In an effort to allocate health resources more efficiently to higher-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 acquired a legitimate role as a way to increase efficiency in healthcare spending.
However, current standards for conducting HTAs may not accurately represent the patient value of specific interventions as these standards do not measure benefits and costs from a patient perspective. A key pillar of patient-centered research and care is the appropriate selection and use of patient inputs, for example around which to improve clinical and economic outcomes, in the design of studies that generate important outcomes for patients. Many research groups and disciplines have begun to adopt patient-relevant findings and take patient perspectives into account 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 believe are important, HTA evidence can be seen as the most useful decision-makers across the healthcare ecosystem, including taxpayers, employers, policy makers, physicians, and most importantly, patients. .
In recent years, we and others have begun to explore ways to incorporate patient input into HTA methods and processes. We developed the Patient-Engaged Healthcare Valuation strategy to achieve patient-relevant treatment goals in a format that could be summarized for population-wide insights. As a proof of concept, RAND and the Innovation and Value Institute have developed a survey tool to ascertain and prioritize goals for people with rheumatoid arthritis (RA), the results of which can be used for deliberative assessment of both individual treatments and treatment sequences.
Obtaining patient input is particularly relevant for a disease such as RA, where there is a discrepancy between, on the one hand, the results included in clinical trials and the value assessments and, on the other hand, the outcomes to which patients they care more. For example, routine blood monitoring is widely used for evaluating disease activity in RA, but many patients report symptoms such as fatigue and functional limitations that are not detected in laboratory results, the so-called “subclinical” manifestations of disease. To date, the best way to understand and evaluate what matters most to patients is to ask patients directly about their personal goals for care.
Downsizing of achievement of goals
The Patient-Engaged Healthcare Valuation strategy is based on Goal Sizing (GAS) methods, a method validated across multiple disease states, to identify and quantify patient-relevant outcomes for use in subsequent care and research. GAS was developed to guide clinical decision making by discussing and completing treatment goals by the patient together with a physician. Using a survey method to collect targets directly from patients is one way to scale this approach at the population level, yielding results for use in HTA. Goals can be effectively “publicly sought” by a broader survey of the patient population, with the option for open goal contribution as well as assessing the importance of a defined set of goals.
Patient goals can be collected from patient representatives within a given community and incorporated with the help of patient representatives in the later stages of deliberation. By collecting patient-generated goals and understanding their relative importance, inputs for deliberative methods such as multi-criteria decision analysis can be directly informed by a large number of patients, increasing the representativeness of those inputs.
We tested some of these ideas by working with two groups of RA patients, AiArthritis and Arthritis Foundation, to jointly develop a menu of patient-relevant goals, supporting their discussion by reviewing relevant studies systematically identified and then reviewing patients on the relative importance of the elements in the list of objectives. Of the 47 respondents we interviewed, the majority were women (93%), white (87%) and university educated (72%). Most of the respondents were between 40 and 50 years old (range: 28-72 years). The results of this initial survey can only be applied to similar populations with similar characteristics in gender, age, education, and race / ethnicity. Collecting data on population characteristics, disease duration and disease severity is critical to understanding the population to which the results can apply. We incorporated input on the approach, project plan and results of a multi-stakeholder steering committee throughout the effort.
All menu items identified by patients were confirmed as important or very important by more than half of the patients interviewed (Figure 1). Participants recommended several additional goals, including trying to find the right specialist who listened to their symptoms and concerns. About half also discussed goals related to their emotional health, including coping with the loneliness or isolation they had experienced and a desire to find support from others with RA. Some have described their goal of wanting to help others with RA. Some respondents wrote in goals that specified ambitious activities, such as lifting weights, practicing yoga, or running.
Attachment 1: A classified menu of patient-identified goals in rheumatoid arthritis
Source: Author analyzed surveys conducted in collaboration with AiArthritis and Arthritis Foundation.
More than half of the respondents further delved into the financial problems they or others had experienced, including the additional costs associated with living with RA. One discussed the additional time required for daily activities while living with RA. Others reiterated that coping with their pain was their most important goal. Several respondents speculated that the goals would vary with age or time with the disease.
In short, there is a lot to be learned from patients eliciting goal-based outcomes. With this work, we have established the feasibility of collecting individualized patient goals on a large scale. The method for goal collection used partnerships with patient organizations for online-only contact and collection of treatment goal data, expanding the goal-based approach from an in-clinic discussion between patient and physician. With a broader collection of “public” data using various platforms, such as web-based interfaces and mobile applications, it is possible to include broader input from patient communities in health care assessment, including HTA of specific interventions or treatment sequences.
This work follows a substantial history of outcome assessment in RA, including recommendations from the European Alliance of Associations for Rheumatology, Outcome Measurements in Rheumatology, and the Joint Fund Patient Reported Outcome Measurement Information System. The method described above, however, focuses specifically on patient care goals and the representation of goals that can be used in a GAS framework. It also allows for collection from large samples. To ensure that the set goal continues to accurately reflect the goals of the patient community, stakeholders should support the continuous elicitation of goals indefinitely. Periodic goal checks through patient / clinician GAS discussions will further strengthen the loyalty of the goal set to the patient community of interest.
Further data collection would allow stakeholders to identify and distinguish between RA disease specific targets and those that are generic or applicable to other patient communities. Given the key goal of informing HTA, this method maintains a critical link with the specific goals of the disease. At the same time, by identifying which goals are relevant to all diseases, we can establish a set of fundamental or fundamental goals on which to build different therapeutic areas.
We see substantial growth in patient-informed research in the biomedical sciences. Our experience has shown that the structures, principles and methods that support patient and stakeholder engagement in research are far less complicated and far more intuitive than many researchers assume. Advocates of HTA could make substantial progress in translating value assessments into all healthcare settings by adopting approaches that can be approved, understood and used by patients, doctors, payers, employers and policy makers.
Note from the authors
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 the Anschutz Medical Campus of the University of Colorado; 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: Institute for Value Improvement, 501c3; PhRMA Foundation, 501c3; and PhRMA, 501c4.