples. To ensure the goal set continues to reflect the patient community’s goals accurately, stakeholders should sustain ongoing open-ended goal elicitation. Periodic checks of goals using patient/clinician GAS discussions will further enhance the goal set’s fidelity to the patient community of interest.
Further data collection would permit stakeholders to identify and distinguish between goals that are RA-disease specific and those that are generic or applicable to other patient communities. Given the key goal of informing HTA, this method maintains a critical connection to disease-specific goals. At the same time, by identifying which goals are relevant across diseases we can establish a foundational or core set of goals upon which different therapeutic areas build.
We see a substantial growth in patient-informed research across the biomedical sciences. Our experience has shown that the frameworks, principles, and methods that support patient and stakeholder engagement in research are far less complicated and much more intuitive than many researchers assume. Advocates of HTA could make substantial headway in the translation of value assessments across health settings by adopting approaches that can be endorsed, understood, and used by patients, clinicians, payers, employers, and policy makers.
Project Steering Committee Members included: Susan Bartlett, PhD, of McGill University; Clifton Bingham, MD, of Johns Hopkins University; Deb Constien, patient representative; Stacy Courtna, patient representative; Julie Eller of the Arthritis Foundation; Erin Giovanetti of Medstar Health; Juan Marcos Gonzalez, PhD, of Duke University School of Medicine; R. Brett McQueen, PhD, of University of Colorado Anschutz Medical Campus; Lawrence “Rick” Phillips, EdD, patient representative; Suz Schrandt of EXxPECT LLC. The authors have funding for several research projects relevant to the content of this article from: the Innovation and Value Initiative, 501c3; PhRMA Foundation, 501c3; and PhRMA, 501c4.