Patient-Centered Outcomes Research Research Articles

263-OR: Older Adults Benefit from Virtual Support for CGM Use but Require Longer Visits

The Virtual Diabetes Specialty Clinic (VDiSC) provided type 1 (T1D) and type 2 diabetes (T2D) adults taking insulin virtual care with remote CGM training and support. HbA1c improved from baseline to 6 months in T1D (7.8% to 7.0%) and T2D (8.2% to 7.0%) with increased time in range (70-180 mg/dL; TIR). Here we compare glycemic outcomes (Table 1) and duration of remote CGM training visits (Table 2) in adults ages <40 yr, 40-59 yr and ≥ 60 yr. HbA1c and %TIR improved in each group. Limitations include a small sample size that was mostly White. Results suggest that both T1D and T2D adults ≥60 years of age experience glycemic benefit after remote training and support in the use of CGM from CDCESs, but when compared to young adults, older adults require more time for training. Support for approaches that address the individual needs of older adults with diabetes is required. Disclosure R.S.Weinstock: Consultant; Jaeb Center for Health Research, Other Relationship; Wolters Kluwer Health, Research Support; Insulet Corporation, Medtronic, Eli Lilly and Company, Novo Nordisk, Boehringer Ingelheim Inc., Hemsley Charitable Trust, National Institute of Diabetes and Digestive and Kidney Diseases, Tandem Diabetes Care, Inc., Kowa Pharmaceuticals America, Inc. T.S.Mcarthur: None. B.A.Olson: Stock/Shareholder; Abbott. S.Oser: Advisory Panel; Cecelia Health, Dexcom, Inc., Consultant; Medscape, Research Support; Abbott Diabetes. T.Oser: Advisory Panel; Cecelia Health, Consultant; Dexcom, Inc., Medscape, Research Support; Abbott. D.Raghinaru: None. R.Beck: Consultant; Eli Lilly and Company, Novo Nordisk, Diasome, Insulet Corporation, Research Support; Tandem Diabetes Care, Inc., Beta Bionics, Inc., Dexcom, Inc., Bigfoot Biomedical, Inc., Medtronic, Ascensia Diabetes Care, Roche Diabetes Care, Eli Lilly and Company, Novo Nordisk. G.Aleppo: Advisory Panel; Medscape, Consultant; Bayer Inc., Insulet Corporation, Research Support; Dexcom, Inc., Eli Lilly and Company, Emmes, Insulet Corporation, Fractyl Health, Inc., WellDoc, Speaker's Bureau; Dexcom, Inc. Z.Thompson: None. R.M.Bergenstal: Advisory Panel; Abbott Diabetes, Eli Lilly and Company, Medtronic, Novo Nordisk, Roche Diabetes Care, Zealand Pharma A/S, Consultant; Ascensia Diabetes Care, Bigfoot Biomedical, Inc., CeQur SA, Dexcom, Inc., Hygieia, Onduo LLC, Sanofi, Vertex Pharmaceuticals Incorporated, Research Support; Abbott Diabetes, Dexcom, Inc., Eli Lilly and Company, Insulet Corporation, Medtronic, Novo Nordisk, Sanofi, UnitedHealth Group. T.L.Cushman: None. R.L.Gal: None. C.Kollman: Research Support; Insulet Corporation, Dexcom, Inc., Tandem Diabetes Care, Inc. D.F.Kruger: Advisory Panel; Abbott Diabetes, Lilly, Medtronic, Novo Nordisk, Research Support; Dexcom, Inc., Beta Bionics, Inc., Speaker's Bureau; Dexcom, Inc., Lilly, Xeris Pharmaceuticals, Inc., Novo Nordisk. K.K.Hood: Consultant; Cecelia Health. M.L.Johnson: Research Support; Abbott, Lilly, Insulet Corporation, NIH - National Institutes of Health, Patient-Centered Outcomes Research Institute, Novo Nordisk, Tandem Diabetes Care, Inc., Medtronic, Hemsley Charitable Trust, Jaeb Center for Health Research. Funding The Leona M. and Harry B. Helmsley Charitable Trust; Dexcom, Inc.

870-P: Empagliflozin and the Risk of Gout—Analysis from the Empagliflozin Comparative Effectiveness and Safety (EMPRISE) Study

Hyperuricemia is frequently observed in patients with type 2 diabetes (T2D) and is associated with increased risk of gout. Empagliflozin lowers serum urate levels by enhancing its urinary excretion. Using claims data from 2 commercial insurance datasets and Medicare (2014-2019), we evaluated the association between treatment with empagliflozin and the risk of incident gout in routine clinical practice. After excluding patients with a history of gout or gout medications, we identified 102,262 pairs of 1:1 propensity score-matched adults with T2D initiating empagliflozin or a DPP4 inhibitor (DPP4i) (Cohort 1), and 131,216 pairs initiating empagliflozin or a GLP-1 receptor agonist (GLP-1RA) (Cohort 2). We estimated pooled HR and RD/1,000 PY with their 95% CI adjusting for 141 baseline covariates and conducted stratified analyses within multiple subgroups (Table). Over a mean follow-up of ~8 months on treatment, the risk of gout was lower in patients initiating empagliflozin compared with patients initiating DPP4i [HR, 0.69 (0.60, 0.79); RD -2.27 (-3.08, -1.46)] or GLP-1RA [HR 0.83 (0.73, 0.94); RD -0.99 (-1.66, -0.32)]. Results were consistent within sex, age, BMI, HF, CKD subgroups and among users of diuretics (Table). In clinical practice, the initiation of empagliflozin was associated with a lower risk of incident gout compared with the initiation of DPP4i or GLP-1RA. Disclosure H.Tesfaye: None. J.M.Paik: None. L.Zabotka: None. P.T.Htoo: Employee; Johnson & Johnson. N.Schmedt: Employee; Boehringer Ingelheim International GmbH. L.Koeneman: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. L.J.Seman: Employee; Boehringer Ingelheim Pharmaceuticals Inc. D.J.Wexler: Other Relationship; Novo Nordisk A/S. E.Patorno: Research Support; Boehringer Ingelheim Inc., National Institutes of Health, Patient-Centered Outcomes Research Institute, US Food and Drug Administration.

1227-P: Cardiorenal Effectiveness of Empagliflozin vs. GLP-1 Receptor Agonists in Patients with Advanced Chronic Kidney Disease—Results from the EMPRISE Study

Patients with chronic kidney disease (CKD) are at an increased risk of cardiovascular disease, heart failure, and mortality. We reported the final year results of the EMPRISE study program in patients with type 2 diabetes (T2D) and advanced CKD. Using Medicare, Optum, and Marketscan data (2014-19), we identified 10,930 pairs of 1:1 propensity score-matched patients aged >18 years with T2D and CKD stage 3-4 who initiated empagliflozin (EMPA) or a glucagon-like peptide-1 receptor agonist (GLP-1RA). Primary outcomes were hospitalization for heart failure (HHF), a composite of myocardial infarction (MI) or stroke, and end-stage renal disease (ESRD). Secondary outcomes were MI, stroke, and all-cause mortality. We estimated hazard ratios (HR) and rate differences (RD), adjusting for 143 baseline covariates. Compared to GLP-1RA, EMPA was associated with a reduced risk of HHF [HR 0.68 (0.55-0.85); RD -9.44 (-14.78, -4.10)], and ESRD [HR 0.70 (0.56-0.87); RD -9.01 (-14.30, -3.72)], and a trend towards reduced risk for the composite outcome [HR 0.88 (0.72-1.07); RD -3.84 (-9.60, 1.93)]. Estimates for the secondary outcomes were: MI [HR 0.78 (0.61-1.00)], stroke [HR 1.16 (0.83-1.62)], and all-cause mortality [HR 1.08 (0.86-1.36)]. In patients with T2D and advanced CKD in routine care, we observed risk reductions for cardiorenal outcomes with EMPA vs. GLP-1RA. Disclosure P.T.Htoo: Employee; Johnson & Johnson. E.Patorno: Research Support; Boehringer Ingelheim Inc., National Institutes of Health, Patient-Centered Outcomes Research Institute, US Food and Drug Administration. H.Tesfaye: None. D.J.Wexler: Other Relationship; Novo Nordisk A/S. R.Glynn: Research Support; Amarin Corporation, AstraZeneca, Kowa Pharmaceuticals America, Inc., Novartis, Pfizer Inc. N.Schmedt: Employee; Boehringer Ingelheim International GmbH. L.Koeneman: Employee; Eli Lilly and Company, Stock/Shareholder; Eli Lilly and Company. S.Schneeweiss: Consultant; Aetion, Inc., Research Support; UCB, Inc., Boehringer Ingelheim International GmbH, Stock/Shareholder; Aetion, Inc. J.M.Paik: None. Funding Boehringer Ingelheim (116283)

149-LB: Telemedicine Intervention Impact on Diabetes Self-Management—Twelve-Month Virtual Diabetes Specialty Clinic (VDISC) Study Outcomes

The impact of a telemedicine model for a virtual diabetes clinic was assessed 6 months following completion of a 6-month intervention of education and support for diabetes self-management, including initiation and use of CGM. One hundred and sixty-one participants ≥18 years old with T1D (N=109) or T2D (N=52) using MDI/pump participated in the 12-month study. At the end of the 6-month intervention period, mean HbA1c decreased from 7.6% at baseline to 7.0% in T1D and from 8.1% to 7.0% in T2D (P<0.001). At 12 months (6 months after discontinuation of the intervention), mean HbA1c was 7.0% for T1D and 7.2% for T2D (P<0.001 compared with baseline). Mean time in range 70-180 mg/dL was 51% at baseline, increasing to 62% at 6 months (P<0.001) and 64% at 12 months in T1D (P<0.001) and from 49% to 66% (P<0.001) and 68% (P<0.001), respectively, in T2D. Similar improvements were observed for mean glucose and hyperglycemia metrics. A sustained reduction in hypoglycemia also was observed. The virtual diabetes clinic intervention was successful in promoting improved glycemic outcomes, which were sustained 6 months after the intervention was completed. These findings demonstrate the impact that virtual care and education can have on self-management over time and provide opportunity to expand care models that could minimize barriers to specialty care access. Disclosure R. L. Gal: None. S. Oser: Advisory Panel; Cecelia Health, Dexcom, Inc., Consultant; Medscape, Research Support; Abbott Diabetes. T. Oser: Advisory Panel; Cecelia Health, Consultant; Dexcom, Inc., Medscape, Research Support; Abbott. K. K. Hood: Consultant; Cecelia Health. T. L. Cushman: None. M. L. Johnson: Research Support; Abbott, Lilly, Insulet Corporation, NIH - National Institutes of Health, Patient-Centered Outcomes Research Institute, Novo Nordisk, Tandem Diabetes Care, Inc., Medtronic, Hemsley Charitable Trust, Jaeb Center for Health Research. C. Kollman: Research Support; Insulet Corporation, Dexcom, Inc., Tandem Diabetes Care, Inc. R. Beck: Consultant; Eli Lilly and Company, Novo Nordisk, Diasome, Insulet Corporation, Research Support; Tandem Diabetes Care, Inc., Beta Bionics, Inc., Dexcom, Inc., Bigfoot Biomedical, Inc., Medtronic, Ascensia Diabetes Care, Roche Diabetes Care, Eli Lilly and Company, Novo Nordisk. D. Raghinaru: None. G. Aleppo: Advisory Panel; Medscape, Consultant; Bayer Inc., Insulet Corporation, Research Support; Dexcom, Inc., Eli Lilly and Company, Emmes, Insulet Corporation, Fractyl Health, Inc., WellDoc, Speaker's Bureau; Dexcom, Inc. B. A. Olson: Stock/Shareholder; Abbott. D. F. Kruger: Advisory Panel; Abbott Diabetes, Lilly, Medtronic, Novo Nordisk, Research Support; Dexcom, Inc., Beta Bionics, Inc., Speaker's Bureau; Dexcom, Inc., Lilly, Xeris Pharmaceuticals, Inc., Novo Nordisk. R. M. Bergenstal: Advisory Panel; Abbott Diabetes, Eli Lilly and Company, Medtronic, Novo Nordisk, Roche Diabetes Care, Zealand Pharma A/S, Consultant; Ascensia Diabetes Care, Bigfoot Biomedical, Inc., CeQur SA, Dexcom, Inc., Hygieia, Onduo LLC, Sanofi, Vertex Pharmaceuticals Incorporated, Research Support; Abbott Diabetes, Dexcom, Inc., Eli Lilly and Company, Insulet Corporation, Medtronic, Novo Nordisk, Sanofi, UnitedHealth Group. R. S. Weinstock: Consultant; Jaeb Center for Health Research, Other Relationship; Wolters Kluwer Health, Research Support; Insulet Corporation, Medtronic, Eli Lilly and Company, Novo Nordisk, Boehringer Ingelheim Inc., Hemsley Charitable Trust, National Institute of Diabetes and Digestive and Kidney Diseases, Tandem Diabetes Care, Inc., Kowa Pharmaceuticals America, Inc. A. Bradshaw: None. T. S. Mcarthur: None.

PCR195 Developing a Framework to Capture Economic Impacts on Patients and Caregivers: Recommendations to Guide Patient-Centered Outcomes Research and Value Assessment

Researchers and decisionmakers have begun to recognize that economic impacts of serious health conditions reach beyond traditional measures of patient and caregiver costs. However, significant questions remain about how and when to include a fuller range of economic impacts in patient-centered research and in health technology assessment. This project aims to: 1) create a decision framework that defines “economic impacts on patients and caregivers,” 2) identify when economic impact data should be collected and for what purpose, and 3) highlight promising approaches to collecting economic impact data.

From the lab to the clinic: Lessons learned from a translational working group.

e12576 Background: Comparing an Operation to Monitoring, with or without Endocrine Therapy (COMET), is a phase III multicenter prospective randomized clinical trial comparing the risks/benefits of active monitoring (AM) versus surgery for women with low-risk DCIS. Funded by the Patient-Centered Outcomes Research Institute, accrual opened 06/30/17 and closed 01/13/23 with 995 women enrolled. Biospecimens (blood/tissue) and breast images were collected at specified timepoints (funded by the Breast Cancer Research Foundation) and stored in central tissue/image repositories. To date, more than 90% of requested samples/images have been submitted. Methods: In 2017, the COMET Translational Working Group (TWG) was formed to facilitate the collection, submission, storage, and subsequent analysis of biospecimens/images. The TWG, consisting of clinicians, pathologists, radiologists, researchers, and patient advocates (PAs), discuss topics such as categorization of biospecimens into discovery/validation sets; development of a pathology workflow/sample tracking process; and potential areas of future research that may improve DCIS diagnostics, prognostics, and care management. Results: The TWG consists of multiple partner institutions, and team members have complementary areas of expertise/experience. While competing views may exist, consensus has been achieved in relation to major issues such as authorship, biospecimen ownership, intellectual property and criteria relevant to patient needs, through targeted discussion at monthly meetings. Logistical barriers, including data sharing and the technicalities of biospecimen release, have also been resolved through this process. The TWG has played an integral role in resolving recruitment challenges. In 2019, guidance from the TWG related to standardizing pathology eligibility criteria resulted in evidence-based protocol amendments that subsequently increased accrual. A retrospective review of biospecimens was performed to determine adequacy for ensuing correlative molecular and spatial profiling studies. PAs are integral to the TWG, assisting with logistical issues (resource requests); identifying study topics relevant to patients; providing guidance on existing (commercial) predictive/prognostic tests; promoting effective stewardship of a finite resource (tissue samples); and ensuring that the overall focus of the TWG remains firmly on advancing clinical utility. Conclusions: The COMET TWG serves the important role of overseeing biospecimen/image collection including its use and sharing. This facilitates development of research and technology that can impact the decision for surgery vs AM in women with low-risk DCIS, and ensure the translation of research findings into everyday clinical practice. Subsequent correlative science studies identified by the TWG stand to improve future DCIS pathology diagnostics, prognostics and care management.

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

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. 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, caregiversand clinicians, and reviewed documents related to each study (e.g., publications, and progress reports submitted to PCORI). Overall, researchers reported that incorporating different viewpoints was a strength or opportunity to learn rather than something to be avoided or dreaded. Across cases, different viewpoints and priorities, often related to ethical or pragmatic considerations, emerged between researchers and stakeholders, between stakeholder groups (e.g., patients and clinicians)or within groups (e.g., amongst researchers). Examples of navigating different viewpoints arose across study phases. The length of time to resolve issues depended on how strongly people disagreed and the perceived importance or impact of decisions on the study. All cases used collaborative decision-making approaches, often described as consensus, throughout the study. Interviewees described consensus as using negotiation, compromiseor working towards an agreeable decision. To encourage consensus, cases actively facilitated group discussions with an openness to diverse opinions, remained flexible and open to trying new things, referenced a ground rule or common goaland delegated decisions to partners or smaller workgroups. When viewpoints were not easily resolved, cases used different approaches to reach final decisions while maintaining relationships with partners, such as elevating decisions to leadership or agreeing to test out an approach. No one engagement structure (e.g., advisory group, coinvestigator) stood out as better able to manage different viewpoints. Teams adjusted engagement structures and behaviours to facilitate an overall culture of inclusion and respect. Partners acknowledged the intentional efforts of researchers to incorporate their perspectives, navigate challengesand communicate the value of partner input. By using collaborative decision-making in the early stages and throughout the study, researchers built trust with partners so that when decisions were difficult to resolve, partners still felt listened to and that their input mattered. Members of the PCORI Patient Engagement Advisory Panel in 2019-2020 provided input into the design of the study, including the research questions and approaches to data collection and analysis.

Ketamine versus ECT for Nonpsychotic Treatment-Resistant Major Depression

BackgroundElectroconvulsive therapy (ECT) and subanesthetic intravenous ketamine are both currently used for treatment-resistant major depression, but the comparative effectiveness of the two treatments remains uncertain.MethodsWe conducted an open-label, randomized, noninferiority trial involving patients referred to ECT clinics for treatment-resistant major depression. Patients with treatment-resistant major depression without psychosis were recruited and assigned in a 1:1 ratio to receive ketamine or ECT. During an initial 3-week treatment phase, patients received either ECT three times per week or ketamine (0.5 mg per kilogram of body weight over 40 minutes) twice per week. The primary outcome was a response to treatment (i.e., a decrease of ≥50% from baseline in the score on the 16-item Quick Inventory of Depressive Symptomatology–Self-Report; scores range from 0 to 27, with higher scores indicating greater depression). The noninferiority margin was −10 percentage points. Secondary outcomes included scores on memory tests and patient-reported quality of life. After the initial treatment phase, the patients who had a response were followed over a 6-month period.ResultsA total of 403 patients underwent randomization at five clinical sites; 200 patients were assigned to the ketamine group and 203 to the ECT group. After 38 patients had withdrawn before initiation of the assigned treatment, ketamine was administered to 195 patients and ECT to 170 patients. A total of 55.4% of the patients in the ketamine group and 41.2% of those in the ECT group had a response (difference, 14.2 percentage points; 95% confidence interval, 3.9 to 24.2; P<0.001 for the noninferiority of ketamine to ECT). ECT appeared to be associated with a decrease in memory recall after 3 weeks of treatment (mean [±SE] decrease in the T-score for delayed recall on the Hopkins Verbal Learning Test–Revised, −0.9±1.1 in the ketamine group vs. −9.7±1.2 in the ECT group; scores range from −300 to 200, with higher scores indicating better function) with gradual recovery during follow-up. Improvement in patient-reported quality-of-life was similar in the two trial groups. ECT was associated with musculoskeletal adverse effects, whereas ketamine was associated with dissociation.ConclusionsKetamine was noninferior to ECT as therapy for treatment-resistant major depression without psychosis. (Funded by the Patient-Centered Outcomes Research Institute; ELEKT-D ClinicalTrials.gov number, NCT03113968.)

Next Steps Toward Improving the Quality, Relevance, and Implementation of Chronic Limb-Threatening Ischemia Research Through Stakeholder Engagement

Stakeholder-engaged research (SER), in which patients and other stakeholders (including academics) engage as research, has been shown to improve the quality, feasibility, relevance, and implementation of research. There is scant SER in vascular surgery. Attitudes toward SER have not been assessed among academic vascular surgery stakeholders (clinicians and clinical researchers). Therefore, it is unclear how to increase SER to improve the care and outcomes of patients with vascular disease. As part of a Patient-Centered Outcomes Research Institute grant to increase stakeholder-centricity and sustainable engagement in chronic limb-threatening ischemia (CLTI) research, we developed an anonymous online survey based off qualitative interviews with 10 academic stakeholders. Survey responses are presented descriptively, grouped by prior SER experience. There were 42 responses to the survey for analysis. Of these, 19 had prior SER experience and 23 did not. A total of 89% of experienced respondents (ER) were moderately or extremely familiar with the term SER vs 35% of inexperienced respondents (IR). In general, ER and IR agreed about the benefits of SER, including improved relevance of research questions (80% ER, 79% IR) and selection of more relevant outcome measures (67% and 63%, respectively; Table I). However, ER more commonly reported that SER was the ethically/morally correct thing to do (60% vs 32%). There were noticeable differences in experienced/perceived drawbacks to SER, particularly in relation to the research design process (reported to be slower by 77% of ER vs 36% of IR) and the scientific interest of SER (reported to be less interesting by 8% of ER vs 29% of IR; Table I). Respondents agreed that lack of awareness was the largest barrier to increasing CLTI-related SER. However, IR identified some additional significant barriers (Table II). ER thought stakeholder training materials would be the most helpful resource to increase CLTI-related SER (73% vs 50%), whereas IR preferred assistance identifying and coordinating stakeholders (55% vs 71%; Table II). ER were more likely to feel that it was very or extremely important to partner with stakeholders in future research design/conduct (72% vs 22%). Researchers experienced with engaging stakeholders balanced a belief in its benefits with a realistic understanding of its associated difficulties. Some inexperienced researchers were strongly interested in future SER but highlighted several barriers to entry. Future efforts to increase SER in CLTI should focus on raising awareness of its benefits and the process, while lowering barriers to entry and assisting experienced researchers with well-defined pain points.Table IExperienced and perceived benefits and drawbacks to stakeholder-engaged research (SER)SER-experienced respondentsSER-inexperienced respondentsBenefits associated with SERN = 15N = 19 More relevant questions8079 More relevant outcomes6763 More ethical6032 Researcher-patient relationships5347 More relevant intervention5358 Follow-up feasibility4737 Recruitment and retention4047 Researcher satisfaction3316 Easier implementation2026 Easier funding1321 Easier publication75Drawbacks associated with SERN = 13N = 14 Slower design7736 Slower conduct4636 More expensive2336 Harder to get funding1521 Less interesting829 Harder to publish814 Less meaningful80 Less satisfying00Data are presented as percentage. Open table in a new tab Table IIBarriers and facilitators to stakeholder-engaged research (SER)SER-experienced respondentsSER-inexperienced respondentsBarriers to SER Awareness6967 Methodologic expertise3840 Interest3833 Access to stakeholders3153 Stakeholder knowledge3153 Stakeholder selection criteria2340 Stakeholder health status2327 Lack of dissemination opportunities150 Lack of regulatory mandate1520 Lack of funding820 Logistical difficulties820Potential facilitators for SER Stakeholder training materials7350 Stakeholder identification and coordination5571 Methodology collaborations4536 Funding aggregation2764 Methodologic training materials2736 Aggregation of regulatory guidance2714 Aggregation of dissemination opportunities914Data are presented as percentage. Open table in a new tab

Federal Data for Conducting Patient-centered Outcomes Research on Economic Outcomes.

Patients are increasingly interested in data on the economic burdens and impacts of health care choices; caregivers, employers, and payers are also interested in these costs. Although there have been various federal investments into patient-centered outcomes research (PCOR), an assessment of the coverage and gaps in federally funded data for PCOR economic evaluations has not been produced to date. To classify relevant categories of PCOR economic costs, to assess current federally funded data for coverage of these categories, and to identify gaps for future research and collection. A targeted internet search was conducted to identify a list of relevant outcomes and data sources. The study team assessed data sources for coverage of economic outcomes. A technical panel and key informant interviews were used for evaluation and feedback. Four types of formal health care sector costs, 3 types of informal health care sector costs, and 10 types of non-health care sector costs were identified as relevant for PCOR economic evaluations. Twenty-nine federally funded data sources were identified. Most contained elements on formal costs. Data on informal costs (eg, transportation) were less common, and non-health care sector costs (eg, productivity) were the least common. Most data sources were annual, cross-sectional, nationally representative individual-level surveys. The existing federal data infrastructure captures many areas of the economic burden of health and health care, but gaps remain. Research from multiple data sources and potential future integrations may offset gaps in individual data sources. Linkages are promising strategies for future research on patient-centered economic outcomes.

Predictive Modeling of Lapses in Care for People Living with HIV in Chicago: Algorithm Development and Interpretation.

Reducing care lapses for people living with HIV is critical to ending the HIV epidemic and beneficial for their health. Predictive modeling can identify clinical factors associated with HIV care lapses. Previous studies have identified these factors within a single clinic or using a national network of clinics, but public health strategies to improve retention in care in the United States often occur within a regional jurisdiction (eg, a city or county). We sought to build predictive models of HIV care lapses using a large, multisite, noncurated database of electronic health records (EHRs) in Chicago, Illinois. We used 2011-2019 data from the Chicago Area Patient-Centered Outcomes Research Network (CAPriCORN), a database including multiple health systems, covering the majority of 23,580 people with an HIV diagnosis living in Chicago. CAPriCORN uses a hash-based data deduplication method to follow people across multiple Chicago health care systems with different EHRs, providing a unique citywide view of retention in HIV care. From the database, we used diagnosis codes, medications, laboratory tests, demographics, and encounter information to build predictive models. Our primary outcome was lapses in HIV care, defined as having more than 12 months between subsequent HIV care encounters. We built logistic regression, random forest, elastic net logistic regression, and XGBoost models using all variables and compared their performance to a baseline logistic regression model containing only demographics and retention history. We included people living with HIV with at least 2 HIV care encounters in the database, yielding 16,930 people living with HIV with 191,492 encounters. All models outperformed the baseline logistic regression model, with the most improvement from the XGBoost model (area under the receiver operating characteristic curve 0.776, 95% CI 0.768-0.784 vs 0.674, 95% CI 0.664-0.683; P<.001). Top predictors included the history of care lapses, being seen by an infectious disease provider (vs a primary care provider), site of care, Hispanic ethnicity, and previous HIV laboratory testing. The random forest model (area under the receiver operating characteristic curve 0.751, 95% CI 0.742-0.759) revealed age, insurance type, and chronic comorbidities (eg, hypertension), as important variables in predicting a care lapse. We used a real-world approach to leverage the full scope of data available in modern EHRs to predict HIV care lapses. Our findings reinforce previously known factors, such as the history of prior care lapses, while also showing the importance of laboratory testing, chronic comorbidities, sociodemographic characteristics, and clinic-specific factors for predicting care lapses for people living with HIV in Chicago. We provide a framework for others to use data from multiple different health care systems within a single city to examine lapses in care using EHR data, which will aid in jurisdictional efforts to improve retention in HIV care.

On crowns, caravans and windmills: Commentary on an essay by Turcotte P., et al.

Patient-oriented research (POR) is a trend that has emerged over several decades and is particularly prominent in Canada, the United States and the United Kingdom. It involves patient and other stakeholder participation in the planning, conduct and dissemination of biomedical and health services research and it can be seen as a form of public participation and engagement in activities that affect the lives and well-being of communities. Criticisms of POR revolve around its susceptibility to tokenistic treatment of patient participants and paternalistic dominance of the research agenda by professional researchers, academics and clinicians. This commentary addresses one such critique by situating the POR agenda within the challenges and dilemmas faced by the health-related research enterprise over the past 30 years. It will explore the interface between POR, community activism and community-based participatory research. The contextual importance of the COVID-19 pandemic experience is stressed. The commentary will particularly focus on the US-based Patient Centred Outcomes Research Institute, its origins within a movement to enhance emphasis on publicly funded comparative effectiveness research, and its more recent evolution in the direction of community empowerment in POR.

Breast and colorectal cancer recurrence-free survival estimates in the US: Modeling versus active data collection

BackgroundA modeling method was developed to estimate recurrence-free survival using cancer registry survival data. This study aims to validate the modeled recurrence-free survival against “gold-standard” estimates from data collected by the National Program of Cancer Registries (NPCR) Patient-Centered Outcomes Research (PCOR) project. MethodsWe compared 5-year metastatic recurrence-free survival using modeling and empirical estimates from the PCOR project that collected disease-free status, tumor progression and recurrence for colorectal and female breast cancer cases diagnosed in 2011 in 5 U.S. state registries. To estimate empirical recurrence-free survival, we developed an algorithm that combined disease-free, recurrence, progression, and date information from NPCR-PCOR data. We applied the modeling method to relative survival for patients diagnosed with female breast and colorectal cancer in 2000–2015 in the SEER-18 areas. ResultsWhen grouping patients with stages I-III, the 5-year metastatic recurrence-free modeled and NPCR-PCOR estimates are very similar being respectively, 90.2 % and 88.6 % for female breast cancer, 74.6 % and 75.3 % for colon cancer, and 68.8 % and 68.5 % for rectum cancer. In general, the 5-year recurrence-free NPCR-PCOR and modeled estimates are still similar when controlling by stage. The modeled estimates, however, are not as accurate for recurrence-free survival in years 1–3 from diagnosis. ConclusionsThe alignment between NPCR-PCOR and modeled estimates supports their validity and provides robust population-based estimates of 5-year metastatic recurrence-free survival for female breast, colon, and rectum cancers. The modeling approach can in principle be extended to other cancer sites to provide provisional population-based estimates of 5-year recurrence free survival.

The Telemedicine Experience in Primary Care Practices in the United States: Insights From Practice Leaders.

The need to rapidly implement telemedicine in primary care during the coronavirus disease 2019 (COVID-19) pandemic was addressed differently by various practices. Using qualitative data from semistructured interviews with primary care practice leaders, we aimed to report commonly shared experiences and unique perspectives regarding telemedicine implementation and evolution/maturation since March 2020. We administered a semistructured, 25-minute, virtual interview with 25 primary care practice leaders from 2 health systems in 2 states (New York and Florida) included in PCORnet, the Patient-Centered Outcomes Research Institute clinical research network. Questions were guided by 3 frameworks (health information technology evaluation, access to care, and health information technology life cycle) and involved practice leaders' perspectives on the process of telemedicine implementation in their practice, with a specific focus on the process of maturation and facilitators/barriers. Two researchers conducted inductive coding of qualitative data open-ended questions to identify common themes. Transcripts were electronically generated by virtual platform software. Twenty-five interviews were administered for practice leaders representing 87 primary care practices in 2 states. We identified the following 4 major themes: (1) the ease of telemedicine adoption depended on both patients' and clinicians' prior experience using virtual health platforms, (2) regulation of telemedicine varied across states and differentially affected the rollout processes, (3) visit triage rules were unclear, and (4) there were positive and negative effects of telemedicine on clinicians and patients. Practice leaders identified several challenges to telemedicine implementation and highlighted 2 areas, including telemedicine visit triage guidelines and telemedicine-specific staffing and scheduling protocols, for improvement.

Add-On Aripiprazole May Benefit Older Adults With Refractory Depression

Add-On Aripiprazole May Benefit Older Adults With Refractory Depression

Lessons Learned from a National Initiative Promoting Publicly Available Standards-Based Clinical Decision Support.

Clinical decision support (CDS), which provides tools to assist clinical decision-making, can improve adherence to evidence-based practices, prevent medical errors, and support high-quality and patient-centered care delivery. Publicly available CDS that uses standards to express clinical logic (i.e., standards-based CDS) has the potential to reduce duplicative efforts of translating the same clinical evidence into CDS across multiple health care institutions. Yet development of such CDS is relatively new and its potential only partially explored. This study aimed to describe lessons learned from a national initiative promoting publicly available, standards-based CDS resources, discuss challenges, and report suggestions for improvement. Findings were drawn from an evaluation of the Agency for Healthcare Research and Quality Patient-Centered Outcomes Research CDS Initiative, which aimed to advance evidence into practice through standards-based and publicly available CDS. Methods included literature and program material reviews, key informant interviews, and a web-based survey about a public repository of CDS artifacts and tools for authoring standards-based CDS. The evaluation identified important lessons for developing and implementing standards-based CDS through publicly available repositories such as CDS Connect. Trust is a critical factor in uptake and can be bolstered through transparent information on underlying evidence, collaboration with experts, and feedback loops between users and developers to support continuous improvement. Additionally, while adoption of standards among electronic health record developers will make it easier to implement standards-based CDS, lower-resourced health systems will need extra support to ensure successful implementation and use. Finally, although we found the resources developed by the Initiative to offer valuable prototypes for the field, health systems desire more information about patient-centered, clinical, and cost-related outcomes to help them justify the investment required to implement standards-based, publicly available CDS. While the standards and technology to publicly share standards-based CDS have increased, broad dissemination and implementation remain challenging.

A History of Abuse is Associated with More Severe Migraine- and Pain-Related Disability: Results from the American Registry of Migraine Research (ARMR) (P11-12.009)

A History of Abuse is Associated with More Severe Migraine- and Pain-Related Disability: Results from the American Registry of Migraine Research (ARMR) (P11-12.009)

Simultaneous Comparisons of 25 Acute Migraine Medications: A Big Data Analysis of 10 Million Patient Self-Reported Treatment Records From A Migraine Smartphone Application (S41.001)

Simultaneous Comparisons of 25 Acute Migraine Medications: A Big Data Analysis of 10 Million Patient Self-Reported Treatment Records From A Migraine Smartphone Application (S41.001)

Psychometric Evaluation of the Activity Impairment in Migraine-Diary (AIM-D) Using Data From a Phase 3 (PROGRESS) Clinical Trial in Patients With Chronic Migraine (P8-12.008)

Psychometric Evaluation of the Activity Impairment in Migraine-Diary (AIM-D) Using Data From a Phase 3 (PROGRESS) Clinical Trial in Patients With Chronic Migraine (P8-12.008)

Characterizing Prodrome (Premonitory Phase) in Migraine: Results From the PRODROME Trial Screening Period (S41.009)

Characterizing Prodrome (Premonitory Phase) in Migraine: Results From the PRODROME Trial Screening Period (S41.009)