In an effort to allocate health care resources most efficiently to the most valuable health interventions, payers have begun to adopt formalized health technology assessments (HTAs). HTA assesses the value of health interventions by assessing their net clinical and economic benefits. Therefore, publicly reported and reproducible approaches to OST are gaining their due role as one of the ways to increase the effectiveness of health care costs.
However, current standards for conducting OST may not accurately represent the patient value of specific interventions because such standards do not measure benefits and costs from the patient’s perspective. A central 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 improve—in designing trials that generate outcomes that matter to patients. Many research teams and disciplines have begun to embrace patient-important outcomes and consider the patient’s perspective in decisions about treatment options.
This strategy has the potential to bring the most important decision-making dyad in medicine—patients and their physicians—to the research table. If researchers perceive outcomes that patients perceive to be important, OST evidence may be considered more useful for decision-making in the health care ecosystem, including among payers, employers, policymakers, clinicians, and—importantly—patients.
In recent years, we and others have begun to explore ways to incorporate patient input into OST methods and processes. We developed the Patient Engaged Healthcare Assessment Strategy to extract patient-relevant treatment goals in a format that can be synthesized for population-level insights. As a proof-of-concept, RAND and the Institute for Innovation and Value developed a survey tool to identify and prioritize goals for people living with rheumatoid arthritis (RA), the outcome of which can be used to purposefully evaluate both individual treatments and sequences of treatments.
Obtaining information from patients is particularly relevant for a disease such as rheumatoid arthritis, where there is a discrepancy between, on the one hand, the outcomes included in clinical trials and value assessments, and, on the other hand, the outcomes that patients are most interested in . For example, routine blood monitoring is widely used to assess disease activity in rheumatoid arthritis, but many patients report symptoms such as fatigue and functional limitations that are not captured in laboratory results—the so-called “subclinical” manifestations of the disease. To date, the best way to understand and assess what is most important to patients is to ask patients directly about their personal goals of care.
Scaling goal achievement
The patient-engaged healthcare assessment strategy is based on Goal Attainment Scaling (GAS) methods, a method validated across multiple disease states to identify and quantify patient-important outcomes for use in follow-up care and research. The GAS was developed to guide clinical decision-making through the patient’s discussion and implementation of treatment goals together with a clinician. Using a survey method to collect targets directly from patients is a way to scale this approach to the population level, generating results for use in OST. Targets can be effectively “crowd-sourced” from a larger patient population study, with the option of unlimited target input in addition to scoring the importance of a particular set of targets.
Patient goals can be gathered from patient representatives in a given community and incorporated with the patient representatives’ help in subsequent discussion steps. By collecting patient-generated goals and understanding their relative importance, inputs to deliberative methods such as multi-criteria decision analysis can be directly informed by large numbers of patients, increasing the representativeness of these inputs.
We tested some of these ideas by working with two rheumatoid arthritis patient groups—AiArthritis and the Arthritis Foundation—to collaboratively develop a menu of patient-relevant goals, supporting their discussion with a review of systematically identified relevant studies, and then by surveying patients about the relative importance of the items on the target list. Of the 47 respondents we surveyed, the majority were female (93 percent), white (87 percent), and college graduates (72 percent). Most respondents were in their 40s or 50s (range: 28–72 years). The results of this initial study may only apply to populations with similar sex, age, education, and racial/ethnic characteristics. Collection of data on population characteristics, disease duration, and disease severity is fundamental to understanding the population to which the results can be applied. We included information on the approach, project plan, and outcomes of the multi-stakeholder steering committee throughout the effort.
All patient-identified menu items were confirmed as important or very important by more than half of the patients surveyed (Appendix 1). Several additional goals were recommended by participants, including trying to find the right professional to listen to their symptoms and concerns. About half also discussed goals related to their emotional health, including coping with loneliness or isolation they 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 indicated aspirational activities, such as lifting weights, practicing yoga, or running.
Appendix 1: A ranked list of patient-identified goals in rheumatoid arthritis
Source: Studies analyzed by the author, conducted in conjunction with AiArthritis and the Arthritis Foundation.
More than half of the respondents further elaborated on the financial problems they or others have experienced, including the additional costs associated with living with RA. One discusses the extra time required for daily tasks while living with RA. Others reiterated that managing their pain was their most important goal. Several respondents suggested that goals would vary depending on age or duration of illness.
In short, there is much to be learned from deriving goal-based patient outcomes. With this work, we established the feasibility of collecting individualized patient goals at scale. The goal-collection method uses partnerships with patient organizations for online-only contact and data collection about treatment goals, extending the goal-based approach from in-clinic discussion between patient and clinician. With wider crowd-sourced data collection using various platforms – such as web-based interfaces and mobile apps – it is possible to incorporate wider input from patient communities into health care evaluation, including HRT of specific interventions or sequences of treatment.
This work follows a significant history of outcome assessment in RA, including the European Alliance of Rheumatology Associations recommendations, Outcome Measures in Rheumatology, and the Common Fund Patient-Reported Outcomes Information System. The method described above, however, focuses specifically on patients’ own care goals and presenting goals that can be used in a GAS framework. It also allows collection from large samples. To ensure that the goal set continues to accurately reflect the goals of the patient community, stakeholders should maintain open-ended goal elicitation on an ongoing basis. Periodic review of goals using GAS patient/clinician discussions will further improve the fidelity of the goal set to the patient community of interest.
Further data collection would allow stakeholders to identify and differentiate goals that are specific to rheumatoid disease and those that are general or applicable to other patient communities. Given the key goal of informing OST, this method maintains a critical relationship with disease-specific goals. At the same time, by identifying which targets are relevant for different diseases, we can establish a foundational or core set of targets on which different therapeutic areas are built.
We are seeing significant growth in patient-informed research in the biomedical sciences. Our experience shows that the frameworks, principles and methods that support patient and stakeholder engagement in research are far less complex and far more intuitive than many researchers assume. OST advocates could make significant progress in translating value assessments across health care settings by adopting approaches that can be endorsed, understood, and used by patients, clinicians, payers, employers, and policymakers.
Members of the Project Steering Committee include: Susan Bartlett, PhD, McGill University; Clifton Bingham, MD, of Johns Hopkins University; Deb Constien, Patient Representative; Stacey Courtna, Patient Representative; Julie Eller of the Arthritis Foundation; Erin Giovanetti of Medstar Health; Juan Marcos Gonzalez, MD, Duke University School of Medicine; R. Brett McQueen, PhD, of the University of Colorado Anschutz Medical Campus; Lawrence “Rick” Phillips, EdD, Patient Advocate; Suze Schrand of EXxPECT LLC. The authors have funding for several research projects related to the content of this article from: The Value Enhancement Institute, 501c3; PhRMA Foundation, 501c3; and PhRMA, a 501c4.