Patient-centered Comparative Effectiveness Research Articles

Enhancing data practices for Whole Health: Strategies for a transformative future.

We explored the challenges and solutions for managing data within the Whole Health System (WHS), which operates as a Learning Health System and a patient-centered healthcare approach that combines conventional and complementary approaches. Addressing these challenges is critical for enhancing patient care and improving outcomes within WHS. The proposed solutions include prioritizing interoperability for seamless data exchange, incorporating patient-centered comparative clinical effectiveness research and real-world data to personalize treatment plans and validate integrative approaches, and leveraging advanced data analytics tools to incorporate patient-reported outcomes, objective metrics, robust data platforms. Implementing these measures will enable WHS to fulfill its mission as a holistic and patient-centered healthcare model, promoting greater collaboration among providers, boosting the well-being of patients and providers, and improving patient outcomes.

ADRD: A REVIEW OF THE PATIENT-CENTERED OUTCOMES RESEARCH INSTITUTE’S (PCORI) RESEARCH PORTFOLIO AND PRIORITIES

Abstract Given the aging of the U.S. population, the number of individuals living with Alzheimer’s Disease and Related Dementias (ADRD) is projected to reach 13 million by 2050. There is an urgent need for research efforts to advance prevention, diagnosis, and treatment strategies for ADRD. Furthermore, caregivers of individuals with ADRD often face distinct challenges and experience significant caregiving burden that has an impact on their well-being. To address unmet needs of individuals with ADRD and their caregivers, PCORI has invested nearly $85 million in patient-centered comparative clinical effectiveness research (CER) focused on ADRD and has initiated several stakeholder convenings to identify future research priorities. PCORI’s ADRD portfolio of 15 CER studies includes interventions focused on caregiving, polypharmacy, telehealth, care models, and palliative care. Additionally, PCORI has funded engagement awards, dissemination & implementation initiatives, and research infrastructure projects to help facilitate the conduct and awareness of research on persons living with ADRD and their caregivers. To support continued research in this area, PCORI plans to re-issue a $50 million funding initiative on Healthy Aging with a special area of emphasis on interventions for caregivers of older adults living with ADRD. By systematically synthesizing PCORI’s portfolio and themes from stakeholder convenings in ADRD, we can identify the strengths of current investments and understand evidence gaps to guide PCORI’s future investments. This will enable us to holistically address future real-world, decisional dilemmas most important to patients, caregivers, and other stakeholders.

Engaging Patients and Caregivers to Develop a Patient-Centered Agenda for Comparative Effectiveness Research Focused on the Treatment of Complex Knee Problems.

Complex articular cartilage loss in the knee is being diagnosed more frequently and earlier in life, and patients are faced with major decisions regarding invasive surgical interventions at increasingly younger ages. There is a critical unmet need to provide patient-centered comparative effectiveness research for the hundreds of thousands of patients faced with these treatment decisions each year. Toward filling the need, we developed the Patient AdvisoR Team iN Orthopaedic ReSearch (PARTNORS) program. We recruited a diverse group of patients and caregivers with lived experiences in dealing with complex knee problems to define patient-centered research priorities for comparative biological and artificial knee surgery research for middle-aged adults. Adapting the Stakeholder Engagement in Question Development and Prioritization Method, PARTNORS defined a 20-question list of patient-centered research questions of factors influencing a patients' choice between biological and artificial knee surgeries. The highest prioritized research question related to functional level postsurgery as it relates to daily activities and recreational activities. The second highest prioritized research questions related to insurance coverage and financial costs. Other prioritized research areas included caregiving needs, implant longevity, recovery and rehabilitation time, patient satisfaction and success rates, individual characteristics, and risks. By engaging a group of patients and caregivers and including them as members of a multidisciplinary research team, comparative effectiveness research that includes patient-centered factors that go beyond typical clinical success indicators for knee surgery can be designed to allow physicians and patients to work together toward evidence-based shared decisions. This shared decision-making process helps to align patients' and health care team's goals and expectations to improve outcomes.

Promoting collaborative psychiatric care decision-making in community mental health centers: Insights from a patient-centered comparative effectiveness trial.

Mental health service-users face important medication decisions; yet not all are active participants in the decision-making process. Little is known about which technology-supported interventions might effectively promote collaborative decision-making in psychiatric care. We compared the effectiveness of two technology-supported collaborative care decision-making approaches. We used a cluster-randomized design with a mixed-methods approach. Participants were Medicaid-enrolled adults receiving psychiatric care in participating community mental health centers. Measurement-based care used computerized systematic symptom and medication screenings to inform provider decision-making. Person-centered care supported participants in completing computerized Health Reports and preparing to work with providers on collaborative decision-making about psychiatric care. Primary study outcomes included the patient experience of medication management and shared decision-making during psychiatric care. Analyses examined the impact of both approaches and explored moderating variables. We used qualitative methods to understand participation and implementation experiences. Across 14 sites 2,363 participants enrolled (1,162 in measurement-based care, 1,201 in person-centered care). We observed statistically significant improvements in patient experience of medication management scores for both study arms; however, the clinical significance of this change was minor. We found no significant changes for shared decision-making. Qualitative interviews revealed a range of factors associated usefulness of intervention assessment, provider-service-user communication, and site-level logistics. We observed modest positive findings related to our patient-centered outcomes. We identified important implementation facilitators and barriers that can inform the implementation of future comparative effectiveness patient-centered research. (PsycInfo Database Record (c) 2021 APA, all rights reserved).

Researchers, patients, and other stakeholders\u2019 perspectives on challenges to and strategies for engagement

BackgroundThere 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.MethodsDescriptive 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.ResultsInvestigators 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.ConclusionMeaningful 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.

0624 Voice of the Patient: A Patient-Centered Exploration of Factors Influencing Obstructive Sleep Apnea Care

Abstract Introduction Despite widely available efficacious treatments for obstructive sleep apnea (OSA), patients commonly report frustration in accessing and adhering to treatments. Sparse research has explored factors influencing OSA care from the patient perspective, which may limit provision of patient-centered care: care responsive to patient preferences, needs, and values. To this end, we conducted qualitative research to identify factors, voiced by patients, that influence OSA treatment initiation and adherence. Methods We performed semi-structured interviews with 15 patients previously diagnosed with OSA from Boston, MA and a national patient portal (MyApnea.Org). Patients were asked about barriers and facilitators to their diagnosis and treatment as well as about their preferences and values that informed their treatment decisions. Interviews were audio-recorded and transcribed. A qualitative content analysis was performed to identify themes. After developing a codebook, interviews were coded. Codes were then audited and finalized by study team consensus. Results Our sample was aged 25-74 years; 71% identified as female. Among participants, 57.1% identified as White, 14.3% Black, 14.3% Asian, and 14.3% Other. Major themes were broadly classified as (1) facilitators (provision of useful information on treatment options, participation in shared decision-making, continued clinician support); (2) barriers (inconvenience of treatment, difficulty of habit formation, treatment side effects, competing comorbid conditions); (3) motivators (value of improving chronic health, family support, positive treatment effects); (4) contextual factors (insufficient knowledge/awareness of OSA, navigating healthcare systems, access to informational resources). Awareness of OSA symptoms and treatments, and ongoing support were cited as the most common factors influencing the patient experience. Conclusion This formative research highlights that diverse factors impact the OSA evaluation and treatment patient experience. Further research should test interventions that promote effective patient-centered care for OSA, such as shared decision-making tools. Support Brigham and Women’s Hospital Research Institute Patient-Centered Comparative Effectiveness Research Center Grant

Engaging parent–child dyad and healthcare provider stakeholders in a patient-centered comparative effectiveness study

Aim: Describe engagement activities in a comparative effectiveness study evaluating two interventions for promoting psychosocial health among youth ages 10-17 who have recently experienced a nonintentional injury. Methods: Institutional, community and patient stakeholders from four children's hospitals were engaged through consultation meetings, individual interviews and a collaborative meeting. Results: 67 engagement activities were conducted across four hospitals. Feedback to improve recruitment, retention and continuous engagement in the study was obtained. Finally, disseminating study interventions to school and healthcare settings, and adding alternative delivery formats were identified as priority next steps. Conclusion: Results highlight diverse methods of engaging patient and professional stakeholders, critical recommendations for improving study engagement and retention, and future directions for this patient-engaged comparative effectiveness research.

Patient-centered recruitment and retention for a randomized controlled study

BackgroundRecruitment and retention strategies for patient-centered outcomes research are evolving and research on the subject is limited. In this work, we present a conceptual model of patient-centered recruitment and retention, and describe the recruitment and retention activities and related challenges in a patient-centered comparative effectiveness trial.MethodsThis is a multicenter, longitudinal randomized controlled trial in localized prostate cancer patients.ResultsWe recruited 743 participants from three sites over 15 months period (January 2014 to March 2015), and followed them for 24 months. At site 1, of the 773 eligible participants, 551 (72%) were enrolled. At site 2, 34 participants were eligible and 23 (68%) enrolled. Of the 434 eligible participants at site 3, 169 (39%) enrolled. We observed that strategies related to the concepts of trust (e.g., physician involvement, ensuring protection of information), communication (e.g., brochures and pamphlets in physicians’ offices, continued contact during regular clinic visits and calling/emailing assessment), attitude (e.g., emphasizing the altruistic value of research, positive attitude of providers and research staff), and expectations (e.g., full disclosure of study requirements and time commitment, update letters) facilitated successful patient recruitment and retention. A stakeholders’ advisory board provided important input for the recruitment and retention activities. Active engagement, reminders at the offices, and personalized update letters helped retention during follow-up. Usefulness of telephone recruitment was site specific and, at one site, the time requirement for telephone recruitment was a challenge.ConclusionsWe have presented multilevel strategies for successful recruitment and retention in a clinical trial using a patient-centered approach. Our strategies were flexible to accommodate site-level requirements. These strategies as well as the challenges can aid recruitment and retention efforts of future large-scale, patient-centered research studies.Trial registrationClinicaltrials.gov, ID: NCT02032550. Registered on 22 November 2013.

Methods and impact of engagement in research, from theory to practice and back again: early findings from the Patient-Centered Outcomes Research Institute

PurposeSince 2012, PCORI has been funding patient-centered comparative effectiveness research with a requirement for engaging patients and other stakeholders in the research, a requirement that is unique among the US funders of clinical research. This paper presents PCORI’s evaluation framework for assessing the short- and long-term impacts of engagement; describes engagement in PCORI projects (types of stakeholders engaged, when in the research process they are engaged and how they are engaged, contributions of their engagement); and identifies the effects of engagement on study design, processes, and outcomes selection, as reported by both PCORI-funded investigators and patient and other stakeholder research partners.MethodsDetailed quantitative and qualitative information collected annually from investigators and their partners was analyzed via descriptive statistics and cross-sectional qualitative content and thematic analysis, and compared against the outcomes expected from the evaluation framework and its underlying conceptual model.ResultsThe data support the role of engaged research partners in refinements to the research questions, selection of interventions to compare, choice of study outcomes and how they are measured, contributions to strategies for recruitment, and ensuring studies are patient-centered.ConclusionsThe evaluation framework and the underlying conceptual model are supported by results to date. PCORI will continue to assess the effects of engagement as the funded projects progress toward completion, dissemination, and uptake into clinical decision making.

Abstract P5-08-23: The share thoughts on breast cancer study: A Greater Plains collaborative cohort characterization project

Abstract Background. The Greater Plains Collaborative (GPC) is a new clinical data research network in PCORNet, the National Patient-Centered Clinical Research Network sponsored by the Patient-Centered Outcomes Research Institute to conduct patient-centered outcomes research and comparative effectiveness research. The Phase I GPC network brought together a diverse population of 10 million people across 1300 miles covering seven states with a combined area of 679 159 square miles. Using input from community members, breast cancer was selected as a focus for cohort building activities. Objective. To test the GPC's integrated data sources, technical components, and governance by demonstrating the ability to select, characterize, and achieve at least a 50% response rate for a cohort of breast cancer patients from across the GPC. Methods. Each participating medical center extracted, transformed and loaded North American Association of Central Cancer Registries (NAACCR)-formatted data from their institution's tumor registry into its Informatics for Integrating Biology and the Bedside (i2b2) research warehouse. The GPC i2b2 research warehouse is fully de-identified with re-identification possible when accompanied by an approved IRB protocol. From these data, each medical center selected a cohort of all patients age 18 or older diagnosed with breast cancer between January 2013 and May 2014. De-identified data files were submitted to the GPC Honest Broker who applied eligibility criteria and selected a random sample of 250 eligible patients from each center's file. Eligible patients were women with microscopically-confirmed ductal carcinoma in situ or invasive (but not metastatic) breast cancer diagnosed during the study period. Women who had previously been diagnosed with cancer per tumor registry records were excluded as were women known to be deceased at the time the sample was selected. The sample of patients plus a list of up to 10 replacement patients was provided to each center for re-identification and mailing. Patients participated in designing the study materials. Results. IRBs at 7 of 8 collaborating medical centers ceded IRB review to the University of Iowa IRB pursuant to the GPC reliance agreement. Upon patient advice to ensure a representative cohort, a mailed questionnaire with patient incentive was employed. All study materials were mailed in a single packet containing a cover letter from the participating medical center, a 21-page questionnaire, medical record consent and $10 incentive. Questionnaires were mailed over a six-week period beginning June 19, 2015 and one remailing to nonrespondents was conducted 4 weeks after the initial mailing. A total of 1,986 patients were invited and 1,235 (62.2%) responded. Signed consent to obtain information from medical records was obtained for 852 (69%). The survey dataset for consented subjects was linked to the research warehouse to obtain tumor and treatment data and diagnosis and procedure codes from billing sources. Conclusion. The GPC's integrated data sources, honest broker-mediated approach to extract data and apply standardized clinical criteria, IRB reliance model, and patient study design partners were implemented successfully. Citation Format: Chrischilles EA, Gryzlak B, McDowell BD, Connolly DW, Waitman LR. The share thoughts on breast cancer study: A Greater Plains collaborative cohort characterization project [abstract]. In: Proceedings of the 2016 San Antonio Breast Cancer Symposium; 2016 Dec 6-10; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2017;77(4 Suppl):Abstract nr P5-08-23.

Health researcher views on comparative effectiveness research and research engagement.

To understand researcher capability for and interest in patient-centered comparative effectiveness research (PC-CER), particularly related to engaging with patients/caregivers. Web-based survey of 508 health researchers recruited via professional health research organizations. 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. 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.

Challenges encountered in the conduct of Optimal Health: A patient-centered comparative effectiveness study of interventions for adults with serious mental illness.

The aim of patient-centered comparative effectiveness research is to conduct stakeholder-driven investigations that identify which interventions are most effective for which patients under specific circumstances. Conducting this research in real-world settings comes with unique experiences and challenges. We provide the study design, challenges confronted, and the solutions we devised for Optimal Health, a stakeholder-informed patient-centered comparative effectiveness study focused on the needs of seriously mentally ill individuals receiving case management services in community mental health centers across Pennsylvania. Optimal Health, supported by the Patient-Centered Outcomes Research Institute, is a cluster-randomized trial of two evidence-based interventions for improving health and wellness across 11 provider sites. Participants were followed for 18-24 months, with repeated measurements of self-reported health status and activation in care and administrative measurements of primary and specialty health service utilization. Health-related quality of life, engagement in care, and service utilization are to be compared via random effects mixed models. Stakeholders were, and continue to be, engaged via focus groups, interviews, and stakeholder advisory board meetings. A learning collaborative model was used to support shared learning and implementation fidelity across provider sites. From 1 November 2013 through 15 July 2014, we recruited 1229 adults with serious mental illness, representing 85.1% of those eligible for study participation. Of these, 713 are in the Provider-Supported arm of the study and 516 in Patient Self-Directed Care. Across five data collection time points, we retained 86% and 83% of the participants in the Provider-Supported and Self-Directed arms, respectively. Lessons learned relate to estimation of the size of our study population, the value of multiple data sources, and intervention training and implementation. The use of historical claims data can lead to an overestimation of eligible participants and, subsequently, a reduced study sample and an imbalance between intervention arms. Disruptions in continuity of care in real-world settings can pose challenges to on-site self-report data collection, although the inclusion of multiple data sources in study design can improve data completeness. Geographic dispersion of rural provider sites and staff turnover can lead to training and intervention fidelity challenges that can be overcome with the use of a "train-the-trainer" model, "wellness champions," and the use of a Learning Collaborative approach. Stakeholder engagement in mitigating these challenges proved to be critical to study progress. Conducting real-world patient-centered comparative effectiveness research in healthcare systems that care for seriously mentally ill persons is an important yet challenging undertaking, one which requires flexibility in identifying potential adaptations within all major study phases. Advice from a wide range of stakeholders is critical in development of successful strategies.

Stakeholder involvement in the design of a patient-centered comparative effectiveness trial of the “On the Move” group exercise program in community-dwelling older adults

BackgroundGroup exercise programs for older adults often exclude the timing and coordination of movement. Stakeholder involvement in the research process is strongly encouraged and improves the relevance and adoption of findings. We describe stakeholder involvement in the design of a clinical trial of a group-based exercise program that incorporates timing and coordination of movement into the exercises. MethodsThe study was a cluster randomized, single-blind intervention trial to compare the effects on function, disability and mobility of a standard group exercise program and the “On the Move” group exercise program in older adults residing in independent living facilities and senior apartment buildings, and attending community centers. Exercise classes were twice weekly for 12weeks delivered by study exercise leaders and facility activity staff personnel. OutcomesThe primary outcomes function, disability and mobility were assessed at baseline and post-intervention. Function and disability were assessed using the Late Life Function and Disability Instrument, and mobility using the Six-Minute Walk Test and gait speed. StakeholdersPatient and provider stakeholders had significant input into the study aims, design, sample, intervention, outcomes and operational considerations. SummaryA community-based exercise program to improve walking can be developed to address both investigator identified missing components in current exercise to improve walking and stakeholder defined needs and interest for the activity program. Involvement of stakeholders substantially improves the relevance of research questions, increases the transparency of research activities and may accelerate the adoption of research into practice.

Young adult and parent stakeholder perspectives on participation in patient-centered comparative effectiveness research.

Explore perspectives of adolescent and young adult (AYA) and parent stakeholders regarding their engagement in comparative effectiveness research (CER) evaluating cholesterol screening and treatment strategies for 17-21 year olds. All nine AYAs and parent stakeholders participating in a 20-member panel of AYAs, parents and professionals (i.e., clinicians, researchers, policy makers, payers), completed a quantitative survey and a semistructured interview at the completion of the core CER study. AYAs and parents stakeholders emphasized the role of power differentials regarding shared knowledge, relationships and trust, and logistics. To mitigate power differentials, stakeholders recommended more materials, clearer definition of roles and in-person meetings. Perceived positive outcomes included diversity of perspectives provided, better understanding their own health and decision-making and improving CER.

Patient Partnerships Transforming Sleep Medicine Research and Clinical Care: Perspectives from the Sleep Apnea Patient-Centered Outcomes Network.

Due to an ongoing recent evolution in practice, sleep medicine as a discipline has been compelled to respond to the converging pressures to reduce costs, improve outcomes, and demonstrate value. Patient "researchers" are uniquely placed to participate in initiatives that address the specific needs and priorities of patients and facilitate the identification of interventions with high likelihood of acceptance by the "customer." To date, however, the "patient voice" largely has been lacking in processes affecting relevant policies and practice guidelines. In this Special Report, patient and research leaders of the Sleep Apnea Patient-Centered Outcomes Network (SAPCON), a national collaborative group of patients, researchers and clinicians working together to promote patient-centered comparative effectiveness research, discuss these interrelated challenges in the context of sleep apnea, and the role patients and patient-centered networks may play in informing evidence-based research designed to meet patient's needs. We first briefly discuss the challenges facing sleep medicine associated with costs, outcomes, and value. We then discuss the key role patients and patient-centered networks can play in efforts to design research to guide better sleep health care, and national support for such initiatives. Finally, we summarize some of the challenges in moving to a new paradigm of patient-researcher-clinician partnerships. By forging strong partnerships among patients, clinicians and researchers, networks such as SAPCON can serve as a living demonstration of how to achieve value in health care.

Ethics and Regulatory Challenges and Opportunities in Patient-Centered Comparative Effectiveness Research.

The Affordable Care Act includes provisions for the conduct of large-scale, patient-centered comparative effectiveness research. Such efforts aim toward the laudable moral goal of having evidence to improve health care decision making. Nevertheless, these pragmatic clinical research efforts that typically pose minimal incremental risk and are enmeshed in routine care settings perhaps surprisingly encounter an array of ethics and regulatory challenges and opportunities for academic health centers. An emphasis on patient-centeredness forces an examination of the appropriateness of traditional methods used to protect the rights, interests, and welfare of participants. At the same time, meaningful collaboration with patients throughout the research process also necessitates ensuring that novel approaches to research (including recruitment and consent) entail necessary protections regarding such issues as privacy. As the scientific and logistical aspects of this research are being developed, substantial attention is being focused on the accompanying ethics and regulatory issues that have emerged, which should help to facilitate ethically appropriate research in a variety of contexts.

Opportunities for Patient-centered Outcomes Research in Radiology

Recently created in 2010, the Patient-Centered Outcomes Research Institute (PCORI) supports patient-centered comparative effectiveness research with a focus on prioritizing high-impact studies and improving trial design methodology. The Association of University Radiologists Radiology Research Alliance Task Force on patient-centered outcomes research in Radiology aims to review recently funded imaging-centric projects that adhere to the methodologies established by PCORI. We provide an overview of the successful application of PCORI standards to radiology topics, highlight how these methodologies differ from other forms of radiology research, and identify opportunities for new projects as well as potential barriers for involvement. Our hope is that review of specific case examples in radiology will clarify the use and value of PCORI methods mandated and supported nationally by the Affordable Care Act.

Anticipated Ethics and Regulatory Challenges in PCORnet: The National Patient-Centered Clinical Research Network

ABSTRACTPCORnet, the National Patient-Centered Clinical Research Network, seeks to establish a robust national health data network for patient-centered comparative effectiveness research. This article reports the results of a PCORnet survey designed to identify the ethics and regulatory challenges anticipated in network implementation. A 12-item online survey was developed by leadership of the PCORnet Ethics and Regulatory Task Force; responses were collected from the 29 PCORnet networks. The most pressing ethics issues identified related to informed consent, patient engagement, privacy and confidentiality, and data sharing. High priority regulatory issues included IRB coordination, privacy and confidentiality, informed consent, and data sharing. Over 150 IRBs and five different approaches to managing multisite IRB review were identified within PCORnet. Further empirical and scholarly work, as well as practical and policy guidance, is essential if important initiatives that rely on comparative effectiveness research are to move forward.

Patient-Centered Research into Outcomes Stroke Patients Prefer and Effectiveness Research: Implementing the patient-driven research paradigm to aid decision making in stroke care

Stroke is common and costly, annually depriving the lives and well-being of 800,000 Americans. Despite demonstrated efficacy in clinical trials, questions remain about the safety and clinical effectiveness of various treatment options given patient characteristics, conditions, preferences, and their desired outcomes. The Patient-Centered Research Into Outcomes Stroke Patients Prefer and Effectiveness Research (PROSPER) is a Patient-Centered Outcomes Research Institute-sponsored project designed to help patients, physicians, and other stakeholders make informed decisions regarding stroke care and improve outcomes through patient-centered comparative effectiveness research. The primary outcomes identified and prioritized by stroke patients are "home time" (time spent alive and outside a hospital) and major adverse cardiovascular events. With inputs from stroke patients themselves, a series of comparative safety and effectiveness analyses will be performed across 3 prioritized therapeutic areas identified as important by stroke survivors: oral anticoagulants, statin therapy, and antidepressants. We obtained data from Get With the Guidelines-Stroke linked with Medicare claims and follow-up telephone interviews. Our combined retrospective and prospective research strategy allows the evaluation of the safety and effectiveness of various treatment options and patient-centered longitudinal outcomes. To ensure the rapid translation of findings into clinical practice, results will be disseminated to stroke survivors, caregivers, and health care providers through traditional and social media, including an online decision aid tool. PROSPER is a patient-centered outcome research study guided by patients, caregivers, and the broader health care community. By addressing knowledge gaps in treatment uncertainties through comparative effectiveness research, PROSPER has the potential to improve decision making in stroke care and patient outcomes reflecting individual patient preferences, needs, and values.

Conceptual and practical foundations of patient engagement in research at the patient-centered outcomes research institute.

PurposeTo provide an overview of PCORI’s approach to engagement in research.MethodsThe Patient-Centered Outcomes Research Institute (PCORI) was established in 2010 to fund patient-centered comparative effectiveness research. Requirements for research funding from PCORI include meaningful engagement of patients and other stakeholders in the research. PCORI’s approach to engagement in research is guided by a conceptual model of patient-centered outcomes research (PCOR), that provides a structure for understanding engagement in research.ResultsTo understand and improve engagement in research PCORI is learning from awardees and other stakeholders. Those efforts are described along with PCORI’s capacity building and guidance to awardees via the Engagement Rubric. PCORI’s unique model of engaging patients and other stakeholders in merit review of funding applications is also described. Additional support for learning about engagement in research is provided through specific research funding and through PCORI’s major infrastructure initiative, PCORnet.ConclusionPCORI requires engagement of stakeholders in the research it funds. In addition PCORI engages stakeholders in activities including review of funding applications and establishment of CER research infrastructure through PCORnet. The comprehensive approach to engagement is being evaluated to help guide the field toward promising practices in research engagement.