Patient-Centered Outcomes Research Research Articles

Outcomes that Matter to Youth and Families in Behavioral Health Services.

Patient-centered outcomes research helps youth and families using behavioral health services make informed decisions about treatments to help them achieve the outcomes most important to them. However, there are few efforts to identify the outcomes valued by youth and families systematically. This project aimed to support the development of behavioral health services that deliver outcomes valued by families by identifying the outcomes that youth and young adults with behavioral health needs and caregivers say matter most to them. We engaged 34 youth and young adults (YYA) with behavioral health needs, alongside 42 caregivers from six U.S. regions, in two rounds of one-hour virtual focus groups. The initial round involved participants identifying what they hoped to gain from using behavioral health services for personal, familial, and parental or child well-being and the attributes of positive service experiences. We coded responses using qualitative analytical software, culminating in synthesized reports. Subsequently, the second round entailed participants' review and refinement of initial findings. Across sessions, each group reported the top three outcomes deemed most important for children, YYA, parents, families, and their service experiences. YYA identified being understood by others, improving their interpersonal relationships, and feeling heard as the highest priority behavioral health service outcomes. Caregivers of children and youth with behavioral health needs identified having accessible services that meet their needs, having providers that collaborate effectively with parents and other service systems, and experiencing consistent and continuous behavioral health care for their child as the most important behavioral health service outcomes. Both YYA with behavioral health needs and caregivers of children and youth prioritized gaining the necessary knowledge, resources, and tools to support their or their child's behavioral health. Additionally, both participant groups emphasized the importance of effective communication with providers, within their families, and with peers. Minimizing judgment and stigma from society, providers, and other professionals also emerged as a critical outcome for these groups. It is essential for research and policy development to focus on and cater to the outcomes that are important and valued by YYA and their families to maximize family engagement in care.

Hormonal Treatments and Vaginal Moisturizers for Genitourinary Syndrome of Menopause : A Systematic Review.

Postmenopausal women commonly experience vulvovaginal, urinary, and sexual symptoms associated with genitourinary syndrome of menopause (GSM). To evaluate effectiveness and harms of vaginal estrogen, nonestrogen hormone therapies, and vaginal moisturizers for treatment of GSM symptoms. Medline, Embase, and CINAHL through 11 December 2023. Randomized controlled trials (RCTs) of at least 8 weeks' duration enrolling postmenopausal women with at least 1 GSM symptom and reporting effectiveness or harms of hormonal interventions or vaginal moisturizers. Risk of bias and data extraction were performed by one reviewer and verified by a second reviewer. Certainty of evidence (COE) was assessed by one reviewer and verified by consensus. From 11 993 citations, 46 RCTs evaluating vaginal estrogen (k = 22), nonestrogen hormones (k = 16), vaginal moisturizers (k = 4), or multiple interventions (k = 4) were identified. Variation in populations, interventions, comparators, and outcomes precluded meta-analysis. Compared with placebo or no treatment, vaginal estrogen may improve vulvovaginal dryness, dyspareunia, most bothersome symptom, and treatment satisfaction. Compared with placebo, vaginal dehydroepiandrosterone (DHEA) may improve dryness, dyspareunia, and distress, bother, or interference from genitourinary symptoms; oral ospemifene may improve dryness, dyspareunia, and treatment satisfaction; and vaginal moisturizers may improve dryness (all low COE). Vaginal testosterone, systemic DHEA, vaginal oxytocin, and oral raloxifene or bazedoxifene may provide no benefit (low COE) or had uncertain effects (very low COE). Although studies did not report frequent serious harms, reporting was limited by short-duration studies that were insufficiently powered to evaluate infrequent serious harms. Most studies were 12 weeks or less in duration and used heterogeneous GSM diagnostic criteria and outcome measures. Few studies enrolled women with a history of cancer. Vaginal estrogen, vaginal DHEA, oral ospemifene, and vaginal moisturizers may improve some GSM symptoms in the short term. Few long-term data exist on efficacy, comparative effectiveness, tolerability, and safety of GSM treatments. Agency for Healthcare Research and Quality and Patient-Centered Outcomes Research Institute. (PROSPERO: CRD42023400684).

Complementary and Alternative Therapies for Genitourinary Syndrome of Menopause : An Evidence Map.

Women seeking nonhormonal interventions for vulvovaginal, urinary, and sexual symptoms associated with genitourinary syndrome of menopause (GSM) may seek out complementary and alternative medicine or therapies (CAMs). To summarize published evidence of CAMs for GSM. Ovid MEDLINE, EMBASE, and CINAHL from inception through 11 December 2023. Randomized controlled trials (RCTs) 8 weeks or more in duration that evaluated the effectiveness or harms of CAMs for postmenopausal women with GSM and reported 1 or more outcomes of interest, with sample sizes of 20 or more participants randomly assigned per group. Data were abstracted by 1 reviewer and verified by a second. An evidence map approach was used to organize and describe trials. Studies were organized by type of intervention, with narrative summaries for population, study characteristics, interventions, and outcomes. Fifty-seven trials were identified that investigated 39 unique interventions. Studies were typically small (n < 200), and most were done in Iran (k = 24) or other parts of Asia (k = 9). Few trials evaluated similar combinations of populations, interventions, comparators, or outcomes. Most studies (k = 44) examined natural products (that is, herbal or botanical supplements and vitamins), whereas fewer reported on mind and body practices (k = 6) or educational programs (k = 7). Most studies reported 1 or 2 GSM symptoms, mainly sexual (k = 44) or vulvovaginal (k = 30). Tools used to measure outcomes varied widely. Most trials reported on adverse events (k = 33). Only English-language studies were used. Effect estimates, risk of bias, and certainty of evidence were not assessed. There is a large and heterogeneous literature of CAM interventions for GSM. Trials were small, and few were done in North America. Standardized population, intervention, comparator, and outcomes reporting in future RCTs are needed. Agency for Healthcare Research and Quality and Patient-Centered Outcomes Research Institute. (PROSPERO: CRD42023400684).

Conducting Comparative Effectiveness, Multisite Palliative Care and Advance Care Planning Trials: Lessons Learned and Future Directions From PCORI-Funded Studies.

The Patient-Centered Outcomes Research Institute (PCORI) funded multiple large-scale comparative effectiveness clinical trials evaluating palliative care (PC) and advance care planning (ACP) healthcare delivery models. This article provides an overview of the most common barriers our investigative teams encountered while implementing these trials and the strategies we utilized to overcome these challenges, with particular attention to identifying research partners for multisite trials; addressing contracting and regulatory issues; creating a team governance structure; training and engaging study staff across sites; recruiting, consenting, and enrolling study participants; collecting PC and ACP data and study outcomes; and managing multisite collaborations. The goal of this article is to provide guidance on how to best plan for and conduct rigorous trials evaluating PC and ACP healthcare delivery interventions moving forward.

Implementing Patient-Centered Outcomes Research Institute Stakeholder Engagement Principles in Models of Palliative Care Delivery and Advance Care Planning Research.

Given the many challenges of conducting research that addresses the palliative and end-of-life care needs of patients with serious illnesses, stakeholder engagement starting from the moment of study conceptualization and design is critical to ensure successful participant recruitment, data collection, intervention delivery, data analysis, and dissemination. Guided by a conceptual model published by the Patient-Centered Outcomes Research Institute (PCORI) entitled, "Measuring What Matters for Advancing the Science and Practice of Engagement"14 and with the support of a PCORI Engagement Officer, representatives from 9 PCORI-funded study teams formed a working group to survey team members and review, outline, and describe key lessons learned and best practices for promoting stakeholder engagement in palliative care research. Almost all study teams engaged with patients/caregivers, clinicians, researchers, and health care system experts as stakeholder partners. About half the teams also included payers and training institutions as part of their stakeholder advisors as well as a range of content experts. Study teams relied on a variety of support structures and resources, and they employed 10 distinct methods for maintaining engagement. All engagement methods were generally considered to be effective by teams who used the method, though there was some variability in team-rated engagement quality of each method. Nine barriers to stakeholder engagement were identified across the 9 studies as well as 9 strategies (or facilitators) to overcome these barriers. We share examples of how stakeholder engagement impacted studies in all phases, including the preparatory phase, study initiation phase, execution phase, and data analysis/dissemination phase. Teams utilized a variety of resources and support structures as well as capitalized on multiple engagement methods for fostering stakeholder engagement, resulting in a high level of collaboration and integration.

Conduct of Large, Multisite, Comparative Clinical Effectiveness Research Studies: Learnings From the Patient-Centered Outcomes Research Institute's Palliative Care Learning Network.

Conduct of Large, Multisite, Comparative Clinical Effectiveness Research Studies: Learnings From the Patient-Centered Outcomes Research Institute's Palliative Care Learning Network.

Palliative Care and Advance Care Planning Intervention Fidelity Monitoring: Methods and Lessons Learned From PCORI-Funded Large-Scale, Pragmatic Clinical Trials.

Over the past decade, the Patient-Centered Outcomes Research Institute (PCORI) funded multiple large-scale, comparative effectiveness clinical trials evaluating palliative care and advance care planning interventions. These are complex multicomponent interventions that need robust but flexible fidelity monitoring. Fidelity is necessary to maintain both internal and external validity within palliative care intervention research and to ultimately evaluate the real-world impact of high-quality interventions. Different trials not only took varying approaches to fidelity monitoring but also uncovered both unique and common challenges and facilitators. This article summarizes 8 of these trials and highlights approaches, adaptations, barriers, and facilitators for intervention fidelity monitoring. Identifying and delivering core elements while simultaneously allowing adaptations of noncore elements is a vital part of fidelity monitoring. Dissemination of such experiences can inform both future palliative care research as well as ongoing implementation of palliative care and advance care planning interventions across diverse clinical practices. Adoption of rigorous intervention fidelity methods is critical to advancing the science and reproducibility of palliative care interventions.

Conversion practice recall and mental health symptoms in sexual and gender minority adults in the USA: a cross-sectional study.

Conversion practices are associated with psychological morbidity, yet few studies have evaluated differences between efforts to change gender identity, sexual orientation, or both. We aimed to examine the individual and joint association of conversion practice recall targeted at gender identity or sexual orientation, or both, with current mental health symptoms among sexual and gender minority people. This cross-sectional study used data from The PRIDE Study, a US-based, online, prospective cohort study of sexual and gender minority adults who were recruited through social media, digital advertisements, and sexual and gender minority community-based events and organisations. For this analysis, we included participants who completed a lifetime questionnaire in 2019-20 and a subsequent annual questionnaire in 2020-21 without missing outcome data. All questionnaires were in English. The exposure was lifetime recall of conversion practice targeting gender identity alone, sexual orientation alone, or both (versus no conversion practice). Mental health outcomes were continuous measures: Generalized Anxiety Disorder 7-item scale, Patient Health Questionnaire 9-item (depression) scale, Post-Traumatic Stress Disorder Checklist 6-item scale, and Suicide Behaviors Questionnaire-Revised scale. We used linear regression to analyse the associations of conversion practice recall and mental health symptoms, controlling for demographic and childhood factors and stratified between cisgender and transgender and gender diverse groups. Sensitivity analyses evaluated the potential impact of unmeasured confounding. Analyses were conducted in R. We included people with related lived experience in the design and implementation of this study. Of 6601 participants who completed the lifetime questionnaire in 2019-20, 4440 completed the subsequent annual questionnaire in 2020 or 2021, and 4426 did not have missing outcome data. Of the 4426 included participants, 4073 (92·0%) identified as White (either alone or in combination with other ethnoracial options), 460 (10·4%) identified with multiple ethnoracial identities, and 1923 (43·4%) were transgender and gender diverse. Participants' age ranged from 18 years to 84 years (median 31·7 years, IQR 25·5-44·1). 149 (3·4%) participants reported sexual orientation-related conversion practice alone, 43 (1·0%) reported gender identity-related conversion practice alone, and 42 (1·0%) reported both. Recalling both forms of conversion practice was most strongly associated with greater post-traumatic stress disorder (PTSD; β 2·84, 95% CI 0·94-4·74) and suicidality (2·14, 0·95-3·32) symptoms. Recall of only sexual orientation-related conversion practice was associated with greater symptoms of PTSD (1·10, 0·22-1·98). Recall of gender identity-related conversion practice alone was most strongly associated with greater depressive symptoms (3·24, 1·03-5·46). Only associations for suicidality differed between cisgender and transgender and gender diverse participants, although the latter showed higher mental health symptoms overall. Findings were moderately robust to potential sources of unmeasured confounding in sensitivity analysis. Recall of conversion practice exposure was associated with a range of mental health symptoms among sexual and gender minority people. These findings support calls to ban conversion practices because of their effects as a structural determinant of mental health. Gill Foundation, Dona Rockstand, and Patient-Centered Outcomes Research Institute.

Increasing research study engagement in minoritized populations: An example from the Black Women Inflammation and Tau Study.

Black women are sorely underrepresented in studies of Alzheimer's disease and related dementias (ADRD) despite higher rates of ADRD diagnoses than in non-Hispanic White women. There are many reasons for underrepresentation, including medical mistrust, limited access to clinical studies, and restrictive study inclusion criteria. These pervasive barriers to research participation are often not considered during study development and, if eventually thought of tend to be after the fact. Community-engaged research (CER) approaches are an effective method for reducing participation barriers. This article describes how CER approaches were used to develop the Black Women Inflammation and Tau Study (BWITS), a prospective study to identify biopsychosocial risk factors for ADRD in Black women. Guidelines discussed here for future ADRD research in diverse populations are informed by Community-Based Participatory Research (CBPR), the National Institute on Minority Health and Health Disparities (NIMHD), and the Patient-Centered Outcomes Research Institute (PCORI). HIGHLIGHTS: Understand the historical tragedies related to medical practices and research designs that may contribute to the underrepresentation of Black Americans in research studies today. Highlight community-engaged research approaches that effectively reduce participation barriers in minoritized groups. Review Community-Based Participatory Research, National Institute of Minority Health and Health Disparities, and the Patient-Centered Outcomes Research Institute guidelines for conducting research with minoritized communities. Describe using the three frameworks to inform the study development protocol for the Black Women Inflammation and Tau Study. Conclude by offering study design considerations that we hope can be a helpful starting point for others conducting research with minoritized communities.

A pragmatic, stepped-wedge, hybrid type II trial of interoperable clinical decision support to improve venous thromboembolism prophylaxis for patients with traumatic brain injury

BackgroundVenous thromboembolism (VTE) is a preventable medical condition which has substantial impact on patient morbidity, mortality, and disability. Unfortunately, adherence to the published best practices for VTE prevention, based on patient centered outcomes research (PCOR), is highly variable across U.S. hospitals, which represents a gap between current evidence and clinical practice leading to adverse patient outcomes.This gap is especially large in the case of traumatic brain injury (TBI), where reluctance to initiate VTE prevention due to concerns for potentially increasing the rates of intracranial bleeding drives poor rates of VTE prophylaxis. This is despite research which has shown early initiation of VTE prophylaxis to be safe in TBI without increased risk of delayed neurosurgical intervention or death. Clinical decision support (CDS) is an indispensable solution to close this practice gap; however, design and implementation barriers hinder CDS adoption and successful scaling across health systems. Clinical practice guidelines (CPGs) informed by PCOR evidence can be deployed using CDS systems to improve the evidence to practice gap. In the Scaling AcceptabLE cDs (SCALED) study, we will implement a VTE prevention CPG within an interoperable CDS system and evaluate both CPG effectiveness (improved clinical outcomes) and CDS implementation.MethodsThe SCALED trial is a hybrid type 2 randomized stepped wedge effectiveness-implementation trial to scale the CDS across 4 heterogeneous healthcare systems. Trial outcomes will be assessed using the RE2-AIM planning and evaluation framework. Efforts will be made to ensure implementation consistency. Nonetheless, it is expected that CDS adoption will vary across each site. To assess these differences, we will evaluate implementation processes across trial sites using the Exploration, Preparation, Implementation, and Sustainment (EPIS) implementation framework (a determinant framework) using mixed-methods. Finally, it is critical that PCOR CPGs are maintained as evidence evolves. To date, an accepted process for evidence maintenance does not exist. We will pilot a “Living Guideline” process model for the VTE prevention CDS system.DiscussionThe stepped wedge hybrid type 2 trial will provide evidence regarding the effectiveness of CDS based on the Berne-Norwood criteria for VTE prevention in patients with TBI. Additionally, it will provide evidence regarding a successful strategy to scale interoperable CDS systems across U.S. healthcare systems, advancing both the fields of implementation science and health informatics.Trial registrationClinicaltrials.gov– NCT05628207. Prospectively registered 11/28/2022, https://classic.clinicaltrials.gov/ct2/show/NCT05628207.

Mosques and Public Health Promotion: A Scoping Review of Faith-Driven Health Interventions.

Religious institutions play a crucial role in health promotion and hold significant influence in the public health field. The aim of this review is to examine outcomes of health-promoting interventions involving the use of mosques as the intervention venue, its role in promoting health behavior change, and the role of theory in each intervention. A scoping review was conducted across 17 databases for relevant publications published up to and including August 2023 that involved the use of mosques as the intervention venue. Fourteen articles met inclusion criteria and were reviewed. The studies featured a wide range of interventions. However, only eight of these studies integrated theoretical frameworks into their approaches, indicating a need for more structured guidance in this field. These theoretical frameworks included Participatory Action Research (PAR), the theory of planned behavior, behavior-change constructs, the patient-centered outcomes research (PCOR) framework, and prolonged exposure techniques within Islamic principles. The review identified three main health-focused intervention categories: mental health, prevention, and communication, each providing valuable insights into initiatives within Muslim communities. This review underscores the significance of inclusive and culturally sensitive health interventions, emphasizing the effectiveness of faith-based approaches in improving health outcomes, promoting positive health behaviors, and addressing communication and cultural barriers. The reviews findings stress the need for further research that incorporates theoretical frameworks and tailored interventions to meet the specific cultural needs of these communities, ultimately contributing to enhanced well-being within them.

Using a Patient-Centered Multicriteria Decision Analysis to Assess the Value of Multiple Sclerosis Treatments in the US: A Study Protocol.

The engagement of patients and family caregivers in value assessment is pivotal since they provide valuable contributions to assessment acceptability and relevance. The proposed study aims to use patient-centered techniques and multicriteria decision analysis (MCDA) to evaluate the values of disease-modifying therapies (DMTs) for multiple sclerosis (MS) from the perspectives of patients and family caregivers living in three 'Deep South' States of the US-Alabama, Louisiana, and Mississippi. This study will follow guidance from the Patient-Centered Outcomes Research Institute (PCORI) for patient engagement and two best practice reports for MCDA from the Professional Society for Health Economics and Outcomes Research (ISPOR) to complete value assessment. Throughout the study, we will engage multiple stakeholders, including patients, family caregivers,healthcare providers, and payers. Forty patients with MS and their family caregivers from Alabama, Louisiana, and Mississippi will be invited to participate in this study. We will intensively train them for value assessment knowledge and MCDA before we engage them in MCDA to determine the value of DMTs for MS. Our approach differs from common MCDA since we incorporated a patient-centered framework in this study. Unlikeprevious studies only briefly inform or prepare participants before the MCDA process, in this study, we will provide basicvalue assessment trainings forpatients and family caregivers to ensure they can effectively engage throughout the patient-centeredMCDAprocess. We expect this study will demonstrate that the patient-centered MCDA approach is feasible and likely leads to improved patients' and family caregivers' engagementin value assessment.

The Tennessee Heart Health Network effectiveness study: A stepped wedge cluster randomized controlled trial to assess the effectiveness of statewide quality improvement cooperative participation on cardiovascular outcomes

BackgroundCardiovascular disease (CVD) is the primary cause of premature morbidity and mortality in the United States and Tennessee ranks among the highest in CVD events. While patient-centered outcomes research (PCOR) evidence-based approaches that reach beyond the traditional doctor-patient visit hold promise to improve CVD care and prevent serious complications, most primary care providers lack time, knowledge, and infrastructure to implement these proven approaches. Statewide primary care quality improvement (QI) collaboratives hold potential to help address primary care needs, however, little is known regarding their effectiveness in improving uptake of PCOR evidence-based population health approaches and improving CVD outcomes. This study describes the design and implementation of a stepped-wedge cluster randomized controlled trial to assess the effectiveness of participation in a statewide quality improvement cooperative (The Tennessee Heart Health Network [TN-HHN]) on cardiovascular outcomes. Methods/designThe TN-HHN Effectiveness Study randomized 77 practices to 4 waves (i.e., clusters), with each wave beginning three months after the start of the prior wave and lasting for 18 months. All practice clusters received one of three Network interventions, and outcomes are measured for each three months both in the control phase and the intervention phase. Primary outcomes include Center for Medicare and Medicaid Services measures for aspirin use, blood pressure control, cholesterol control, and smoking cessation (ABCS). ConclusionsThis trial, upon its conclusion, will allow us to assess the effect of participation in a statewide quality improvement cooperative on cardiovascular outcomes as well as key contributors to successful practice transformation.

Reducing disparities in behavioral health treatment in pediatric primary care: a randomized controlled trial comparing Partnering to Achieve School Success (PASS) to usual ADHD care for children ages 5 to 11 – study protocol

BackgroundIntegrating behavioral health services into pediatric primary care can improve access to care, especially for children marginalized by poverty and racial/ethnic minority status. In primary care, a common presenting concern is attention-deficit/hyperactivity disorder (ADHD). Services in primary care for marginalized children with ADHD typically include medication alone; therapy to improve skills and build relationships is less available. This study evaluates the effectiveness of a behavioral intervention offered through primary care for marginalized families coping with ADHD (Partnering to Achieve School Success, PASS) compared to treatment as usual (TAU).MethodThree hundred participants will be randomly assigned to PASS or TAU. Participants include children ages 5 to 11 who have ADHD and are from economically marginalized families. PASS is a personalized, enhanced behavioral intervention that includes evidence-based behavior therapy strategies and enhancements to promote family engagement, increase caregiver distress tolerance, and provide team-based care to improve academic and behavioral functioning. TAU includes services offered by primary care providers and referral for integrated behavioral health or community mental health services. Outcomes will be assessed at mid-treatment (8 weeks after baseline), post-treatment (16 weeks), and follow-up (32 weeks) using parent- and teacher-report measures of service use, child academic, behavioral, and social functioning, parenting practices, family empowerment, and team-based care. Mixed effects models will examine between-group differences at post-treatment and follow-up. Analyses will examine the mediating role of parenting practices, family empowerment, and team-based care. Subgroup analyses will examine differential effects of intervention by child clinical characteristics and socioeconomic factors.DiscussionThis study is unique in targeting a population of children with ADHD marginalized by low socioeconomic resources and examining an intervention designed to address the challenges of families coping with chronic stress related to poverty.Trial registrationThis study was registered on clinicaltrials.gov (NCT04082234) on September 5, 2019, prior to enrollment of the first participant. The current version of the protocol and IRB approval date is October 4, 2023. Results will be submitted to ClinicalTrials.gov no later than 30 days prior to the due date for the submission of the draft of the final research report to the Patient-Centered Outcomes Research Institute.

Evaluating Intensity, Complexity, and Potential for Causal Inference in Social Needs Interventions

Interventions that address needs such as low income, housing instability, and safety are increasingly appearing in the health care sector as part of multifaceted efforts to improve health and health equity, but evidence relevant to scaling these social needs interventions is limited. To summarize the intensity and complexity of social needs interventions included in randomized clinical trials (RCTs) and assess whether these RCTs were designed to measure the causal effects of intervention components on behavioral, health, or health care utilization outcomes. This review of a scoping review was based on a Patient-Centered Outcomes Research Institute-funded evidence map of English-language US-based RCTs of social needs interventions published between January 1, 1995, and April 6, 2023. Studies were assessed for features related to intensity (defined using modal values as providing as-needed interaction, 8 participant contacts or more, contacts occurring every 2 weeks or more often, encounters of 30 minutes or longer, contacts over 6 months or longer, or home visits), complexity (defined as addressing multiple social needs, having dedicated staff, involving multiple intervention components or practitioners, aiming to change multiple participant behaviors [knowledge, action, or practice], requiring or providing resources or active assistance with resources, and permitting tailoring), and the ability to assess causal inferences of components (assessing interventions, comparators, and context). This review of a scoping review of social needs interventions identified 77 RCTs in 93 publications with a total of 135 690 participants. Most articles (68 RCTs [88%]) reported 1 or more features of high intensity. All studies reported 1 or more features indicative of high complexity. Because most studies compared usual care with multicomponent interventions that were moderately or highly dependent on context and individual factors, their designs permitted causal inferences about overall effectiveness but not about individual components. Social needs interventions are complex, intense, and include multiple components. Our findings suggest that RCTs of these interventions address overall intervention effectiveness but are rarely designed to distinguish the causal effects of specific components despite being resource intensive. Future studies with hybrid effectiveness-implementation and sequential designs, and more standardized reporting of intervention intensity and complexity could help stakeholders assess the return on investment of these interventions.

Experiences of underrepresented adolescent and young adult (AYA) cancer survivors engaging in research-related activities.

10073 Background: Adolescent and young adult (AYA) cancer survivors are an age-defined cohort (15-39 years at diagnosis) confronting unique biopsychosocial needs throughout their cancer experience. Underrepresented (sexual and gender minority [SGM] and/or Black, Indigenous, and People of Color [BIPOC]) AYAs face additional challenges associated with discrimination, mistrust of providers, and culturally incongruent services. As a result, many underrepresented AYAs experience suboptimal oncological care and disparate outcomes when compared to cis-gendered heterosexual and/or white peers. This study explored the experiences of underrepresented AYA cancer survivors engaging in research-related activities. Methods: This study is part of a larger Patient Centered Outcomes Research Institute (PCORI)-funded project that is evaluating multilevel facilitators and barriers to oncological care and research for underrepresented AYA cancer survivors. With support from five academic cancer centers and five community-based organizations supporting AYAs across the US, we have assembled an intersectional community advisory panel (CAP) of 10 AYAs who identify as BIPOC and/or SGM. Together with the CAP, a semistructured interview guide was developed, and from January to February 2024, we conducted video interviews. Data were analyzed using thematic analysis of verbatim transcribed interview scripts. The CAP assisted the investigative team in the analyses and interpretation of data. Results: Of the 20 participants interviewed, roughly 70% identified as BIPOC, 40% as SGM, and 10% as both BIPOC and SGM. Participants included underrepresented AYA cancer survivors who, prior to this specific study, had or had not participated in cancer-related research. Three major themes affecting underrepresented AYAs and engagement in research-related activities were discovered, and included: 1) Layering of identities, culture, and stigma; 2) The role of trusted conduits in facilitating engagement; and 3) Beyond compensation: reasons of pursuance. Conclusions: The resultant themes illuminate multilevel facilitators and barriers to research engagement among underrepresented AYA cancer survivors. Findings will assist in guiding researchers, oncology care providers and program administrators both in academic and community settings in the design of inclusive and equitable cancer care delivery programs for underrepresented AYA cancer survivors.

Race and ethnicity and pharmacy dispensing of SGLT2 inhibitors and GLP-1 receptor agonists in type 2 diabetes

Sodium-Glucose Cotransporter 2 Inhibitors (SGLT2i) and Glucagon-Like Peptide-1 Receptor Agonists (GLP-1 RA) improve cardiorenal outcomes in patients with type 2 diabetes. Equitable use of SGLT2i and GLP-1 RA has the potential to reduce racial and ethnic health disparities. We evaluated trends in pharmacy dispensing of SGLT2i and GLP-1 RA by race and ethnicity. Retrospective cohort study of patients (≥18 years) with type 2 diabetes using 2014-2022 electronic health record data from six US care delivery systems. Entry was at earliest pharmacy dispensing of any type 2 diabetes medication. We used multivariable logistic regression to evaluate the association between pharmacy dispensing of SGLT2i and GLP1-RA and race and ethnicity. Our cohort included 687,165 patients (median 6 years of dispensing data; median 60 years; 0.3% American Indian/Alaska Native (AI/AN), 16.6% Asian, 10.5% Black, 1.4% Hawaiian or Pacific Islander (HPI), 31.1% Hispanic, 3.8% Other, and 36.3% White). SGLT2i was lower for AI/AN (OR 0.80, 95% confidence interval 0.68-0.94), Black (0.89, 0.86-0.92) and Hispanic (0.87, 0.85-0.89) compared to White patients. GLP-1 RA was lower for AI/AN (0.78, 0.63-0.97), Asian (0.50, 0.48-0.53), Black (0.86, 0.83-0.90), HPI (0.52, 0.46-0.57), Hispanic (0.69, 0.66-0.71), and Other (0.78, 0.73-0.83) compared to White patients. Dispensing of SGLT2is, and GLP-1 RAs was lower in minority group patients. There is a need to evaluate approaches to increase use of these cardiorenal protective drugs in patients from racial and ethnic minority groups with type 2 diabetes to reduce adverse cardiorenal outcomes and improve health equity. Patient-Centered Outcomes Research Institute and National Institutes of Health.

Abstract PO4-13-04: Added Value from Patient Advocates in a Translational Working Group: the COMET Study

Abstract Introduction: A phase III multicenter prospective randomized clinical trial called “Comparing an Operation to Monitoring, with or without Endocrine Therapy (COMET)” (NCT02926911) assesses the risks/benefits of active monitoring (AM) versus surgery for women with low-risk ductal carcinoma in situ (DCIS). It is funded by the Patient-Centered Outcomes Research Institute and enrolled 997 women between 06/30/17 and 01/13/23. Research Patient Advocates (PAs) have been embedded from the study design stage and continue to provide guidance as part of the COMET leadership team. The COMET biobank is funded by independent foundations and is managed through monthly Translational Working Group (TWG) meetings. The TWG includes PAs, clinicians, pathologists, radiologists, and researchers from multiple institutions who discuss topics like categorization of biospecimens into discovery/validation sets; development of a pathology workflow/sample tracking process; use of small samples; and potential areas of future research/technologies that may improve DCIS diagnostics, prognostics, and care management. Methods: The TWG leveraged the existing Alliance Foundation Trials, LLC biobank infrastructure and facilitates the collection, submission, storage and analysis/use of blood, tissue, and breast images collected at specified timepoints and stored in central tissue/image repositories. PAs are active and integral in the TWG, assisting with logistical issues (contracts, transfer agreements, resource requests); communicating with sites; identifying study topics and biomarkers relevant to diverse patients; providing guidance on commercial predictive/prognostic tests; promoting effective stewardship of samples; ensuring overall focus remains on advancing clinical utility; and reporting translational study results to trial participants who agreed to donate samples/images. Results: Over 90% of requested samples/images have been submitted by ˃85 sites. Each patient’s biospecimens and pathology images are linked and de-identified for research. Use of artificial intelligence (AI) is being considered to assist in sample review. TWG members have complementary areas of expertise/experience and promote active stewardship for effective management of these scarce resources. PAs have played key roles concerning equitable data-sharing and acceleration of data and material transfer agreements. They have also been active in development of standards for discovery/training sets, validation test sets; communication procedures for patient cases that progress to future breast events; ways to catalog technologies (e.g., multi-omics, AI); and in research proposal review. Consensus has been achieved regarding major issues such as authorship, biospecimen custodianship, intellectual property, and criteria relevant to patient needs. Logistical barriers, including data sharing and technicalities of biospecimen release, have been resolved. The TWG also played an integral role in resolving recruitment challenges to COMET by creating standard pathology eligibility criteria, resulting in evidence-based protocol amendments that increased accrual. A retrospective review of biospecimens has been performed to determine adequacy for ensuing correlative molecular and spatial profiling studies. Conclusion: PAs help the COMET TWG set policy, and oversee biospecimen/image collection, and biobank use and sharing. PA input also facilitates equitable, transparent research studies and technology development that can improve personalized decisions for surgery versus AM in women with low-risk DCIS. PAs in the TWG aim to integrate the diagnostic and prognostic tools developed as part of the COMET study into future patient care. Citation Format: Deborah Collyar, Desiree Basila, Thomas Lynch, Stuart Schnitt, Jeffrey Marks, Siri Strand, Terry Hyslop, Sunil Badve, Mark Watson, H. T. Carisa Le-Petross, Lars Grimm, Robert West, Anna Weiss, Anna Rapperport, Lorraine King, Rachel Factor, Marc Ryser, Ann Partridge, E Shelley Hwang, Alastair Thompson. Added Value from Patient Advocates in a Translational Working Group: the COMET Study [abstract]. In: Proceedings of the 2023 San Antonio Breast Cancer Symposium; 2023 Dec 5-9; San Antonio, TX. Philadelphia (PA): AACR; Cancer Res 2024;84(9 Suppl):Abstract nr PO4-13-04.

Improving Data Infrastructure for Person-Centered Outcomes Research on Intellectual and Developmental Disabilities.

Individuals with intellectual and developmental disabilities (IDD) continue to experience disparities in health and well-being despite improved provisions of person-centered care. Patient-centered outcomes research (PCOR) translates evidence into practice for meaningful outcomes. This piece describes findings from an environmental scan and stakeholder outreach to identify and prioritize opportunities to enhance IDD PCOR data infrastructure. These opportunities include developing a standardized research definition; advancing data standards for service systems; improving capture of IDD at point of care; developing standardized outcome measures; and encouraging Medicaid data use for IDD research. Within this piece, we discuss the implications of addressing data gaps for enhanced research. While the identified activities provide a path towards advancing IDD PCOR data infrastructure, collaborative efforts between government, researchers, and others are paramount.

Abstract 1302: Development and implementation of a systematic approach to measure patient-reported outcomes in early-phase cancer trials

Abstract Background: There has been a shift in the clinical trial landscape toward early-phase studies where data from well-designed and pivotal phase I/II trials is leading to accelerated drug approval. Our goal was to develop a standardized approach to the measurement and reporting of patient-reported outcomes (PROs) in early-phase trials to improve assessment of tolerability, address noted barriers to inclusion, and enhance patient-provider communication to inform treatment decisions and ultimately improve outcomes. Methods: The Office of Patient Centered Outcomes Research (OPCORe) was established in 2022 within the Center for Cancer Research at the National Cancer Institute (NCI) with the mission to advance understanding of the clinical benefit and tolerability of cancer therapies by integrating patient-centered approaches into CCR clinical trials by fostering education and collaboration with stakeholders. Implementation of regulatory/consortia guidelines related to use of core constructs, assessment frequency, and hypothesis-driven objectives and analysis plans through protocol template, development of data capture, email-based alerts and data visualization for clinical providers was developed and standardized templates for protocol language, research staff education, tools for workflow, and tracking implemented. Results: The OPCORe protocol template includes measurement of 3 constructs, including tolerability, clinical benefit and disease-specific impact, as well as anchors of meaningful change. Core symptomatic toxicities are measured to inform understanding across trials with agent/class-specific symptomatic toxicities added to assure relevance. Baseline and standardized weekly electronic capture of tolerability during early cycles with alerts to clinical research staff if moderate-severe toxicities reported helps facilitate patient check-ins. Repeat PROs assessments in later cycles are expanded to include disease-specific assessments and align with disease evaluation to further inform clinical impact. To date, we have collaborated with 7 research groups and developed approaches for first-in-human, phase I, and phase II studies with trial start-up and data collection in process. Conclusion: The value of including PROs in cancer trials is increasingly recognized by regulators, clinicians, patients, and advocates to advance understanding of the clinical benefit &amp; tolerability of emerging therapies. Standardization and incorporation of PROs in earlier phase trials may enable PROs to be optimized, improve later phase trial design, and help inform health policy, support drug approval, and promote shared decision making between providers and patients to inform treatment decisions. Citation Format: Amanda L. King, Jason E. Levine, Elizabeth Vera, Yuti Gandhi, Elaine Novakovich, Theresa Yu, Sefanit Berhanu, Ciara Locke, Kaelyn Julmeus, Tito Mendoza, Terri S. Armstrong. Development and implementation of a systematic approach to measure patient-reported outcomes in early-phase cancer trials [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 1302.