Treatment goals for rheumatoid arthritis: patient engagement and goal collection.

Aim: We developed the Patient-Engaged Health Technology Assessment strategy for survey-based goal collection from patients to yield patient-important outcomes suitable for use in multi-criteria decision analysis. Methods: Rheumatoid arthritis patients were recruited from online patient networks for proof-of-concept testing of goal collection and prioritization using a survey. A Project Steering Committee and Expert Panel rated the feasibility of scaling to larger samples. Results: Survey respondents (n = 47) completed the goal collection exercise. Finding effective treatments was rated by respondents as the most important goal, and reducing stiffness was rated as the least important. Feedback from our steering committee and expert panel support the approach's feasibility for goal identification and ranking. Conclusion: Goals relevant for treatment evaluation can be identified and rated for importance by patients to permit wide input from patients with lived experience of disease.

Implications of response shift for micro-, meso-, and macro-level healthcare decision-making using results of patient-reported outcome measures.

PURPOSE: Results of patient-reported outcome measures (PROMs) are increasingly used to inform healthcare decision-making. Research has shown that response shift can impact PROM results. As part of an international collaboration, our goal is to provide a framework regarding the implications of response shift at the level of patient care (micro), healthcare institute (meso), and healthcare policy (macro). METHODS: Empirical evidence of response shift that can influence patients' self-reported health and preferences provided the foundation for development of the framework. Measurement validity theory, hermeneutic philosophy, and micro-, meso-, and macro-level healthcare decision-making informed our theoretical analysis. RESULTS: At the micro-level, patients' self-reported health needs to be interpreted via dialogue with the clinician to avoid misinterpretation of PROM data due to response shift. It is also important to consider the potential impact of response shift on study results, when these are used to support decisions. At the meso-level, individual-level data should be examined for response shift before aggregating PROM data for decision-making related to quality improvement, performance monitoring, and accreditation. At the macro-level, critical reflection on the conceptualization of health is required to know whether response shift needs to be controlled for when PROM data are used to inform healthcare coverage. CONCLUSION: Given empirical evidence of response shift, there is a critical need for guidelines and knowledge translation to avoid potential misinterpretations of PROM results and consequential biases in decision-making. Our framework with guiding questions provides a structure for developing strategies to address potential impacts of response shift at micro-, meso-, and macro-levels.

Engaging Patients and Other Non-Researchers in Health Research: Defining Research Engagement.

With the increase in patient and consumer activism through the late twentieth century and into this century, patient roles in research evolved into a new model of research engagement, with patients serving as active advisors and co-leading or leading clinical research. By requiring active engagement of patients and other stakeholders, several government research funders have advanced this model, particularly in Canada, the United States (US), United Kingdom (UK), and Australia. A consortium of individuals from these countries formed a Multi-Stakeholder Engagement (MuSE) consortium to examine critical issues in engaged research, establish consensus on definitions, and provide guidance for the field, beginning with an overview of how to involve stakeholders in health research (Concannon et al. J Gen Intern Med. 2019;34(3):458-463) and continuing here with an examination of definitions of research engagement. The political and advocacy roots of engaged research are reflected in definitions. Engagement is conceptualized with reference to research project goals, from informing specific clinical decisions to informing health-system level decisions. Political and cultural differences across countries are evident. Some of these government funders focus on empirical rather than ethical rationales. In countries with centralized health technology assessment, the link between societal values and engaged research is explicit. Ethical rationales for engagement are explicit in most of the published literature on research engagement. Harmonization of definitions is recommended so that research engagement elements, methods, and outcomes and impacts can be clearly examined and understood, and so that the field of research engagement can proceed from a clear conceptual foundation. Specific recommendations for terminology definitions are provided. Placing engaged research on a continuum from specific clinical decisions to more global public and social justice concerns clarifies the type of engaged research, supports appropriate comparisons, and improves the rigor of engaged research methods. The results help identify knowledge gaps in this growing field.

Patient, caregiver and clinician views on engagement in comparative effectiveness research.

AIM: Describe patient, caregiver and clinician views toward engagement as partners in health research. MATERIALS & METHODS: Online surveys of patients and caregivers managing rare (n = 560 patients, n = 609 caregivers) or chronic conditions (n = 762 patients, n = 776 caregivers) and practicing clinicians (n = 638). RESULTS: Over half of respondents were unfamiliar with the concept of partnering with researchers but most expressed interest in working in a research partnership. Potential facilitators endorsed were ensuring research is meaningful, applying results in an understandable way, and sharing results. Lack of time is a potential barrier. Clinicians were most interested in helping researchers decide on intervention comparisons and identifying implications for clinical practice. CONCLUSION: Patients, caregivers and clinicians are interested in research roles that emphasize usefulness and understandability of research.

Patient and Stakeholder Engagement in the PCORI Pilot Projects: Description and Lessons Learned.

BACKGROUND: Patients and healthcare stakeholders are increasingly becoming engaged in the planning and conduct of biomedical research. However, limited research characterizes this process or its impact. OBJECTIVE: We aimed to characterize patient and stakeholder engagement in the 50 Pilot Projects funded by the Patient-Centered Outcomes Research Institute (PCORI), and identify early contributions and lessons learned. DESIGN: A self-report instrument was completed by researchers between 6 and 12 months following project initiation. PARTICIPANTS: Forty-seven principal investigators or their designees (94 % response rate) participated in the study. MAIN MEASURES Self-report of types of stakeholders engaged, stages and levels of engagement, facilitators and barriers to engagement, lessons learned, and contributions from engagement were measured. KEY RESULTS: Most (83 %) reported engaging more than one stakeholder in their project. Among those, the most commonly reported groups were patients (90 %), clinicians (87 %), health system representatives (44 %), caregivers (41 %), and advocacy organizations (41 %). Stakeholders were commonly involved in topic solicitation, question development, study design, and data collection. Many projects engaged stakeholders in data analysis, results interpretation, and dissemination. Commonly reported contributions included changes to project methods, outcomes or goals; improvement of measurement tools; and interpretation of qualitative data. Investigators often identified communication and shared leadership strategies as "critically important" facilitators (53 and 44 % respectively); lack of stakeholder time was the most commonly reported challenge (46 %). Most challenges were only partially resolved. Early lessons learned included the importance of continuous and genuine partnerships, strategic selection of stakeholders, and accommodation of stakeholders' practical needs. CONCLUSIONS: PCORI Pilot Projects investigators report engaging a variety of stakeholders across many stages of research, with specific changes to their research attributed to engagement. This study identifies early lessons and barriers that should be addressed to facilitate engagement. While this research suggests potential impact of stakeholder engagement, systematic characterization and evaluation of engagement at multiple stages of research is needed to build the evidence base.

Engaging patients and stakeholders in research proposal review: the patient-centered outcomes research institute.

The inaugural round of merit review for the Patient-Centered Outcomes Research Institute (PCORI) in November 2012 included patients and other stakeholders, as well as scientists. This article examines relationships among scores of the 3 reviewer types, changes in scoring after in-person discussion, and the effect of inclusion of patient and stakeholder reviewers on the review process. In the first phase, 363 scientists scored 480 applications. In the second phase, 59 scientists, 21 patients, and 31 stakeholders provided a "prediscussion" score and a final "postdiscussion" score after an in-person meeting for applications. Bland-Altman plots were used to characterize levels of agreement among and within reviewer types before and after discussion. Before discussion, there was little agreement among average scores given by the 4 lead scientific reviewers and patient and stakeholder reviewers. After discussion, the 4 primary reviewers showed mild convergence in their scores, and the 21-member panel came to a much stronger agreement. Of the 25 awards with the best (and lowest) scores after phase 2, only 13 had ranked in the top 25 after the phase 1 review by scientists. Five percent of the 480 proposals submitted were funded. The authors conclude that patient and stakeholder reviewers brought different perspectives to the review process but that in-person discussion led to closer agreement among reviewer types. It is not yet known whether these conclusions are generalizable to future rounds of peer review. Future work would benefit from additional data collection for evaluation purposes and from long-term evaluation of the effect on the funded research.

Improving dementia care: the role of screening and detection of cognitive impairment.

The value of screening for cognitive impairment, including dementia and Alzheimer's disease, has been debated for decades. Recent research on causes of and treatments for cognitive impairment has converged to challenge previous thinking about screening for cognitive impairment. Consequently, changes have occurred in health care policies and priorities, including the establishment of the annual wellness visit, which requires detection of any cognitive impairment for Medicare enrollees. In response to these changes, the Alzheimer's Foundation of America and the Alzheimer's Drug Discovery Foundation convened a workgroup to review evidence for screening implementation and to evaluate the implications of routine dementia detection for health care redesign. The primary domains reviewed were consideration of the benefits, harms, and impact of cognitive screening on health care quality. In conference, the workgroup developed 10 recommendations for realizing the national policy goals of early detection as the first step in improving clinical care and ensuring proactive, patient-centered management of dementia.

A qualitative assessment of the concept of dependence in Alzheimer's disease.

BACKGROUND: The Dependence Scale (DS) was designed to assess levels of patient need for care due to deficits typical of Alzheimer's disease (AD). This study examined content validity of the DS based on input from patients, caregivers, and clinicians. METHODS: Qualitative interviews with experts, patients, and caregivers were used to collect information on the concept of dependence and to assess content validity. RESULTS: Nine clinicians rated item relevance ''high'' with consensus on the primacy of functional abilities and dependence in the measurement of AD progression. Twenty-two US, 11 UK, and 14 informal caregivers from Spain participated in focus groups; 18 patients participated in 3 separate focus groups. Discussion supported DS hierarchy of dependence, capture of mild-to-severe dependence, suitability of response options, and short recall time frame. CONCLUSIONS: Clinicians, caregivers, and patients support content validity of the DS in mild-to-moderate AD. The DS may be valuable to capture dependence within future clinical dementia trials.

The patient experience of depression and remission: focus group results.

Few depression measures were developed with the patient perspective. To obtain patient views on depression and early symptom resolution, 4 focus groups (N=31) were conducted. Patients also completed the Quick Inventory of Depressive Symptomology (Self-Report), SF-36, and a rating of the bothersomeness and significance of 12 symptoms of depression. Transcripts were reviewed for major themes. Irritability was a key symptom and it remitted earlier than others. Important to participants were low mood, low energy, lack of motivation, lack of focus/concentration, feelings of guilt, self-critical thoughts, feeling overwhelmed, lack of enjoyment, hypersomnia, restlessness, anger, and irritability. Gender differences emerged; most men reported irritability as one of the first symptoms to remit; for women, motivation level and energy commonly remitted first. Results suggest that new measures of treatment outcome should encompass irritability, anger, and ability to cope with life stressors.

Commentary on "Health economics and the value of therapy in Alzheimer's disease." Report from the Alzheimer's Association Research Roundtable on patient-reported outcomes and dementia research.

The release of the Food and Drug Administration's draft guidance on the patient-reported outcomes (PROs) measurement in February 2006 has specific implication for outcomes research in dementia. Advances in treatments for probable Alzheimer's disease present unique challenges to the measurement of clinical outcomes. Standards adapted for PROs measurement can be applied to provide an alternative perspective on efficacy, and to access a more comprehensive range of outcomes than is currently assessed in dementia trials. The draft guidance provides only limited direction with regard to proxy measurements, but the principles of PROs assessment should be applied to proxy respondent measures. Taking a broad view of outcome measurements can offset limitations related to utility assessment and the use of data in pharmacoeconomic evaluations of dementia treatments. Measurements that formally encompass patients and caregivers can enhance our understanding of the disorder, and improve conclusions about treatment effectiveness.

Depression and health-related quality of life for low-income African-American women in the U.S.

The health-related quality of life (HRQL) impact of depression for low-income young African-American women has not been quantified. Baseline scores on a generic HRQL measure, the SF-36, from a randomized controlled trial of depression treatments were used as a basis for describing the HRQL of depressed (n = 124) and non-depressed (n = 44) low-income African-American young women. Results were compared to U.S. normative values for well adults and for depressed adults. Relationship between SF-36 scores and demographic variables were examined, and SF-36 scores were compared for those with depression only and those with comorbid anxiety. SF-36 scores were lower for all eight subscales relative to the U.S. norms (p < 0.05) and lower on two subscales relative to a general U.S. depressed sample (p < 0.05). Higher age and higher number of children was associated with poorer scores. Comorbid anxiety was present in 66% of the depressed sample; all SF-36 scores for the sample with comorbid anxiety were significantly lower than scores for subjects with depression alone. Results indicate the substantial HRQL impact of depression among low-income young African-American women with depression.

Cost-effectiveness of evidence-based pharmacotherapy or cognitive behavior therapy compared with community referral for major depression in predominantly low-income minority women.

BACKGROUND: Few clinical trials have evaluated interventions for major depressive disorder in samples of low-income minority women, and little is known about the cost-effectiveness of depression interventions for this population. OBJECTIVE: To evaluate the cost-effectiveness of pharmacotherapy or cognitive behavior therapy (CBT) compared with community referral for major depression in low-income minority women. DESIGN, SETTING, AND PARTICIPANTS: A randomized clinical trial was conducted in 267 women with current major depression. INTERVENTIONS: Participants were randomly assigned to pharmacotherapy (paroxetine hydrochloride or bupropion hydrochloride) (n = 88), CBT (n = 90), or community referral (n = 89). MAIN OUTCOME MEASURES: The main outcomes were intervention and health care costs, depression-free days, and quality-adjusted life years based on Hamilton Depression Rating Scale scores and Medical Outcomes Study 36-Item Short-Form Health Survey summary scores for 12 months. Cost-effectiveness ratios were estimated to compare incremental patient outcomes with incremental costs for pharmacotherapy relative to community referral and for CBT relative to community referral. RESULTS: Compared with the community referral group, the pharmacotherapy group had significantly lower adjusted mean Hamilton Depression Rating Scale scores from the 3rd month through the 10th month (P = .04 to P<.001) of the study, and the CBT group had significantly lower adjusted mean scores from the 5th month through the 10th month (P = .03 to P = .049). There were significantly more depression-free days in the pharmacotherapy group (mean, 39.7; 95% confidence interval, 12.9-66.5) and the CBT group (mean, 25.80; 95% confidence interval, 0.04-51.50) than in the community referral group. The cost per additional depression-free day was USD 24.65 for pharmacotherapy and USD 27.04 for CBT compared with community referral. CONCLUSIONS: Effective treatment for depression in low-income minority women reduces depressive symptoms but increases costs compared with community referral. The pharmacotherapy and CBT interventions were cost-effective relative to community referral for the health care system.

Psychometric performance of an assessment scale for strain in nursing care: the M-NCAS.

BACKGROUND: Multiple instruments exist to measure dementia behaviors, but the nursing staff perspective on those behaviors and their level of burden has not been well measured. The goal of this study was to examine the psychometric performance of the Modified Nursing Care Assessment Scale (M-NCAS), a 28-item nurse rating of burden associated with care for institutionalized individuals with dementia. Nurses rate items in terms of extent to which the behavior or characteristic is present ("attitude" domain), and extent to which it is a burden ("strain" domain). METHODS: Data from 282 patients enrolled in a 12-week, double-blind, randomized clinical trial comparing risperidone treatment to placebo was used to evaluate M-NCAS item performance, internal consistency reliability, and construct validity. Empirical subscales were identified via exploratory factor analysis (EFA). RESULTS: Four poorly-performing items were deleted from further analyses. EFA identified 3 "attitude" subscales and 5 "strain" subscales. Cronbach's alphas were 0.65 and above. Correlation with the Cohen-Mansfield Agitation Inventory and the BEHAVE-AD, clinical ratings of dementia behaviors, were low to moderate. CONCLUSION: The M-NCAS provides a valid and reliable means of obtaining care burden ratings from formal caregivers in long-term care, and provides a method for evaluating dementia interventions from the perspective of nursing staff.

The effect of risperidone on nursing burden associated with caring for patients with dementia.

OBJECTIVES: To compare risperidone treatment with placebo for the burden to nursing staff of older nursing-home residents with dementia. DESIGN: Randomized, double-blind, placebo-controlled trial. SETTING: Long-term care institutions in Australia and New Zealand. PARTICIPANTS: Two hundred seventy-nine residents had evaluable data for comparison of oral risperidone with placebo treatment at 4 weeks, 8 weeks, and endpoint across the 12-week trial. MEASUREMENTS: The Modified Nursing Care Assessment Scale (M-NCAS) is a 28-item instrument that measures behavior occurrence in patients (patient outcome) and difficulty of those behaviors for nursing staff as rated by nurse carers (nurse outcome). Treatment groups were compared via analysis of covariance (ANCOVA). To aid score interpretation, M-NCAS effect sizes were examined by response category, with response defined as subjects with mean Cohen-Mansfield Agitation Inventory total aggression subscale score change of 4 points or greater, a clinical outcome for patients. RESULTS: Between-group ANCOVAs showed statistically significant reductions in nurse-rated M-NCAS scores from baseline to endpoint for the patients randomized to risperidone relative to placebo (P<.05). Effects were evident at 4 weeks and maintained over time. Effect sizes were moderate to large for responders and near zero for nonresponders. CONCLUSION: Risperidone treatment was associated with an early and sustained reduction in nursing staff burden as measured using the M-NCAS compared with placebo treatment for older nursing home residents with dementia.

Time and economic cost of constipation care in nursing homes.

OBJECTIVE: Chronic constipation is a common disorder among residents in long-term care; yet the cost to the nursing home of constipation-related care is not known. The objective of this study was to quantify the nursing staff and supply-related cost of constipation care to nursing homes from the perspective of the nursing home. DESIGN: Prospective, observational time-and-motion design. SETTING: Two United States nursing homes. PARTICIPANTS: A total of 59 nursing home residents with chronic constipation and nursing staff providing constipation care to them. MEASUREMENTS: Actual time to complete constipation care-related tasks was measured via stopwatch by trained observers, and the number and professional level (eg, staff nurse, CNA) of staff performing each task was recorded. Frequency of constipation care task data was obtained through 60-day retrospective medical chart review for each subject. Nurse wage rate data was obtained from the Nursing Home Salary and Benefits Report, a US-based national source. Resident and nursing home descriptive information was also collected. RESULTS: The average cost per task occurrence ranged from 0.72 US dollars for enema administration to 1.74 US dollars for oral medication administration. Average nursing staff costs per subject per year were 1577 US dollars for oral medication administration, 215 US dollars for dietary supplement administration, 39 US dollars for constipation assessment, 17 US dollars for suppository administration, and 6 US dollars for enema administration. Based on estimates of frequency of occurrence, the total annual labor and supply cost per long-term care resident with constipation was 2253 US dollars. CONCLUSION: Nursing staff performance of constipation care-related tasks is time consuming and costly in the long-term care setting.