Informing Patient-Centered Care Through Stakeholder Engagement and Highly Stratified Quantitative Benefit-Harm Assessments.

OBJECTIVES: In a previous project aimed at informing patient-centered care for people with multiple chronic conditions, we performed highly stratified quantitative benefit-harm assessments for 2 top priority questions. In this current work, our goal was to describe the process and approaches we developed and to qualitatively glean important elements from it that address patient-centered care. METHODS: We engaged patients, caregivers, clinicians, and guideline developers as stakeholder representatives throughout the process of the quantitative benefit-harm assessment and investigated whether the benefit-harm balance differed based on patient preferences and characteristics (stratification). We refined strategies to select the most applicable, valid, and precise evidence. RESULTS: Two processes were important when assessing the balance of benefits and harms of interventions: (1) engaging stakeholders and (2) stratification by patient preferences and characteristics. Engaging patients and caregivers through focus groups, preference surveys, and as co-investigators provided value in prioritizing research questions, identifying relevant clinical outcomes, and clarifying the relative importance of these outcomes. Our strategies to select evidence for stratified benefit-harm assessments considered consistency across outcomes and subgroups. By quantitatively estimating the range in the benefit-harm balance resulting from true variation in preferences, we clarified whether the benefit-harm balance is preference sensitive. CONCLUSIONS: Our approaches for engaging patients and caregivers at all phases of the stratified quantitative benefit-harm assessments were feasible and revealed how sensitive the benefit-harm balance is to patient characteristics and individual preferences. Accordingly, this sensitivity can suggest to guideline developers when to tailor recommendations for specific patient subgroups or when to explicitly leave decision making to individual patients and their providers.

Outcome preferences of older people with multiple chronic conditions and hypertension: a cross-sectional survey using best-worst scaling.

BACKGROUND: Older people with hypertension and multiple chronic conditions (MCC) receive complex treatments and face challenging trade-offs. Patients' preferences for different health outcomes can impact multiple treatment decisions. Since evidence about outcome preferences is especially scarce among people with MCC our aim was to elicit preferences of people with MCC for outcomes related to hypertension, and to determine how these outcomes should be weighed when benefits and harms are assessed for patient-centered clinical practice guidelines and health economic assessments. METHODS: We sent a best-worst scaling preference survey to a random sample identified from a primary care network of Kaiser Permanente (Colorado, USA). The sample included individuals age 60 or greater with hypertension and at least two other chronic conditions. We assessed average ranking of patient-important outcomes using conditional logit regression (stroke, heart attack, heart failure, dialysis, cognitive impairment, chronic kidney disease, acute kidney injury, fainting, injurious falls, low blood pressure with dizziness, treatment burden) and studied variation across individuals. RESULTS: Of 450 invited participants, 217 (48%) completed the survey, and we excluded 10 respondents who had more than two missing choices, resulting in a final sample of 207 respondents. Participants ranked stroke as the most worrisome outcome and treatment burden as the least worrisome outcome (conditional logit parameters: 3.19 (standard error 0.09) for stroke, 0 for treatment burden). None of the outcomes were always chosen as the most or least worrisome by more than 25% of respondents, indicating that all outcomes were somewhat worrisome to respondents. Predefined subgroup analyses according to age, self-reported life-expectancy, degree of comorbidity, number of medications and antihypertensive treatment did not reveal meaningful differences. CONCLUSIONS: Although some outcomes were more worrisome to patients than others, our results indicate that none of the outcomes should be disregarded for clinical practice guidelines and health economic assessments.

Balance of benefits and harms of different blood pressure targets in people with multiple chronic conditions: a quantitative benefit-harm assessment.

OBJECTIVE: Recent studies suggest that a systolic blood pressure (SBP) target of 120 mm Hg is appropriate for people with hypertension, but this is debated particularly in people with multiple chronic conditions (MCC). We aimed to quantitatively determine whether benefits of a lower SBP target justify increased risks of harm in people with MCC, considering patient-valued outcomes and their relative importance. DESIGN: Highly stratified quantitative benefit-harm assessment based on various input data identified as the most valid and applicable from a systematic review of evidence and based on weights from a patient preference survey. SETTING: Outpatient care. PARTICIPANTS: Hypertensive patients, grouped by age, gender, prior history of stroke, chronic heart failure, chronic kidney disease and type 2 diabetes mellitus. INTERVENTIONS: SBP target of 120 versus 140 mm Hg for patients without history of stroke. PRIMARY AND SECONDARY OUTCOME MEASURES: Probability that the benefits of a SBP target of 120 mm Hg outweigh the harms compared with 140 mm Hg over 5 years (primary) with thresholds >0.6 (120 mm Hg better), <0.4 (140 mm Hg better) and 0.4 to 0.6 (unclear), number of prevented clinical events (secondary), calculated with the Gail/National Cancer Institute approach. RESULTS: Considering individual patient preferences had a substantial impact on the benefit-harm balance. With average preferences, 120 mm Hg was the better target compared with 140 mm Hg for many subgroups of patients without prior stroke, especially in patients over 75. For women below 65 with chronic kidney disease and without diabetes and prior stroke, 140 mm Hg was better. The analyses did not include mild adverse effects, and apply only to patients who tolerate antihypertensive treatment. CONCLUSIONS: For most patients, a lower SBP target was beneficial, but this depended also on individual preferences, implying individual decision-making is important. Our modelling allows for individualised treatment targets based on patient preferences, age, gender and co-morbidities.

A benefit-harm analysis of adding basal insulin vs. sulfonylurea to metformin to manage type II diabetes mellitus in people with multiple chronic conditions.

OBJECTIVES: The benefits and harms of diabetes treatments need to be carefully weighed in people with type II diabetes mellitus (DM) and multiple chronic conditions (MCCs). Our objective was to quantitatively assess the benefits and harms of the addition of basal insulin (insulin) vs. sulfonylurea (SU) to metformin in people with DM and MCCs. STUDY DESIGN AND SETTING: Data inputs into the benefit-harms analysis included (1) baseline risks of patient-centered outcomes (death, myocardial infarction, stroke, severe hypoglycemia, diarrhea, nausea) from cohorts and trials; (2) treatment effects for the addition of insulin vs. SU from a network meta-analysis; and (3) patient preference survey for outcome weights. Statistical analysis calculated the probability that adding insulin has greater benefits than harms, when compared with an SU, overall and by prespecified subgroups. RESULTS: Including the six outcomes, the probability of net benefit for insulin compared with SU was similar, across subgroups by age and diabetes duration (probability range, using conditional logit weights: 0.44-0.56). Adding patient preferences for treatment burden associated with insulin injections shifted the probability to favor SU over insulin (probability range, using conditional logit weights: 0.01-0.12). CONCLUSION: In people with DM and MCCs, we demonstrated incomplete evidence to conclude if basal insulin or SU should be added in people with DM and MCCs on metformin alone. The benefit-harm balance was sensitive to treatment preferences, that is., perceived treatment burden, indicating the importance of shared-decision making in caring for people with MCCs who are at high risk for experiencing harms associated with diabetes management.

An environmental scan of shared access to patient portals.

We sought to understand the comprehensiveness of consumer-oriented information describing the availability of shared access to adult patient portals from publicly reported information on institutional websites of 20 large and geographically diverse health systems. All 20 health systems reported that they offer patients the ability to share access to their patient portal account with a family member or friend; however, the comprehensiveness of information regarding registration procedures, features, and terminology varied widely. Half of the systems (n = 10) reported having shared access available on their patient portal registration webpage. Few systems (n = 2) reported affording patients the ability to differentiate specific role-based privileges. No systems reported uptake of shared access among adult patients, which was variably described as "proxy," "caregiver," "parental," or "delegate" access. Findings suggest that engaging families through health information technology will require greater efforts to promote awareness and differentiate privileges that respect patients' choice and control in information-sharing preferences.

Engaging Stakeholders to Inform Clinical Practice Guidelines That Address Multiple Chronic Conditions.

BACKGROUND: Having more than one chronic condition is common and is associated with greater health care utilization, higher medication burden and complexity of treatment. However, clinical practice guidelines (CPGs) do not routinely address the balance between harms and benefits of treatments for people with multiple chronic conditions (MCCs). OBJECTIVE: To partner with the Kaiser Permanente Integrated Cardiovascular Health (ICVH) program to engage multiple stakeholders in a mixed-methods approach in order to: 1) identify two high-priority clinical questions related to MCCs, and 2) understand patients' and family caregivers' perceptions of meaningful outcomes to inform benefit/harm assessments for these two high-priority questions. These clinical questions and outcomes will be used to inform CPG recommendations for people with MCCs. DESIGN AND PARTICIPANTS: The ICVH program provided 130 topics rank-ordered by the potential for finding evidence that would change clinical recommendations regarding the topic. We used a modified Delphi method to identify and reword topics into questions relevant to people with MCCs. We used two sets of focus groups (n = 27) to elicit patient and caregiver perspectives on two important research questions and relevant patient-important outcomes on benefit/harm balance for people with MCCs. KEY RESULTS: Co-investigators, patients and caregivers identified "optimal blood pressure goals" and "diabetes medication management" as important clinical topics for CPGs related to people with MCCs. Stakeholders identified a list of relevant outcomes to be addressed in future CPG development including 1) physical function and energy, 2) emotional health and well-being, 3) avoidance of treatment burden, side effects and risks, 4) interaction with providers and health care system, and 5) prevention of adverse long-term health outcomes. CONCLUSIONS: Through the application of a mixed-methods process, we identified the questions regarding optimal blood pressure goals and diabetes medication management, along with related patient-centered outcomes, to inform novel evidence syntheses for those with MCCs. This study provides the lessons learned and a generalizable process for CPG developers to engage patient and caregivers in priority-setting for the translation of evidence into future CPGs. Ultimately, engaging patient and stakeholders around MCCs could improve the relevance of CPGs for the care of people with MCCs.

The double helix: when the system fails the intertwined needs of caregiver and patient.

When a family caregiver becomes injured, she learns the difficulties--and costs--of caring for herself and her chronically ill husband at the same time.

Understanding the context of health for persons with multiple chronic conditions: moving from what is the matter to what matters.

PURPOSE: An isolated focus on 1 disease at a time is insufficient to generate the scientific evidence needed to improve the health of persons living with more than 1 chronic condition. This article explores how to bring context into research efforts to improve the health of persons living with multiple chronic conditions (MCC). METHODS: Forty-five experts, including persons with MCC, family and friend caregivers, researchers, policy makers, funders, and clinicians met to critically consider 4 aspects of incorporating context into research on MCC: key contextual factors, needed research, essential research methods for understanding important contextual factors, and necessary partnerships for catalyzing collaborative action in conducting and applying research. RESULTS: Key contextual factors involve complementary perspectives across multiple levels: public policy, community, health care systems, family, and person, as well as the cellular and molecular levels where most research currently is focused. Needed research involves moving from a disease focus toward a person-driven, goal-directed research agenda. Relevant research methods are participatory, flexible, multilevel, quantitative and qualitative, conducive to longitudinal dynamic measurement from diverse data sources, sufficiently detailed to consider what works for whom in which situation, and generative of ongoing communities of learning, living and practice. Important partnerships for collaborative action include cooperation among members of the research enterprise, health care providers, community-based support, persons with MCC and their family and friend caregivers, policy makers, and payers, including government, public health, philanthropic organizations, and the business community. CONCLUSION: Consistent attention to contextual factors is needed to enhance health research for persons with MCC. Rigorous, integrated, participatory, multimethod approaches to generate new knowledge and diverse partnerships can be used to increase the relevance of research to make health care more sustainable, safe, equitable and effective, to reduce suffering, and to improve quality of life.