Accelerating evidence generation: Addressing critical challenges and charting a path forward.

Efficient evidence generation to assess the clinical and economic impact of medical therapies is critical amid rising healthcare costs and aging populations. However, drug development and clinical trials remain far too expensive and inefficient for all stakeholders. On October 25-26, 2023, the Duke Clinical Research Institute brought together leaders from academia, industry, government agencies, patient advocacy, and nonprofit organizations to explore how different entities and influencers in drug development and healthcare can realign incentive structures to efficiently accelerate evidence generation that addresses the highest public health needs.

Navigating the Path to Inclusion: Understanding Barriers and Facilitators to Clinical Trial Participation Among Chinese Older Adults in the United States with Multimorbidity.

Context: Older adults with multimorbidity are underrepresented in clinical trials, with enrollment of Asians particularly low.

Objective: Understand perspectives of US Chinese older adults regarding clinical trial participation.

Study Design And Analysis: Focus group interviews analyzed using thematic analysis.

Filled Opioid Prescriptions Following Pediatric Dental Procedures Among Medicaid-Insured Children in Ohio.

To investigate opioid prescription fills following pediatric/adolescent dental procedures in central/southeastern Ohio. This population-based, retrospective cohort study utilized health insurance claims from a pediatric public accountable care organization (???Medicaid???) in central/southeastern Ohio. Patients aged 18 years and younger who had a dental procedure between January 2012 and February 2019 were identified, and claims were searched for opioid prescription fills within 14 days post-procedure.

Framework of the strengths and challenges of clinically integrated trials: An expert panel report.

The limitations of the explanatory clinical trial framework include the high expense of implementing explanatory trials, restrictive entry criteria for participants, and redundant logistical processes. These limitations can result in slow evidence generation that is not responsive to population health needs, yielding evidence that is not generalizable. Clinically integrated trials, which integrate clinical research into routine care, represent a potential solution to this challenge and an opportunity to support learning health systems.

Using a human-factors engineering approach to evaluate environmental cleaning in Veterans' Affairs acute and long-term care facilities: A qualitative analysis.

Background: Environmental cleaning is important in the interruption of pathogen transmission. Although prevention initiatives have targeted environmental cleaning, practice variations exist and compliance is low. Evaluation of human factors influencing variations in cleaning practices can be valuable in developing interventions to standardized practices.

Examining how study teams manage different viewpoints and priorities in patient-centered outcomes research: Results of an embedded multiple case study.

Introduction: Limited evidence exists about which patient and stakeholder engagement practices support or hinder study teams as they negotiate different viewpoints in decisions about the design and conduct of patient-centered outcomes research.

Methods: We applied a multiple-embedded descriptive case study design for six studies funded by the Patient-Centered Outcomes Research Institute (PCORI). We interviewed 32 researchers and stakeholder partners, including patients, caregivers and clinicians, and reviewed documents related to each study (e.

Evaluation of daily environmental cleaning and disinfection practices in veterans affairs acute and long-term care facilities: A mixed methods study.

Objectives: To describe daily environmental cleaning and disinfection practices and their associations with cleaning rates while exploring contextual factors experienced by healthcare workers involved in the cleaning process.

Methods: A convergent mixed methods approach using quantitative observations (ie, direct observation of environmental service staff performing environmental cleaning using a standardized observation form) and qualitative interviews (ie, semistructured interviews of key healthcare workers) across 3 Veterans Affairs acute and long-term care facilities.

Results: Between December 2018 and May 2019 a total of sixty-two room observations (N = 3602 surfaces) were conducted.

Health funders' dissemination and implementation practices: results from a survey of the Ensuring Value in Research (EViR) Funders' Forum.

Background: A significant gap persists between evidence from research and its use in practice. Research funders, important actors in the health research system, can help reduce this gap by initiating dissemination and implementation (D&I) activities. The specific types of D&I activities funders currently lead have not been explored thoroughly.

Understanding the Influence and Impact of Stakeholder Engagement in Patient-centered Outcomes Research: a Qualitative Study.

Background: Engaging patients and other stakeholders as partners in research offers promise in improving the relevance and usefulness of research findings.

Objective: To explore the influence and impact of patient and other stakeholder engagement on the planning and conduct of comparative effectiveness research studies.

Design: Qualitative study with virtual, hour-long semi-structured interviews.

GA4GH: International policies and standards for data sharing across genomic research and healthcare.

The Global Alliance for Genomics and Health (GA4GH) aims to accelerate biomedical advances by enabling the responsible sharing of clinical and genomic data through both harmonized data aggregation and federated approaches. The decreasing cost of genomic sequencing (along with other genome-wide molecular assays) and increasing evidence of its clinical utility will soon drive the generation of sequence data from tens of millions of humans, with increasing levels of diversity. In this perspective, we present the GA4GH strategies for addressing the major challenges of this data revolution.

Empirical validation of an automated approach to data use oversight.

The current paradigm for data use oversight of biomedical datasets is onerous, extending the timescale and resources needed to obtain access for secondary analyses, thus hindering scientific discovery. For a researcher to utilize a controlled-access dataset, a data access committee must review her research plans to determine whether they are consistent with the data use limitations (DULs) specified by the informed consent form. The newly created GA4GH data use ontology (DUO) holds the potential to streamline this process by making data use oversight computable.

Understanding and applying the RE-AIM framework: Clarifications and resources.

Introduction: Understanding, categorizing, and using implementation science theories, models, and frameworks is a complex undertaking. The issues involved are even more challenging given the large number of frameworks and that some of them evolve significantly over time. As a consequence, researchers and practitioners may be unintentionally mischaracterizing frameworks or basing actions and conclusions on outdated versions of a framework.

Drivers of differential views of health equity in the U.S.: is the U.S. ready to make progress? Results from the 2018 National Survey of Health Attitudes.

Objectives: The public health sector has long recognized the role of the social determinants of health in health disparities and the importance of achieving health equity. We now appear to be at an inflection point, as we hear increasing demands to dismantle structures that have perpetuated inequalities. Assessing prevailing mindsets about what causes health inequalities and the value of health equity is critical to addressing larger issues of inequity, including racial inequity and other dimensions.

Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI).

Introduction: Complex health interventions (CHIs) are increasingly studied in comparative effectiveness research (CER), and there is a need for improvements in CHI research practices. The Patient-Centered Outcomes Research Institute (PCORI) Methodology Committee (MC) launched an effort in 2016 to develop formal guidance on this topic.

Objective: To develop a set of minimal standards for scientifically valid, transparent, and reproducible CER studies of CHIs.

Hydroxychloroquine in Patients with Rheumatic Disease Complicated by COVID-19: Clarifying Target Exposures and the Need for Clinical Trials.

Objective: To characterize hydroxychloroquine (HCQ) exposure in patients with rheumatic disease receiving longterm HCQ compared to target concentrations with reported antiviral activity against the coronavirus disease 2019 caused by SARS-CoV-2 (COVID-19).

Methods: We evaluated total HCQ concentrations in serum and plasma from published literature values, frozen serum samples from a pediatric systemic lupus erythematosus trial, and simulated concentrations using a published pharmacokinetic model during pregnancy. For each source, we compared observed or predicted HCQ concentrations to target concentrations with reported antiviral activity against SARS-CoV-2.

A review and synthesis of frameworks for engagement in health research to identify concepts of knowledge user engagement.

Background: Engaging those who influence, administer and/or who are active users ("knowledge users") of health care systems, as co-producers of health research, can help to ensure that research products will better address real world needs. Our aim was to identify and review frameworks of knowledge user engagement in health research in a systematic manner, and to describe the concepts comprising these frameworks.

Methods: An international team sharing a common interest in knowledge user engagement in health research used a consensus-building process to: 1) agree upon criteria to identify articles, 2) screen articles to identify existing frameworks, 3) extract, analyze data, and 4) synthesize and report the concepts of knowledge user engagement described in health research frameworks.

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.

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.

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).