Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing-Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative.

Real-world data (RWD) and the derivations of these data into real-world evidence (RWE) are rapidly expanding from informing healthcare decisions at the patient and health system level to influencing major health policy decisions, including regulatory approvals and coverage. Recent examples include the approval of palbociclib in combination with endocrine therapy for male breast cancer and the inclusion of RWE in the label of paliperidone palmitate for schizophrenia. This interest has created an urgency to develop processes that promote trust in the evidence-generation process. Key stakeholders and decision-makers include patients and their healthcare providers; learning health systems; health technology assessment bodies and payers; pharmacoepidemiologists and other clinical reseachers, and policy makers interested in bioethical and regulatory issues. A key to optimal uptake of RWE is transparency of the research process to enable decision-makers to evaluate the quality of the methods used and the applicability of the evidence that results from the RWE studies. Registration of RWE studies-particularly for hypothesis evaluating treatment effectiveness (HETE) studies-has been proposed to improve transparency, trust, and research replicability. Although registration would not guarantee better RWE studies would be conducted, it would encourage the prospective disclosure of study plans, timing, and rationale for modifications. A joint task force of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the International Society for Pharmacoepidemiology (ISPE) recommended that investigators preregister their RWE studies and post their study protocols in a publicly available forum before starting studies to reduce publication bias and improve the transparency of research methods. Recognizing that published recommendations alone are insufficient, especially without accessible registration options and with no incentives, a group of experts gathered on February 25 and 26, 2019, in National Harbor, Maryland, to explore the structural and practical challenges to the successful implementation of the recommendations of the ISPOR/ISPE task force for preregistration. This positioning article describes a plan for making registration of HETE RWE studies routine. The plan includes specifying the rationale for registering HETE RWE studies, the studies that should be registered, where and when these studies should be registered, how and when analytic deviations from protocols should be reported, how and when to publish results, and incentives to encourage registration. Table 1 summarizes the rationale, goals, and potential solutions that increase transparency, in addition to unique concerns about secondary data studies. Definitions of terms used throughout this report are provided in Table 2.

Expanding Data Reporting Capacity of Free and Charitable Clinics: A Quality Improvement Project.

BACKGROUND: In 2018, member clinics of the Virginia Association of Free and Charitable Clinics (VAFCC) provided over 235,500 visits to un/underinsured patients. A survey of VAFCC members found that only 67% report on clinical outcome measures and 56% do not collect social determinant of health (SDOH) data. OBJECTIVE: The purpose of this project was to determine if the provision of web-based technical assistance toolkits and peer mentoring improve quality and data reporting capacity of VAFCC member clinics. METHODS: Clinics that self-selected were provided with 16 weeks of customized interventions including SDOH Data Reporting Toolkit, CMS Quality Data Reporting Toolkit, Electronic Health Record Implementation Toolkit, and peer mentoring. RESULTS: Post-implementation, 100% of participating clinics reported that the resources provided benefited their organizations and increased their capacity to report. CONCLUSIONS: The provision of technical assistance, tangible resources, and customized peer mentoring can better equip Free and Charitable Clinics (FCCs) to tell the story of their patients' social barriers and clinical outcomes. IMPLICATIONS FOR NURSING: DNP prepared nurses working with vulnerable populations are positioned to assist FCCs in documenting their relevance in the safety net system. Enhancing the ability of FCCs to collect and report data will allow them to demonstrate the provision of high-quality care, despite limited resources.

Defining Patient Engagement in Research: Results of a Systematic Review and Analysis: Report of the ISPOR Patient-Centered Special Interest Group.

OBJECTIVES: Lack of clarity on the definition of "patient engagement" has been highlighted as a barrier to fully implementing patient engagement in research. This study identified themes within existing definitions related to patient engagement and proposes a consensus definition of "patient engagement in research." METHODS: A systematic review was conducted to identify definitions of patient engagement and related terms in published literature (2006-2018). Definitions were extracted and qualitatively analyzed to identify themes and characteristics. A multistakeholder approach, including academia, industry, and patient representation, was taken at all stages. A proposed definition is offered based on a synthesis of the findings. RESULTS: Of 1821 abstracts identified and screened for eligibility, 317 were selected for full-text review. Of these, 169 articles met inclusion criteria, from which 244 distinct definitions were extracted for analysis. The most frequently defined terms were: "patient-centered" (30.5%), "patient engagement" (15.5%), and "patient participation" (13.4%). The majority of definitions were specific to the healthcare delivery setting (70.5%); 11.9% were specific to research. Among the definitions of "patient engagement," the most common themes were "active process," "patient involvement," and "patient as participant." In the research setting, the top themes were "patient as partner," "patient involvement," and "active process"; these did not appear in the top 3 themes of nonresearch definitions. CONCLUSION: Distinct themes are associated with the term "patient engagement" and with engagement in the "research" setting. Based on an analysis of existing literature and review by patient, industry, and academic stakeholders, we propose a scalable consensus definition of "patient engagement in research."

Things Fall Apart: the British Health Crisis 2010-2020.

BACKGROUND: A very large number of studies have reported a stalling of health improvements in the UK since 2010. SOURCES OF DATA: Almost all relevant data are produced by the Office for National Statistics and other national statistical agencies. AREAS OF AGREEMENT: There has been a dramatic slowdown in life expectancy and diverging trends in infant mortality in the UK as a whole and England and Wales, respectively. AREAS OF CONTROVERSY: Many commentators are loath to describe the falls in life expectancy as actual falls or to ascribe blame to the political situation in the UK. GROWING POINTS: Health trends in the UK are worrying and raise important questions about government policies. AREAS TIMELY FOR DEVELOPING RESEARCH: These findings point to a need for greater investment in research on the political determinants of health, on the timely detection and interpretation of evidence of worsening health, and on how political and policy processes respond to such findings.

Nurse Case Manager: Measurement of Care Coordination Activities and Quality and Resource Use Outcomes When Caring for the Complex Patient With Hematologic Cancer.

BACKGROUND: The lack of coordination of care for complex patients in the hematology setting has prompted nurse case managers (NCMs) to coordinate that care. OBJECTIVES: This article aimed to identify the frequency of NCM care coordination activities and quality and resource use outcomes in the complex care of patients in the hematology setting. METHODS: NCM aggregate data from complex outpatients with hematologic cancer were retrieved from electronic health records at a comprehensive cancer center in the midwestern United States. Total volume of activities and outcomes were calculated as frequency and percentage. FINDINGS: Care coordination activities included communicating; monitoring, following up, and responding to change; and creating a proactive plan of care. Quality outcomes included improving continuity of care and change in health behavior, and resource use outcomes most documented were patient healthcare cost savings.

Surgeon work captured by the National Surgical Quality Improvement Program across specialties.

BACKGROUND: The National Surgical Quality Improvement Program (NSQIP) database is increasingly used for surgical research. However, it is unclear how well this database represents the breadth of work performed by different specialties. METHODS: Using the 2017 NSQIP participant use file and the 2017 Medicare Physician/Supplier Procedure Summary file, we evaluated (1) what proportion of surgical work is captured by NSQIP, (2) what procedures and disciplines are undersampled, and (3) the overall concordance between the NSQIP sample and a national sample. RESULTS: The NSQIP database reported at least one case for 4,463 out of the 5,272 Current Procedures Terminology codes in the Medicare file, potentially capturing 97.8% of surgical work across all 10 specialties. However, this proportion decreased to 72.1% when only procedures with at least 100 cases in NSQIP were considered. Limiting our analysis to only those procedures with 100 cases had markedly different effects by specialty. In part, this was owing to undersampling of minor procedures, which are more common in disciplines such as otolaryngology and urology. The overall association between the size of the NSQIP sample and the Medicare sample was 0.08. CONCLUSION: Although NSQIP has the potential to capture a diverse surgical caseload, some specialties and procedures are undersampled, limiting the ability for NSQIP to generate valid benchmarks. There was little correlation between the sample sizes in NSQIP and a national sample. Increasing sampling of underrepresented procedures and developing weights to scale NSQIP to a national sample would strengthen the program's ability to inform health outcomes research and provide valid comparisons across procedures and specialties.

Achieving Appropriate Model Transparency: Challenges and Potential Solutions for Making Value-Based Decisions in the United States.

Transparency in decision modeling remains a topic of rigorous debate among healthcare stakeholders, given tensions between the potential benefits of external access during model development and the need to protect intellectual property and reward research investments. Strategies to increase decision model transparency by allowing direct external access to a model's structure, source code, and data can take on many forms but are bounded between the status quo and free publicly available open-source models. Importantly, some level of transparency already exists in terms of methods and other technical specifications for published models. The purpose of this paper is to delineate pertinent issues surrounding efforts to increase transparency via direct access to models and to offer key considerations for the field of health economics and outcomes research moving forward from a US academic perspective. Given the current environment faced by modelers in academic settings, expected benefits and challenges of allowing direct model access are discussed. The paper also includes suggestions for pathways toward increased transparency as well as an illustrative real-world example used in work with the Institute for Clinical and Economic Review to support assessments of the value of new health interventions. Potential options to increase transparency via direct model access during model development include adequate funding to support the additional effort required and mechanisms to maintain security of the underlying intellectual property. Ultimately, the appropriate level of transparency requires balancing the interests of several groups but, if done right, has the potential to improve models and better integrate them into healthcare priority setting and decision making in the US context.

Navigating joint HTA, procurement, and fair pricing: evidence-based insights and practical recommendations - A meeting report from ISPOR regional conference in Warsaw, 2019.

Introduction: The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) organized its first Central Eastern European regional meeting in 2019 in Warsaw, Poland. Area covered: The scientific program of the two-day conference covered a broad range of topics presented from the perspective of the region. Specifically, the focus was on cross-country collaboration within different steps of health technology assessment (HTA) and the need for local HTA adaptations in decision-making. Expert commentary: Attended by approximately 200 delegates from many countries and by several high level ISPOR leaders, the conference provided a valuable opportunity to exchange knowledge and strengthen the scientific network among experts from different stakeholder groups on issues specific to the region.

Development of a Structured Outcomes Assessment and Implementation Program in the Pediatric Intensive Care Unit.

This article reports on the Outcomes Program (OP) that the pediatric intensive care unit (PICU) developed to (1) monitor unit-based outcomes trends and safety data, (2) systematically identify targets for process improvement, and (3) implement new projects and care protocols with the aim of improving patient care. Following development of the OP structure in 2013, the authors have coordinated the components of outcomes data and reporting, clinical performance review, outcomes committee, knowledge translation, and implementation science programs to impact practice. Through routine provider updates, educational strategies, and prioritization of focused projects that include structured implementation plans, the model of PICU care has been improved. Described herein is the development of the process to evaluate intensive care unit outcomes and address the need for programmatic change through implementation science principles. Such a process may be of use in other PICUs.

Social Work Assessment and Outcomes Measurement in Hospice and Palliative Care.

While psychosocial assessment is required in hospice, no requirement exists for documentation of psychosocial outcomes. Social work research has identified the psychosocial and spiritual domains most often and appropriate for social workers to address, and standardized assessment tools have been developed by social workers based on this research. The aims of this study were to document the current use of standardized assessment measures for assessment and documenting outcomes and specifically the use of the Social Work Assessment Tool. A national sample of 203 hospice and palliative care social workers was recruited for this mixed methods study. Results indicated that most social workers used a form developed by a nonsocial worker in the agency for the initial assessment, and many palliative care social workers were not responsible for the assessment. Fewer conducted a follow-up assessment or documented outcomes. No common standardized measure was used. This study confirmed anecdotal evidence about lack of standardized social work assessment and outcomes measurement occurring in hospice and palliative care. As the Centers for Medicare and Medicaid Services is likely to select a standardized tool to measure psychosocial outcomes, social workers have an opportunity to contribute a quantitatively driven outcomes measure, developed by social workers, that will demonstrate effectiveness of social work intervention and lead to a better understanding of the social work role in hospice and palliative care.

The Current Status of Outcomes-Based Contracting for Manufacturers and Payers: An AMCP Membership Survey.

BACKGROUND: As the United States health care system shifts from traditional volume-based payments to value-based payments, outcomes-based contracts (OBCs) are gaining popularity among payers and manufacturers as a mechanism for the shift toward value. Under this model, stakeholders hope to align drug payment and value to real-world performance metrics (e.g., biomarkers and health care resource utilization). OBJECTIVE: To understand the experiences, perceptions, and needs of payers and manufacturers related to OBCs. METHODS: The Academy of Managed Care Pharmacy (AMCP) and Xcenda conducted an online survey with AMCP payer and manufacturer members. Participants were asked a series of questions regarding their use of OBCs, barriers to implementation, and elements required in establishing successful OBCs. The importance and urgency of specific impediments to successful OBC implementation were also assessed. RESULTS: The survey was fielded May 12, 2017, to June 7, 2017, yielding 65 responses (35 payers/30 manufacturers). While a minority of payers/manufacturers had at least 1 OBC in place (20%/33%), a majority had interest in future OBC use (71%/63%). Among those with at least 1 OBC in place, 86%/80% of payers/manufacturers had renewed at least 1 OBC in the past 5 years. All payers and 60% of manufacturers with OBCs included compliance measures. Improvement in clinical outcomes was also common (71%/70%) (e.g., reaching set laboratory values goals), and 71%/60% included avoidance of unnecessary medical resource use (e.g., hospitalization and emergency department visit). The barrier most frequently identified by payers in implementing OBCs was evidence that OBCs reduced pharmacy spending (60%), while manufacturers identified the inability to obtain accurate data/outcome measures (73%) as a major limiting factor. Payers/manufacturers endorsed the use of easily measurable outcomes (91%/100%) as most important in establishing successful OBCs. Manufacturers, and to a lesser extent payers, indicated that regulations and legal issues need to be addressed to make progress in OBC implementation (e.g., safe harbor for preapproval health care economic information [77%/46%] and exemption of OBCs for best-price requirements [83%/51%]). The only exception was the clarification of regulations for discussing information outside of an FDA-approved label, in which both manufacturers and payers indicated a very strong need (100%) to be addressed. CONCLUSIONS: Surveyed AMCP members are interested in OBCs and recognize their alignment to societal health goals and health care affordability, although actual use of these contracts has been somewhat limited to date. Results from this survey indicate that there is potential for OBC use to increase as barriers and limitations are addressed. DISCLOSURES: This research was sponsored by the Academy of Managed Care Pharmacy and Xcenda. Duhig, Kaufman, and Hughes are employed by Xcenda. Saha is employed by the Academy of Managed Care Pharmacy. Smith has nothing disclose. Study concept and design were contributed by Duhig, Kaufman, Saha, and Hughes. Kaufman and Hughes collected the data, and data interpretation was performed by all the authors. The manuscript was written by Saha, Smith, and Duhig, along with Kaufman and Hughes.

[Measuring nursing sensitive outcomes in publicly funded hospitals in the Veneto Region, Italy: the development of a regional policy]. / Misurare gli esiti dell'assistenza infermieristica nelle Aziende Ospedaliere e Socio Sanitarie Locali della Regione del Veneto: sintesi dell'esperienza di sviluppo di una policy regionale.

INTRODUCTION: The indicators used in the Italian National Outcome Strategy does not include measurement of nursing care outcomes so these have not yet been assessed systematically in our country but only in the context of specific research projects. Positive and negative outcomes of nursing care have been documented in the literature, the latter associated with missed nursing care, a phenomenon that occurs when conditions are such that nurses are unable to deliver planned care to patients. OBJECTIVES: To describe the rationale, methodology and main results achieved to date in the development of a regional policy aimed at establishing a panel of indicators for monitoring nursing care-sensitive outcomes hospitals in publicly funded hospitals in the Veneto Region (northern Italy). METHODS: A plurennial, multi-method project based on [1] identification of selection criteria for a Minimum Data Set of indicators; [2] a rapid review of the literature and of the policies established internationally to measure nursing care-sensitive outcomes; [3] the establishment of an initial panel of indicators and evaluation of any critical issues with the chosen indicators, and [4] identification of the most appropriate tool for measuring missed nursing care. RESULTS: The medical and surgical units were considered to be the most suitable settings for the pilot study. Following the literature review, indicators that were already being monitored in the current regional information system were chosen, with the intent to prevent an additional administrative burden to nurses. By using a progressive consensus process, five outcome indicators (functional status, falls, pressure sores, urinary tract infections, aspiration pneumonia) and one process indicator (missed nursing care) were selected., and the tools for measuring the above-mentioned indicators and their related risks, were identified. A regional policy was then established to measure these indicators in a pilot phase, with the intent of implementing them as stable indicators to be measured in the new computerized hospital information system. CONCLUSIONS: An initial panel of nursing-sensitive outcome indicators has been defined to be used in Internal Medicine and General Surgery units of hospitals in Italy's Veneto Region. Despite its limitations, the project represents the first effort to create a regional policy to measure the contribution of nursing care to the health outcomes of patients and that will also the identification of potential relations with other variables such as personnel staffing and/or skill mix.

Involving clinical experts in prioritising topics for health technology assessment: a randomised controlled trial.

OBJECTIVES: The objective of this study was to explore whether reducing the material supplied to external experts during peer review and decreasing the burden of response would maintain review quality into prioritising research questions for a major research funder. METHODS AND ANALYSIS: Clinical experts who agreed to review documents outlining research for potential commissioning were screened for eligibility and randomised in a factorial design to two types of review materials (long document versus short document) and response modes (structured review form versus free text email response). Previous and current members of the funder's programme groups were excluded. Response quality was assessed by use of a four-point scoring tool and analysed by intention to treat. RESULTS: 554 consecutive experts were screened for eligibility and 460 were randomised (232 and 228 to long document or short document, respectively; 230 each to structured response or free text). 356 participants provided reviews, 90 did not respond and 14 were excluded after randomisation as not eligible.The pooled mean quality score was 2.4 (SD=0.95). The short document scored 0.037 (Cohen's d=0.039) extra quality points over the long document arm, and the structured response scored 0.335 (Cohen's d=0.353) over free text. The allocation did not appear to have any effect on the experts' willingness to engage with the task. CONCLUSIONS: Neither providing a short or a long document outlining suggested research was shown to be superior. However, providing a structured form to guide the expert response provided more useful information than allowing free text. The funder should continue to use a structured form to gather responses. It would be acceptable to provide shorter documents to reviewers, if there were reasons to do so. TRIAL REGISTRATION NUMBER: ANZCTR12614000167662.

Priorities for Cardiovascular Outcomes Research: A Report of the National Heart, Lung, and Blood Institute's Centers for Cardiovascular Outcomes Research Working Group.

The Centers for Cardiovascular Outcomes Research (CCORs) held a meeting to review how cardiovascular outcomes research had evolved in the decade since the National Heart, Lung, and Blood Institute 2004 working group report and to consider future directions. The conference involved representatives from governmental agencies, outcomes research thought leaders, and public and private healthcare partners. The main purposes of this meeting were to (1) advance collaborative high-yield, high-impact outcomes research; (2) identify priorities and barriers to important cardiovascular outcomes research; and (3) define future needs for the field. This report highlights the key topics covered during the meeting, including an examination of the recent history of outcomes research, an evaluation of the current academic climate, and a vision for the future of cardiovascular outcomes research.

Priorities for Patient-Centered Outcomes Research: The Views of Minority and Underserved Communities.

OBJECTIVE: To learn how minority and underserved communities would set priorities for patient-centered outcomes research (PCOR). DATA SOURCES: Sixteen groups (n = 183) from minority and underserved communities in two states deliberated about PCOR priorities using the simulation exercise CHoosing All Together (CHAT). Most participants were minority, one-third reported income <$10,000, and one-fourth reported fair/poor health. DESIGN: Academic-community partnerships adapted CHAT for PCOR priority setting using existing research agendas and interviews with community leaders, clinicians, and key informants. DATA COLLECTION: Tablet-based CHAT collected demographic information, individual priorities before and after group deliberation, and groups' priorities. PRINCIPAL FINDINGS: Individuals and groups prioritized research on Quality of Life, Patient-Doctor, Access, Special Needs, and (by total resources spent) Compare Approaches. Those with less than a high school education were less likely to prioritize New Approaches, Patient-Doctor, Quality of Life, and Families/Caregivers. Blacks were less likely to prioritize research on Causes of Disease, New Approaches, and Compare Approaches than whites. Compare Approaches, Special Needs, Access, and Families/Caregivers were significantly more likely to be selected by individuals after compared to before deliberation. CONCLUSIONS: Members of underserved communities, in informed deliberations, prioritized research on Quality of Life, Patient-Doctor, Special Needs, Access, and Compare Approaches.

[Settings-based prevention of overweight in childhood and adolescents : Theoretical foundation, determinants and intervention planning]. / Settingbezogene Prävention von Übergewicht im Kindes- und Jugendalter : Theoretische Fundierung, Einflussfaktoren und Interventionsplanung.

BACKGROUND: Childhood and adolescent overweight can still be seen as a global public health problem. Based on our socioeconomic understanding, overweight is the result of a complex interplay of a diverse array of factors acting on different levels. Hence, in addition to individual level determinants overweight prevention should also address environmental related factors as part of a holistic and integrated setting approach. OBJECTIVE: This paper aims to discuss the setting approach with regard to overweight prevention in childhood and adolescence. In addition to a summary of environmental factors and their empirical influence on the determinants of overweight, theoretical approaches and planning models of settings-based overweight prevention are discussed. RESULTS: While settings can be characterized as specific social-spatial subsystems (e. g. kindergarten, schools), living environments relate to complex subject-oriented environments that may include various subsystems. Direct social contexts, educational contexts and community contexts as relevant systems for young people contain different evidence-based influences that need to be taken into account in settings based overweight prevention. To support a theory-driven intervention, numerous planning models exist, which are presented here. DISCUSSION: Given the strengthening of environments for health within the prevention law, the underlying settings approach also needs further development with regard to overweigth prevention. This includes the improvement of the theoretical foundation by aligning intervention practice of planning models, which also has a positive influence on the ability to measure its success.