Comparatively Ineffective? PCORI and the Uphill Battle to Make Evidence Count in US Medicine.

The Patient-Centered Outcomes Research Institute (PCORI) was established as part of the Affordable Care Act to promote research on the comparative effectiveness of treatment options. Advocates hoped this information would help reduce wasteful spending by identifying low-value treatments, but many conservatives and industry groups feared PCORI would ration care and threaten physicians' autonomy. PCORI faced three challenges during its first decade of operation: overcoming the controversy of its birth and escaping early termination, shaping medical practice, and building a public reputation for relevance. While PCORI has won reauthorization, it has not yet had a major impact on the decisions of clinicians or payers. PCORI's modest footprint reflects not only the challenges of getting a new organization off the ground but also the larger political, financial, and cultural barriers to the uptake of medical evidence in the US health care system. The growing attention among policymakers and researchers to provider prices (rather than utilization) as the driver of health care spending could be helpful to the political prospects of the evidence-based medicine project by making it appear to be less as rationing driven by costs and more as an effort to improve quality and uphold medical professionalism.

An evidence-based framework for identifying technologies of no or low-added value (NLVT).

OBJECTIVE: To synthetize the state of the art of methods for identifying candidate technologies for disinvestment and propose an evidence-based framework for executing this task. METHODS: An interpretative review was conducted. A systematic literature search was performed to identify secondary or tertiary research related to disinvestment initiatives and/or any type of research that specifically described one or more methods for identifying potential candidates technologies, services, or practices for disinvestment. An iterative and critical analysis of the methods described alongside the disinvestment initiatives was performed. RESULTS: Seventeen systematic reviews on disinvestment or related terms (health technology reassessment or medical reversal) were retrieved and methods of 45 disinvestment initiatives were compared. On the basis of this evidence, we proposed a new framework for identifying these technologies based on the wide definition of evidence provided by Lomas et al. The framework comprises seven basic approaches, eleven triggers and thirteen methods for applying these triggers, which were grouped in embedded and ad hoc methods. CONCLUSIONS: Although identification methods have been described in the literature and tested in different contexts, the proliferation of terms and concepts used to describe this process creates considerable confusion. The proposed framework is a rigorous and flexible tool that could guide the implementation of strategies for identifying potential candidates for disinvestment.

The Bureau of Sages: Incorporating Older Adult Voice into Meaningful Research.

BACKGROUND: Older adults receiving long-term care are often excluded from the design, development, and implementation of health-related research. The project aimed to create, implement and evaluate a sustainable research advisory board consisting of researchers, clinicians, and older adults living at home or in a skilled nursing facility (SNF). METHODS: To initiate Bureau development, 15 older adults, researchers, and clinicians came together to engage in research. In meetings and retreats, stakeholders mutually developed group procedures, roles, and values. Process evaluation focused on stakeholder experiences, development of the Bureau, and its impact. RESULTS: Trained Sages were able to offer meaningful input to researchers and present their own proposal at a conference. Lessons learned centered around continuously seeking feedback from participants, emphasizing co-learning, adapting to virtual engagement, and remaining flexible in structure and content of meetings. CONCLUSIONS: Results can inform future development of research advisory boards of older adult populations.

Factors associated with evidence-based decision-making among patients and providers.

Aim: For comparative effectiveness research to achieve its purpose, providers and patients must use research evidence to make medical decisions. Therefore, this study examined factors associated with evidence-based decision-making by patients and providers. Methods: Data were collected via cross-sectional online surveys of patients (n = 603) and providers (n = 628) between November 2011 and January 2012. Results: For both patients and providers, evidence-based medical decision-making is associated with perceptions, that is, some combination of self efficacy, attitudes and opinions. However, whereas knowledge is the most consistent factor associated with decision-making for providers, it is not associated at all for patients. Conclusion: Efforts to promote evidence-based medical decision-making among patients and providers should focus on skills training to improve self efficacy, and messages that highlight the benefits of patient engagement in medical decisions.

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.

The "pragmatic trial": An essentially contested concept?

For over 50 years, clinical research methodology has wrestled with the problem of the lack of correspondence between tests of treatments and applications of treatments. The former comprise of trials featuring scrupulous control of patient eligibility, treatment compliance, clinician expertise, follow-up intensity, and so on. In applying a validated treatment, the practitioner has to confront considerable real-world variation in potential patients and in implementation regimes. The remedy, going by the name of "pragmatic trials," is to conduct clinical trials in conditions corresponding more closely to everyday practice. This solution has proved easier to utter than to execute, and the paper reviews the extensive literature on pragmatic trials, seeking to assess whether it has terminated in clarity or contestation.

Model-Based Meta-Analysis: Optimizing Research, Development, and Utilization of Therapeutics Using the Totality of Evidence.

Model-based meta-analysis (MBMA) is a valuable component of the quantitative pharmacology toolkit for model-informed drug discovery and development. It enables principled decision making with a totality of evidence mindset through integration of internal and external data across multiple dimensions (e.g., targets/mechanisms, molecules/drugs, doses/regimens, diseases/indications, populations, endpoints, and clinical trial designs). MBMA distinguishes itself from traditional meta-analysis by infusing pharmacologic plausibility into the statistical rigor that typifies meta-analytic data integration. This is possible through mechanism-informed formulation of pharmacologically inspired cause-effect and dose-response relationships, time course of treatment effects, and interrelationships between proximal and distal outcomes of modulation of disease biology and pathophysiology. In this review, we offer a question-based approach to enhance appreciation of the value of MBMA across the continuum from drug discovery and translational research through clinical development, comparative effectiveness research, and postapproval optimization of therapeutics using illustrative examples across therapeutic areas.

A Culture-Centered Community-Grounded Approach to Disseminating Health Information among African Americans.

This study highlights the role of local communities in creating culturally rooted health information resources based on comparative effectiveness research (CER), depicting the role of culture in creating entry points for building community-grounded communication structures for evidence-based health knowledge. We report the results from running a year-long culture-centered campaign that was carried out among African American communities in two counties, Lake and Marion County, in Indiana addressing basic evidence-based knowledge on four areas of cardiovascular disease (CVD). Campaign effectiveness was tested through an experimental design with post-test knowledge of CER among African Americans in these counties compared to CER knowledge among African Americans in a comparable control county (Allen). Our campaign, based on the principles of the culture-centered approach (CCA), increased community CER knowledge in the experimental communities relative to a community that did not receive the culturally centered health information campaign. The CCA-based campaign developed by community members and distributed through the mass media, community wide channels such as health fairs and church meetings, postcards, and face-to-face interventions explaining the postcards improved CER knowledge in specific areas (ACE-I/ARBs, atrial fibrillation, and renal artery stenosis) in the CCA communities as compared to the control community.

The Qualitative Descriptive Approach in International Comparative Studies: Using Online Qualitative Surveys.

International comparative studies constitute a highly valuable contribution to public policy research. Analysing different policy designs offers not only a mean of knowing the phenomenon itself but also gives us insightful clues on how to improve existing practices. Although much of the work carried out in this realm relies on quantitative appraisal of the data contained in international databases or collected from institutional websites, countless topics may simply not be studied using this type of methodological design due to, for instance, the lack of reliable databases, sparse or diffuse sources of information, etc. Here then we discuss the use of the qualitative descriptive approach as a methodological tool to obtain data on how policies are structured. We propose the use of online qualitative surveys with key stakeholders from each relevant national context in order to retrieve the fundamental pieces of information on how a certain public policy is addressed there. Starting from Sandelowski's seminal paper on qualitative descriptive studies, we conduct a theoretical reflection on the current methodological proposition. We argue that a researcher engaged in this endeavour acts like a composite-sketch artist collecting pieces of information from witnesses in order to draw a valid depiction of reality. Furthermore, we discuss the most relevant aspects involving sampling, data collection and data analysis in this context. Overall, this methodological design has a great potential for allowing researchers to expand the international analysis of public policies to topics hitherto little appraised from this perspective.

A Multilevel Approach to Stakeholder Engagement in the Formulation of a Clinical Data Research Network.

OBJECTIVES: To ensure meaningful engagement of stakeholders (patients, clinicians, and communities) in developing the Mid-South Clinical Data Research Network (MS-CDRN), we implemented a comprehensive, multilevel approach: (1) identify barriers to involving stakeholders in governance, network design, and implementation; (2) engage stakeholders in priority setting and research topic generation; (3) develop strategies to fully integrate stakeholders in CDRN governance and oversight; and (4) solicit guidance on patient-centered tools and strategies for recruiting research participants. METHODS: We engaged stakeholders: (1) as integral research team members; (2) on oversight and advisory committees; (3) as consultants (using Community Engagement Studios); and (4) through interviews and surveys. We recruited stakeholders from community health centers, churches, barbershops, health fairs, a volunteer registry, and a patient portal. We prioritized recruitment from populations often underrepresented in research. RESULTS: During the first 18 months, we engaged 5670 stakeholders in developing the MS-CDRN. These were research team members and on governance committees (N=10), consultants (N=58), survey respondents (N=5543), and interviewees (N=59). Stakeholders identified important barriers and facilitators to engagement, developed stakeholder-informed policies, provided feedback on priority topics and research questions, and developed an intake process for data requests and interventional studies that included reviewing for appropriate patient-centeredness, patient engagement, and dissemination. DISCUSSION: Multilevel stakeholder engagement is a novel systematic approach to developing a meaningful patient-centered and patient-engaged research program. This approach allows ongoing input from highly engaged stakeholders while leveraging focused input from larger, more diverse groups to enhance the patient-centeredness of research and increase relevance to broader audiences.

Crohn's and Colitis Foundation of America Partners Patient-Powered Research Network: Patient Perspectives on Facilitators and Barriers to Building an Impactful Patient-Powered Research Network.

BACKGROUND: To build a Patient-Powered Research Networks (PPRN) that prioritizes the needs of its members who have inflammatory bowel diseases (IBD), we sought to better understand patients' preferences for what are the essential features that will facilitate and sustain engagement. METHODS: We conducted a two-phase qualitative study. Seven focus groups involving 62 participants with IBD were conducted (phase 1). Focus group results informed the phase 2 cognitive interviews, which included 13 phone interviews. Topics included experiences with IBD and research, PPRN engagement, patient-generated health data, and resources/tools to facilitate self-management. All focus groups and interviews were digitally recorded, transcribed verbatim, and analyzed in ATLAS.ti 7.5. Thematic categories were derived from the data, and codes were grouped into emergent themes and relationships. RESULTS: Four major themes emerged through inductive coding: (1) the impact of knowing; (2) participation barriers and challenges; (3) engagement and collaboration; and (4) customizable patient portal features/functionalities. Participants were motivated to participate in the PPRN because the knowledge gained from research studies would benefit both society and the individual. Main concerns included credibility of online resources, pharmaceutical industry profiting from their data, data security, and participation expectations. Participants wanted a true and equal partnership in every phase of building a PPRN. Participants felt it was important to have access to personal health records and be able to track health status and symptoms. CONCLUSION: Partnering with participants throughout PPRN development was critical to understanding the needs and preferences of patients with IBDs and for shaping engagement strategies and the portal's design.

Engaging Stakeholders to Develop a Patient-centered Research Agenda: Lessons Learned From the Research Action for Health Network (REACHnet).

BACKGROUND: Patient-centered research requires a focus on the needs and priorities of patients. Because patient engagement can result in the discovery of important topics not currently prioritized by research programs, topic generation, and prioritization activities conducted with patients, caregivers, and other stakeholders are essential. To develop patient-centered research agendas for obesity and diabetes, the Research Action for Health Network conducted topic generation and prioritization activities with multistakeholder research advisory groups. OBJECTIVES: The purpose of this case study was to demonstrate how methods for engaging patients in topic generation and prioritization can be implemented in practice for the development of a patient-centered research agenda. RESEARCH DESIGN: Four multistakeholder groups comprising patients, clinicians, and researchers met 4-5 times between November 2014 and July 2015 to generate and prioritize topics for obesity and diabetes research. Topics were prioritized using an iterative engagement process, in which themes were identified and resulting topics were refined and ranked over multiple meetings. PARTICIPANTS: Sixty-four patients, clinicians, and researchers participated in 2 obesity and 2 diabetes advisory groups. The majority of participants (64.0%) were patients, followed by clinicians (23.4%), researchers (9.4%), and parents of children with diabetes (3.1%). RESULTS: Ten and 12 priority topics were identified for obesity and diabetes, respectively. The resulting research agendas were disseminated to patients, researchers, and clinicians. CONCLUSIONS: Patient engagement has the potential to enrich our understanding of patient priorities for research. The results from this process suggest that convening in-person multistakeholder groups can be an effective way to generate research topics that reflect patients' priorities. Engagement strategies should be focused not only on the development of patient-centered research topics but also on the implementation of these topics into research studies.

A Novel Stakeholder Engagement Approach for Patient-centered Outcomes Research.

INTRODUCTION/OBJECTIVES: The engagement of patients and other stakeholders is a critical element in the design of patient-centered outcomes research studies. However, methodology for scalable engagement in research management particularly activities such as operationalization of principles and setting of priorities is not well-developed. The objective of this study is to describe a novel approach for scalable stakeholder engagement in research aligned with the Patient-Centered Outcomes Research Institute (PCORI) engagement principles, which was evaluated in a national clinical data research network. MATERIALS AND METHODS: Patient, patient advocate, clinician, and researcher stakeholders were recruited from clinical sites, as well as social media sites related to the 3 conditions of focus, heart failure, obesity, and Kawasaki disease. The engagement strategy was designed, implemented, and mapped to the PCORI engagement principles. Evaluation included internal assessment and quantitative measures of online engagement. RESULTS: We operationalized the PCORI principles with 12 stakeholder engagement strategies and convened stakeholder advisory boards and online research prioritization panels to determine research priorities in a rigorous, deliberative process. A total of 46 advisors (20 patients) and 339 panelists (159 patients) actively participated. There were not significant differences between patients and clinicians in level of online engagement. Nonetheless, while patients reported a slightly greater challenge with following online discussion, they overall had a more favorable opinion about use of the online format. DISCUSSION/CONCLUSION: An efficient way to engage large numbers of representative stakeholders in research is a necessary first step to assure the public of trustworthy use of data networks for health research. This paper describes a comprehensive approach to engagement in patient-centered outcomes research management that informs ongoing development of rigorous methodologies in this area.

MoodNetwork: An Innovative Approach to Patient-centered Research.

INTRODUCTION: Research has often been viewed as a passive process by which participants enroll in studies developed by researchers. It is becoming clearer that to understand the nuances of mood episodes and how to prevent them, we need to conduct large clinical trials that have the power to investigate moderators and mediators, or catalysts and mechanisms of change. MoodNetwork, the first online, patient-centered research community for individuals with mood disorders, aims to change the way that traditional research has been conducted by involving patients, their caregivers, and advocates in the process of research. The aim of this report is to share lessons learned from developing MoodNetwork. METHODS: Participants enroll by completing a demographic survey and consent form. Once enrolled, participants are encouraged to complete optional surveys about their mood disorders and areas of research priority. Stakeholder and advocacy partners developed the website, web-based surveys, and recruitment materials. RESULTS: MoodNetwork has enrolled 4103 participants to date. Of this sample, 96.9% report experiencing depression and 79.7% endorse symptoms of mania or hypomania. Participants rated reducing stigma and alleviating symptoms as their 2 largest research priorities. Recruitment has been slower than expected. Recruiting a diverse sample has been challenging, and this impacts the Network's ability to conduct comparative effectiveness research studies. DISCUSSION: We discuss lessons learned from recruiting individuals with mood disorders to MoodNetwork, an innovative approach to conducting clinical trials. We identify and review 5 strategies for increasing enrollment as well as future directions.

Patient vs. Community Engagement: Emerging Issues.

BACKGROUND: The value proposition of including patients at each step of the research process is that patient perspectives and preferences can have a positive impact on both the science and the outcomes of comparative effectiveness research. How to accomplish engagement and the extent to which approaches to community engagement inform strategies for effective patient engagement need to be examined to address conducting and accelerating comparative effectiveness research. OBJECTIVES: To examine how various perspectives and diverse training lead investigators and patients to conflicting positions on how best to advance patient engagement. RESEARCH DESIGN: Qualitative methods were used to collect perspectives and models of engagement from a diverse group of patients, researchers and clinicians. The project culminated with a workshop involving these stakeholders. The workshop used a novel approach, combining World Café and Future Search techniques, to compare and contrast aspects of patient engagement and community engagement. SUBJECTS: Participants included patients, researchers, and clinicians. MEASURES: Group and workshop discussions provided the consensus on topics related to patient and community engagement. RESULTS: Participants developed and refined a framework that compares and contrasts features associated with patient and community engagement. CONCLUSIONS: Although patient and community engagement may share a similar approach to engagement based on trust and mutual benefit, there may be distinctive aspects that require a unique lexicon, strategies, tactics, and activities.

Building Meaningful Patient Engagement in Research: Case Study From ADVANCE Clinical Data Research Network.

BACKGROUND: Strategies to engage patients to improve and enhance research and clinical care are increasingly being implemented in the United States, yet little is known about best practices for or the impacts of meaningful patient engagement. OBJECTIVE: We describe and reflect on our patient stakeholder groups, engagement framework, experiences, and lessons learned in engaging patients in research, from generating proposal ideas to disseminating findings. SETTING: The ADVANCE (Accelerating Data Value Across a National Community Health Center Network) clinical data research network is the nation's largest clinical dataset on the safety net, with outpatient clinical data from 122 health systems (1109 clinics) in 23 states. RESULTS: Patients stakeholders codeveloped the ADVANCE engagement framework and its implementation in partnership with network leaders. In phase I of ADVANCE, patients were involved with designing studies (input on primary outcome measures and methods) and usability testing (of the patient portal). In phase II, the network is prioritizing research training, dissemination opportunities, an "ambassador" program to pair more experienced patient stakeholders with those less experienced, and evaluation of engagement activities and impacts. DISCUSSION: The ADVANCE framework for patient engagement has successfully involved a diverse group of patients in the design, implementation, and interpretation of comparative effectiveness research. Our experience and framework can be used by other organizations and research networks to support patient engagement activities.