What motivates patients and caregivers to engage in health research and how engagement affects their lives: Qualitative survey findings.

BACKGROUND: US research organizations increasingly are supporting patient and stakeholder engagement in health research with a goal of producing more useful, relevant and patient-centered evidence better aligned with real-world clinical needs. The Patient-Centered Outcomes Research Institute (PCORI) engages patients, family caregivers and other health-care stakeholders, including clinicians, payers and policymakers, as active partners in prioritizing, designing, conducting and disseminating research as a key strategy to produce useful evidence for health-care decision making. OBJECTIVE: To inform effective engagement practices and policies, we sought to understand what motivates patients and caregivers to engage as partners on PCORI-funded research projects and how such engagement changed their lives. METHODS: We conducted thematic analysis of open-ended survey responses from 255 patients, family caregivers and individuals from advocacy and community-based organizations who engaged as partners on 139 PCORI-funded research projects focusing on a range of health conditions. RESULTS: Partners' motivations for engaging in research were oriented primarily towards benefiting others, including a desire to improve patients' lives and to support effective health-care interventions. In addition to feeling they made a positive difference, many partners reported direct benefits from engagement, such as new relationships and improved health habits. DISCUSSION AND CONCLUSIONS: By identifying patient and caregiver motivations for engaging in research partnerships and what they get out of the experience, our study may help research teams and organizations attract partners and foster more satisfying and sustainable partnerships. Our findings also add to evidence that engagement benefits the people involved as partners, strengthening the case for more widespread engagement.

Unique Review Criteria and Patient and Stakeholder Reviewers: Analysis of PCORI's Approach to Research Funding.

OBJECTIVE: The Patient-Centered Outcomes Research Institute (PCORI) uses a unique approach to Merit Review that includes patients and stakeholders as reviewers with scientists, and includes unique review criteria (patient-centeredness and active engagement of end users in the research). This study assessed the extent to which different reviewer types influence review scores and funding outcomes, the emphasis placed on technical merit compared to other criteria by a multistakeholder panel, and the impact of the in-person discussion on agreement among different reviewer types. METHODS: Cross-sectional analysis of administrative data from PCORI online and in-person Merit Review (N = 1312 applications from the five funding cycles from November 2013 to August 2015). Linear and logistic regression models were used to analyze the data. RESULTS: For all reviewer types, final review scores were associated with at least one review criterion score from each of the three reviewer types. The strongest predictor of final overall scores for all reviewer types was scientists' prediscussion ratings of technical merit. All reviewers' prediscussion ratings of the potential to improve health care and outcomes, and scientists' ratings of technical merit and patient-centeredness, were associated with funding success. For each reviewer type, overall impact scores from the online scoring were changed on at least half of the applications at the in-person panel discussion. Score agreement across reviewer types was greater after panel discussion. CONCLUSIONS: Scientist, patient, and stakeholder views all contribute to PCORI Merit Review of applications for research funding. Technical merit is critical to funding success but patient and stakeholder ratings of other criteria also influence application disposition.

Researchers, Patients, and Stakeholders Evaluating Comparative-Effectiveness Research: A Mixed-Methods Study of the PCORI Reviewer Experience.

OBJECTIVES: The Patient-Centered Outcomes Research Institute (PCORI) includes patients and stakeholders alongside scientists in reviewing research applications using unique review criteria including patient-centeredness and patient and/or stakeholder engagement. To support extension of this unique collaborative model to other funders, information from the reviewers on the review process is needed to understand how scientists and nonscientists evaluate research proposals together. Thus, this study aimed to describe reviewers' perspectives of the interactions during the in-person review panel; to examine the value and challenges of including scientists, patients, and stakeholders together; and to understand the perceived importance of PCORI's review criteria. METHODS: This study utilized anonymous, cross-sectional surveys (N = 925 respondents from 5 funding cycles: 470 scientists, 217 patients, 238 stakeholders; survey completion rates by cycle: 70-89%) and group interviews (N = 18). RESULTS: Reviewers of all types describe PCORI Merit Review as respectful, balanced, and one of reciprocal influence among different reviewer types. Reviewers indicate strong support and value of input from all reviewer types, receptivity to input from others, and the panel chair's incorporation of all views. Patients and stakeholders provide real-world perspectives on importance to patients, research partnership plans, and study feasibility. Challenges included concerns about a lack of technical expertise of patient/stakeholder reviewers and about scientists dominating conversations. The most important criterion for assigning final review scores was technical merit-either alone or in conjunction with patient-centeredness or patient/ stakeholder engagement. CONCLUSIONS: PCORI Merit Reviewers' self-reports indicate that the perspectives of different reviewer types are influential in panel discussions and Merit Review outcomes.

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.

Association between serious psychological distress and health care use and expenditures by cancer history.

BACKGROUND: Serious psychological distress (SPD) is associated with adverse health outcomes such as poor quality of life and shorter survival in cancer survivors, but to the authors' knowledge, the relationship between SPD and health care use and medical expenditures is not clear. METHODS: A total of 4326 cancer survivors and 57,109 noncancer participants were identified from the 2008 through 2010 Medical Expenditure Panel Survey, a nationwide population-based survey, and their psychological distress was assessed with the 6-item Kessler Psychological Distress Scale (SPD defined by a score ≥13). The association between SPD and use and medical expenditures of various types of health care (office-based, outpatient, hospital inpatient, emergency department, dental, and prescriptions) was examined using a 2-part modeling approach that adjusted for demographic, personal, and comorbidity factors. The marginal effects of SPD on health care use and expenditures were calculated for cancer survivors and were compared with those of noncancer participants. RESULTS: The weighted prevalence of SPD in cancer survivors was 8.2% compared with 4.8% in the noncancer participants. SPD was significantly associated with higher use of all care types except dental care in cancer survivors. Cancer survivors with SPD spent $4431 (95% confidence interval, $3419-$5443) more than survivors without SPD on medical services each year, whereas this extra expenditure associated with SPD for participants without cancer was $2685 (95% confidence interval, $2099-$3271). CONCLUSIONS: In a national representative sample of cancer survivors, SPD was found to be associated with higher health care use and medical expenditures. Distress screening and psychosocial care in cancer survivors may help reduce the economic burden of cancer in the United States.

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.

A systematic review of approaches for engaging patients for research on rare diseases.

BACKGROUND: Patients with rare diseases have limited access to useful information to guide treatment decisions. Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes. Rare disease organizations may provide an effective means to facilitate patient engagement in research. However, the effectiveness of patient-engagement approaches, particularly for the study of rare diseases, has not been well studied. OBJECTIVES: To synthesize evidence about engagement of patients and other stakeholders in research on rare diseases, including the role of rare disease organizations in facilitating patient-centered research. METHODS/RESEARCH DESIGN: A systematic review and gray literature search were guided by a technical expert panel composed of patient representatives, clinicians, and researchers. English-language studies that engaged patients or other stakeholders in research on rare diseases or evaluated engagement were included. Studies were assessed on how well key research questions were answered, based on the level of detail describing engagement activities and whether outcomes from engagement were assessed. RESULTS: Thirty-five studies were included, although many reported minimal information on engagement. Patients and other stakeholders were most commonly engaged to identify patient-centered research agendas, to select which study outcomes were important to patients, to provide input on study design, and to identify strategies for increasing enrollment in trials. Rare disease organizations mainly helped provide access to patients and communicated research opportunities and findings. They also helped promote collaborative networks and provided financial support for research infrastructures. Although authors reported benefits of engagement and identified changes to their research processes, no empirical assessments of engagement practices and their effectiveness were found. CONCLUSIONS: Researchers studying rare diseases can obtain patient input regarding which research questions and health outcomes to study; however, the most effective approaches to engagement have not been well defined.

Social support, self-efficacy for decision-making, and follow-up care use in long-term cancer survivors.

OBJECTIVE: Cancer survivors play an important role in coordinating their follow-up care and making treatment-related decisions. Little is known about how modifiable factors such as social support are associated with active participation in follow-up care. This study tests associations between social support, cancer-related follow-up care use, and self-efficacy for participation in decision-making related to follow-up care (SEDM). We also identified sociodemographic and clinical factors associated with social support among long-term survivors. METHODS: The FOllow-up Care Use among Survivors study is a cross-sectional, population-based survey of breast, prostate, colon, and gynecologic cancer survivors (n=1522) 4-14 years post-diagnosis. Multivariable regression models were used to test associations between perceived social support (tangible and emotional/informational support modeled separately), follow-up care use (past 2 years), and SEDM, as well as to identify factors associated with perceived support. RESULTS: Neither support type was associated with follow-up care use (all p>0.05), although marital status was uniquely, positively associated with follow-up care use (p<0.05). Both tangible support (B for a standard deviation increase (SE)=9.75(3.15), p<0.05) and emotional/informational support (B(SE)=12.61(3.05), p<0.001) were modestly associated with SEDM. Being married, having adequate financial resources, history of recurrence, and better perceived health status were associated with higher perceived tangible and emotional support (all p<0.05). CONCLUSIONS: While perceived social support may facilitate survivor efficacy for participation in decision-making during cancer follow-up care, other factors, including marital satisfaction, appear to influence follow-up care use. Marital status and social support may be important factors to consider in survivorship care planning.

Role of oncologists and primary care physicians in providing follow-up care to non-Hodgkin lymphoma survivors within 5 years of diagnosis: a population-based study.

PURPOSE: The growing population of non-Hodgkin lymphoma (NHL) survivors living longer with high physical and psychological treatment burden, in combination with the projected shortage of medical professionals, necessitates redesigning cancer follow-up care. This study examined follow-up care patterns, factors associated with follow-up care, and attitudes towards follow-up care among NHL survivors. METHODS: We surveyed survivors of aggressive NHL 2 to 5 years post-diagnosis (N = 363) using a population-based sample from the Los Angeles County Surveillance Epidemiology and End Results registry. RESULTS: Most survivors (82 %) received cancer-related follow-up care in the past year from an oncologist. History of recurrence, more comorbidities, more symptoms, and a shorter survivor-oncologist relationship were associated with high-frequency care with the oncologist [(≥5 visits in the past year), p < 0.05]. Many survivors followed up by oncologists (71 %) also saw a primary care provider (PCP) and 47 % also saw both a PCP and other specialists. Factors associated with seeing a PCP in addition to an oncologist included more symptoms, more health information needs, no history of recurrence, perceived excellent quality of cancer follow-up care, and fewer visits with the oncologist (p < 0.05). Survivors generally reported high reassurance from, and low negative anticipation towards, follow-up care. CONCLUSIONS: The high proportion of NHL survivors receiving care from multiple physicians, and the sizable proportion (∼30 %) who have not recently seen a PCP, suggests that coordinating care across specialties is critical to ensure comprehensive, non-duplicative care. Understanding factors associated with cancer-related follow-up is a first step towards more effective, efficient, patient-centered care.

Patient engagement in designing, conducting, and disseminating clinical pain research: IMMPACT recommended considerations.

ABSTRACT: In the traditional clinical research model, patients are typically involved only as participants. However, there has been a shift in recent years highlighting the value and contributions that patients bring as members of the research team, across the clinical research lifecycle. It is becoming increasingly evident that to develop research that is both meaningful to people who have the targeted condition and is feasible, there are important benefits of involving patients in the planning, conduct, and dissemination of research from its earliest stages. In fact, research funders and regulatory agencies are now explicitly encouraging, and sometimes requiring, that patients are engaged as partners in research. Although this approach has become commonplace in some fields of clinical research, it remains the exception in clinical pain research. As such, the Initiative on Methods, Measurement, and Pain Assessment in Clinical Trials convened a meeting with patient partners and international representatives from academia, patient advocacy groups, government regulatory agencies, research funding organizations, academic journals, and the biopharmaceutical industry to develop consensus recommendations for advancing patient engagement in all stages of clinical pain research in an effective and purposeful manner. This article summarizes the results of this meeting and offers considerations for meaningful and authentic engagement of patient partners in clinical pain research, including recommendations for representation, timing, continuous engagement, measurement, reporting, and research dissemination.

Cancer survivorship research in Europe and the United States: where have we been, where are we going, and what can we learn from each other?

The growing number of cancer survivors worldwide has led to of the emergence of diverse survivorship movements in the United States and Europe. Understanding the evolution of cancer survivorship within the context of different political and health care systems is important for identifying the future steps that need to be taken and collaborations needed to promote research among and enhance the care of those living after cancer. The authors first review the history of survivorship internationally and important related events in both the United States and Europe. Lessons learned from survivorship research are then broadly discussed, followed by examination of the infrastructure needed to sustain and advance this work, including platforms for research, assessment tools, and vehicles for the dissemination of findings. Future perspectives concern the identification of collaborative opportunities for investigators in Europe and the United States to accelerate the pace of survivorship science going forward.

Lost productivity and burden of illness in cancer survivors with and without other chronic conditions.

BACKGROUND: Cancer survivors may experience long-term and late effects from treatment that adversely affect health and limit functioning. Few studies examine lost productivity and disease burden in cancer survivors compared with individuals who have other chronic conditions or by cancer type. METHODS: We identified 4960 cancer survivors and 64,431 other individuals from the 2008-2010 Medical Expenditure Panel Survey and compared multiple measures of disease burden, including health status and lost productivity, between conditions and by cancer site for cancer survivors. All analyses controlled for the effects of age, sex, race/ethnicity, and number of comorbid conditions. RESULTS: Overall, in adjusted analyses in multiple models, cancer survivors with another chronic disease (heart disease or diabetes) experienced higher levels of burden compared with individuals with a history of cancer only, chronic disease only, and neither cancer, heart disease, nor diabetes across multiple measures (P < .05). Among cancer survivors, individuals with short survival cancers and multiple cancers consistently had the highest levels of burden across multiple measures (P < .0001). CONCLUSIONS: Cancer survivors who have another chronic disease experience more limitations and higher levels of burden across multiple measures. Limitations are particularly severe in cancer survivors with short survival cancer and multiple cancers.

Are survivors who report cancer-related financial problems more likely to forgo or delay medical care?

BACKGROUND: Financial problems caused by cancer and its treatment can substantially affect survivors and their families and create barriers to seeking health care. METHODS: The authors identified cancer survivors diagnosed as adults (n=1556) from the nationally representative 2010 National Health Interview Survey. Using multivariable logistic regression analyses, the authors report sociodemographic, clinical, and treatment-related factors associated with perceived cancer-related financial problems and the association between financial problems and forgoing or delaying health care because of cost. Adjusted percentages using the predictive marginals method are presented. RESULTS: Cancer-related financial problems were reported by 31.8% (95% confidence interval, 29.3%-34.5%) of survivors. Factors found to be significantly associated with cancer-related financial problems in survivors included younger age at diagnosis, minority race/ethnicity, history of chemotherapy or radiation treatment, recurrence or multiple cancers, and shorter time from diagnosis. After adjustment for covariates, respondents who reported financial problems were more likely to report delaying (18.3% vs 7.4%) or forgoing overall medical care (13.8% vs 5.0%), prescription medications (14.2% vs 7.6%), dental care (19.8% vs 8.3%), eyeglasses (13.9% vs 5.8%), and mental health care (3.9% vs 1.6%) than their counterparts without financial problems (all P<.05). CONCLUSIONS: Cancer-related financial problems are not only disproportionately represented in survivors who are younger, members of a minority group, and have a higher treatment burden, but may also contribute to survivors forgoing or delaying medical care after cancer.

Pain in long-term breast cancer survivors: the role of body mass index, physical activity, and sedentary behavior.

Although pain is common among post-treatment breast cancer survivors, studies that are longitudinal, identify a case definition of clinically meaningful pain, or examine factors contributing to pain in survivors are limited. This study describes longitudinal patterns of pain in long-term breast cancer survivors, evaluating associations of body mass index (BMI), physical activity, sedentary behavior with mean pain severity and above-average pain. Women newly diagnosed with stages 0-IIIA breast cancer (N = 1183) were assessed, on average, 6 months (demographic/clinical characteristics), 30 months (demographics), 40 months (demographics, pain), 5 years (BMI, physical activity, and sedentary behavior), and 10 years (demographics, pain, BMI, physical activity, and sedentary behavior) post-diagnosis. This analysis includes survivors who completed pain assessments 40 months post-diagnosis (N = 801), 10 years post-diagnosis (N = 563), or both (N = 522). Above-average pain was defined by SF-36 bodily pain scores ≥1/2 standard deviation worse than age-specific population norms. We used multiple regression models to test unique associations of BMI, physical activity, and sedentary behavior with pain adjusting for demographic and clinical factors. The proportion of survivors reporting above-average pain was higher at 10 years than at 40 months (32.3 vs. 27.8 %, p < 0.05). Approximately one-quarter of survivors reported improved pain, while 9.0 % maintained above-average pain and 33.1 % reported worsened pain. Cross-sectionally at 10 years, overweight and obese survivors reported higher pain than normal-weight survivors and women meeting physical activity guidelines were less likely to report above-average pain than survivors not meeting these guidelines (p < 0.05). Longitudinally, weight gain (>5 %) was positively associated, while meeting physical activity guidelines was inversely associated, with above-average pain (OR, 95 % CI = 1.76, 1.03-3.01 and 0.40, 0.20-0.84, respectively) (p < 0.05). Weight gain and lack of physical activity place breast cancer survivors at risk for pain long after treatment ends. Weight control and exercise interventions should be tested for effects on long-term pain in these women.

The cancer psychosocial care matrix: a community-derived evaluative tool for designing quality psychosocial cancer care delivery.

OBJECTIVE: Although the Institute of Medicine provided a vision for effective psychosocial care for cancer survivors, limited guidance exists regarding the essential components of comprehensive care or progressive steps for implementing each component. This paper describes the development of a unique tool for assessing capacity to provide quality psychosocial care to cancer survivors and the results of the first implementation of this tool in community settings. METHODS: The psychosocial working group of the National Cancer Institute Community Cancer Centers Program (NCCCP) developed the Cancer Psychosocial Care Matrix assessment tool. All NCCCP sites (n=30, enrolled in 2007 nd 2010) completed the matrix indicating their capacity for providing psychosocial care at entry into NCCCP ('baseline') after 2 years of NCCCP participation (2007 sites only) and within the coming year ('future aspirations'). RESULTS: At baseline, matrix responses reflected few or no systematic processes in place for most components of comprehensive psychosocial care. However, reported capacity to deliver specific components improved at 2 years post-NCCCP entry for the 2007 sites and in all NCCCP sites' future aspirations. CONCLUSIONS: With growing demand on cancer centers to meet new metrics of quality care, the psychosocial matrix can help centers systematically identify and develop steps to address gap areas in their capacity to meet these new standards. The Cancer Psychosocial Care Matrix appears to enable evaluation of psychosocial programs, may promote intentions to improve psychosocial services, and can facilitate communication of 'best practices' among cancer centers.

Daytime sleepiness and sleep duration in long-term cancer survivors and non-cancer controls: results from a registry-based survey study.

PURPOSE: Sleep-related complaints are common in cancer survivors. Although daytime sleepiness and sleep duration are associated with poor functional status, quality of life, and mortality in the general population, little is known about these issues in long-term cancer survivors. This study examined differences in daytime sleepiness and sleep duration between long-term cancer survivors and non-cancer controls. METHODS: Survey data were analyzed from individuals diagnosed with cancer ≥2 years in the past (n = 1,171, mean age = 64.30, 80.8% white, 22.8% male) and spouse/friends controls (n = 250, mean age = 60.78, 88.0% white, 64.8% male). Daytime sleepiness was assessed using the Epworth Sleepiness Scale. Associations between sleep-related variables and history of cancer were estimated with multivariable logistic regression, adjusting for potential confounders. Stratified analyses were conducted to identify subgroups of survivors most at risk for sleep problems. RESULTS: Cancer survivors were more likely than controls to report excessive daytime sleepiness (OR = 1.64, 95% CI: 1.07, 2.50). A cancer diagnosis was associated with longer sleep duration among males (OR = 1.25, 95% CI: 1.02, 1.53), but not in females (OR = 0.87, 95% CI: 0.37, 1.05). All other associations were similar regardless of cancer site, histology, time since diagnosis, treatment history, and history of multiple cancers. CONCLUSIONS: Disturbances in daytime sleepiness and sleep duration persist among long-term cancer survivors and should be monitored in routine survivorship care. More research is needed to identify cancer survivors who are at increased risk for daytime sleepiness and disturbed sleep duration, as well as to identify causal mechanisms for, and interventions to mitigate, persistent differences.

Researchers, patients, and other stakeholders' perspectives on challenges to and strategies for engagement.

BACKGROUND: There is growing interest in patient and stakeholder engagement in research, yet limited evidence about effective methods. Since 2012, the Patient-Centered Outcomes Research Institute (PCORI) has funded patient-centered comparative effectiveness research with a requirement for engaging patients and other stakeholders as research partners in study planning, conduct, and dissemination. This requirement, unique among large healthcare research funders in the US, provides an opportunity to learn about challenges encountered and specific strategies used by PCORI-funded study teams. The primary objective of this study is to describe -- from the perspective of PCORI investigators and research partners-the most common engagement challenges encountered in the first two years of the projects and promising strategies to prevent and overcome these challenges. METHODS: Descriptive information about investigators, partners, and their engagement was collected from investigators via annual (N = 235) and mid-year (N = 40) project progress reporting to PCORI, and from their partners (N = 260) via voluntary survey. Qualitative data were analyzed using content and thematic analyses. RESULTS: Investigators and partners most often described engagement challenges in three domains: (1) infrastructure to support engagement, (2) building relationships, and (3) maintaining relationships. Infrastructure challenges related to financial and human resources, including funding support and dedicated staff, identifying diverse groups of partners, and partners' logistical needs. Challenges for both building and maintaining relationships encompass a variety of aspects of authentic, positive interactions that facilitate mutual understanding, full participation, and genuine influence on the projects. Strategies to prevent or mitigate engagement challenges also corresponded overall to the same three domains. Both groups typically described strategies more generally, with applicability to a range of challenges rather than specific actions to address only particular challenges. CONCLUSION: Meaningful engagement of patients and other stakeholders comes with challenges, as does any innovation in the research process. The challenges and promising practices identified by these investigators and partners, related to engagement infrastructure and the building and maintenance of relationships, reveal actionable areas to improve engagement, including organizational policies and resources, training, new engagement models, and supporting engagement by viewing it as an investment in research uptake and impact.

Examining the role of funders in ensuring value and reducing waste in research: An organizational case-study of the Patient-Centered Outcomes Research Institute.

International experts have recommended actions that funders can take to improve the value of research investments. They state that self-assessment and public sharing are the basis for accountability and improvement. We examined our policies and practice to determine the extent to which the Patient-Centered Outcomes Research Institute's (PCORI) policies and practices as a research funder align with international best practice recommendations. A self-audit of current policies and practice against 17 recommendations and 35 sub-recommendations representing five major stages of research production, based on adapted methods used for self-assessment by another funder, was performed.  Fit of existing PCORI policies and practices with 35 sub-recommendations, qualitative assessment of adequacy (area of strength; area of partial strength; area of growth; not applicable) for 17 recommendations for five stages of research production was assessed. Of the 17 recommendations, 15 were applicable to PCORI's research mission and focus.  PCORI has policies and practices in place for all elements of six recommendations ("area of strength") and policies that address each element but with some still in active development for three ("area of partial strength"). PCORI is partially addressing six of the 15 relevant recommendations ("area of growth"). Areas for growth include making study protocols publicly available, improving policies on data sharing, and enhancing collaboration with other funders to reduce redundant funding. A voluntary consortium of international funders is underway to encourage further progress, including additional self-assessment and public sharing for accountability. These findings indicate PCORI has undertaken efforts to align its funding practices with international recommendations to ensure the value of public dollars invested in research.  Further efforts will likely require additional coordination and collaboration between funders and stakeholders.

Patient Engagement In Research: Early Findings From The Patient-Centered Outcomes Research Institute.

Charged with ensuring that research produces useful evidence to inform health decisions, the Patient-Centered Outcomes Research Institute (PCORI) requires investigators to engage patients and other health care stakeholders, such as clinicians and payers, in the research process. Many PCORI studies result in articles published in peer-reviewed journals that detail research findings and engagement's role in research. To inform practices for engaging patients and others as research partners, we analyzed 126 articles that described engagement approaches and contributions to research. PCORI projects engaged patients and others as consultants and collaborators in determining the study design, selecting study outcomes, tailoring interventions to meet patients' needs and preferences, and enrolling participants. Many articles reported that engagement provided valuable contributions to research feasibility, acceptability, rigor, and relevance, while a few noted trade-offs of engagement. The findings suggest that engagement can support more relevant research through better alignment with patients' and clinicians' real-world needs and concerns.

Health researcher views on comparative effectiveness research and research engagement.

AIM: To understand researcher capability for and interest in patient-centered comparative effectiveness research (PC-CER), particularly related to engaging with patients/caregivers. MATERIALS & METHODS: Web-based survey of 508 health researchers recruited via professional health research organizations. RESULTS: Most respondents (94%) were familiar with CER and many (69%) reported having previously conducting some form of CER. Most respondents were familiar with (81%) and interested in (87%) partnering with patients and/or caregivers in research. Resources to assist in training, coordination of partners, guidance in apply for funding and improved infrastructure were commonly cited factors that would help researchers conduct PC-CER. CONCLUSION: There is a significant opportunity for researchers to engage patients and caregivers as partners in CER. Researchers recognize the need for additional training and expertise to leverage those opportunities.