De Novo Experience of Individuals with Li-Fraumeni Syndrome: Sometimes the people in your family are never going to be the ones who understand best

Background/Objectives: Li-Fraumeni Syndrome (LFS) is a rare hereditary cancer predisposition syndrome characterized by high lifetime risks for multiple primary malignancies. Although most individuals with LFS inherit a pathogenic TP53 variant from a parent, approximately 20% have de novo variants with no suggestive family cancer history. This may result in an LFS experience distinct from individuals with affected relatives. This multi-case study report examines the unique psychosocial experiences of three young adults with de novo TP53 variants. Method(s): The National Cancer Institute's LFS study (NCT01443468) recruited adolescents and young adults (AYAs;aged 15-39 years) with LFS for qualitative interviews. Three participants had a de novo TP53 variant and a personal cancer history. An interprofessional team analyzed interview data using extended case study and narrative methods. Result(s): De novo participants lacked familiarity with LFS to situate a cancer diagnosis, interpret genetic test results, or adjust to chronic cancer risk. Communicating with and receiving support from family was challenged by their lack of common experience. De novo participants experienced socioemotional isolation, which was amplified during the COVID-19 pandemic. To cope, they sought support in online rare disease communities or through mental health providers. Conclusion(s): Individuals with de novo variants may lack familial guides and familiar providers to address disease management and uncertainty. Specialty health and mental health providers may support de novo patients across hereditary cancer syndromes by validating their uncertainties and connecting them with diseasespecific patient advocacy groups that support adjustment to chronic cancer risk.

How to read a paper about inequality in respiratory disease: Basic epidemiological principles and how inequalities are measured

Health inequalities in respiratory disease are widespread, and monitoring them is important for advocacy, the design and delivery of health services, and informing wider health policy. In this chapter, we introduce the different ways in which health inequalities can be quantified, including measures that quantify absolute and relative inequalities, and those that measure gaps between groups or differences across the entire social gradient. We consider the strengths and limitations of these different approaches and highlight things to look out for when reading a paper on health inequalities in respiratory health. These include how common the outcome is and whether other factors have been adjusted for, as both can have a crucial impact on interpretation and can lead to misleading conclusions.Copyright © ERS 2023.

Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases: Third of Three Sets of Expanded Proceedings from the 2020 ISCTM Autumn Conference on Pediatric Drug Development

This article expands on a session, titled "Patient Centricity: Design and Conduct of Clinical Trials in Orphan Diseases," that was presented as part of a two-day meeting on Pediatric Drug Development at the International Society for Central Nervous System (CNS) Clinical Trials and Methodology (ISCTM) Autumn Conference in Boston, Massachusetts, in October 2020. Speakers from various areas of pediatric drug development addressed a variety of implications of including children in drug development programs, including implications for rare/orphan diseases. The speakers have written summaries of their talks. The session's lead Chair was Dr. Joan Busner, who wrote introductory and closing comments. Dr. Simon Day, regulatory consultant, outlined some of the past mistakes that have plagued trials that did not consult with patient groups in the early design phase. Dr. Atul Mahableshwarkar provided an industry perspective of a recent trial that benefited from the inclusion of patient input. Drs. Lucas Kempf and Maria Sheean provided regulatory input from the perspectives of the United States (US) Food and Drug Administration (FDA) and European Medicines Agency (EMA), respectively. Dr. Judith Dunn outlined a novel approach for assessing and rank ordering patient and clinician clinical meaningfulness and the disconnect that may occur. Dr. Busner provided closing comments, tied together the presented issues, and provided a synopsis of the lively discussion that followed the session. In addition to the speakers above, the discussion included two representatives from patient advocacy groups, as well as an additional speaker who described the challenges of conducting a pediatric trial in the US and European Union (EU), given the often competing regulatory requirements. This article should serve as an expert-informed reference to those interested and involved in CNS drug development programs that are aimed at children and rare diseases and seek to ensure a patient-centric approach.Copyright © 2023, Matrix Medical Communications. All rights reserved.

Frontline Workers in the Backrooms of COVID-19.

OBJECTIVES: To review the response to the coronavirus disease 2019 (COVID-19) pandemic in a forensics center that integrates an academic department of pathology with multiple regional county medical examiners' offices. METHODS: Faculty and staff were asked to volunteer stories, data, and photographs describing their activities from March through May 2020. The information was assembled into a narrative summary. RESULTS: Increased deaths challenged capacity limits in a hospital morgue and a large urban medical examiner's office (MEO) successfully managed by forensic teams and monitored by an institutional command center. Autopsies of suspected and proven cases of COVID-19 were performed in both facilities. Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) testing of decedents was performed in a MEO serving a large urban area. Scene investigators worked directly with families to meet needs unique to a pandemic. Artful photographs of decedent's hands and/or tattoos were offered to those unable to have in-person viewings. Pathologists and social workers were available to families of the deceased and created novel solutions to facilitate the grieving process. CONCLUSIONS: Forensic pathology is important to successfully navigating emerging diseases like the COVID-19 pandemic. Direct conversations with families are common in forensic pathology and serve as a model for patient- and family-centered care.

SAFER framework for moving forward on the medical device right to repair

Across various industries, the right to repair (RTR) movement has gained momentum as more than 20 states have proposed RTR laws to expand access to repair of consumer products. Medical device equipment shortages during the COVID-19 pandemic demonstrated that stronger repair mechanisms are necessary for the US health system to become more efficient, affordable and sustainable. We propose a 5-point SAFER framework including safety and security, adaptability, fiscal, environmental and regulatory factors for consideration in implementing medical device RTR. The healthcare community can help advance RTR legislation in a manner that serves our patients and healthcare system best.Copyright © 2023 BMJ Publishing Group. All rights reserved.

Dutch Myositis Working Group (Dmwg)

Introduction. The poster of the Dutch Myositis Working Group (DMWG) aims to inform people about her goals, activities and ambitions. The group is run by seven patients, representing all types of myositis, supported by Spierziekten Nederland, the umbrella patient organization for neuromuscular disorders in The Netherlands and 4 myositis specialists as medical advisors. Chair: Ingrid de Groot. Contact email: myositis@spierziekten.nl Goals and ambitions of the Dutch myositis working group: * I n collaboration with medical advisors to provide information about IIM (idiopathic inflammatory myopathies) or myositis to newly diagnosed patients and their families: IIM types, symptoms, diagnosis, (new) treatment options, prognosis, inform them about the myositis expertise centres etc. * To connect and support people with all types of IIM: dermatomyositis (DM), polymyositis (PM), Anti Synthetase Syndrome (ASyS), immune mediated necrotizing myopathy (IMNM), juvenile dermatomyositis (JDM), overlap myositis. * To raise awareness of myositis among the public, health care professionals and researchers, pharmaceutical companies? * To collaborate with clinicians, researchers and funds on a national and international level with the aim to improve (clinical) care and research. * To stimulate and participate in the development and conducting of clinical trials. * To collaborate with myositis working groups and patient organisations abroad. * To represent the patient perspective within in the Myositis Network Netherlands and (inter)national myositis study groups. * Patient advocacy. Activities and services: * In person or online meetings aiming to offer moral support and an opportunity to share experiences, concerns etc. or just to socialize. Three times a year we organize separate meetings for people with IBM, for people with other IIM and for caregivers. * Website updates on treatment, guidelines, (inter)national research, activities and actualities (e.g. Covid situation). * Supply patients with brochures for GP/ family doctor, physiotherapist etc. * Online (secured) platform for members. * Annual patient conference with diagnosis specific scientific programs. * Monthly newsletters: these are personalized which means they contain mainly news on the receivers type of IIM (e.g. IBM or ASyS) and information on general topics concerning all people with IIM or neuromuscular disorder. * In person meetings and / or online webinars on general topics e.g. living with a chronic condition, work, pain, fatigue. * Annual meetings with medical advisors: the working group pays a visit to all medical advisors in their respective hospitals. * Representation at (inter)national conferences. * Representation in projects such as guidelines development. * Collaboration in (inter)national studies leading to enrolling Dutch patients, researchers and clinicians in multi-centre studies, (co-) authorships in publications and to presentations during conferences (Treat NMD, IMACS, MNN). * To advise and recommend on research proposals from patient perspective. * To advise decision makers on continuation of expert centres from patient perspective. Collaborations: * Myositis Network Netherlands: patient representation on the board. * OMERACT (Outcome Measures in Rheumatology): Patient Research Partner of the Myositis Working Group. * IMACS (International Myositis Assessment and Clinical Studies Group): steering committee member of Exercise & Rehabilitation Group, led by Helene Alexanderson, ass.prof PhD, RPT). * ENMC (European Neuromuscular Centre): patient representation in myositis workshops. * EULAR (European League against Rheumatism): member of PARE and Patient Research Partner. * GCOM. * ERN - NMD (European Reference Network for Neuromuscular Diseases): member of NMD working group led by em. prof. dr. Marianne de Visser. * Patient organizations for people living with myositis . We are in this together Since myositis is a (very) rare disease, the 'myositis community' is a small one although we're happy to say that it is expanding quite rapidly. Through our inte sive involvement in several national and international studies and research projects we now have close contacts with many myositis experts across the globe, which makes it easier to keep up with actualities and developments concerning research, treatment etc. and to disseminate this knowledge to our members. This helps us to inform, support and advocate for the Dutch people living with myositis and their families and at the same time it offers opportunities to give something back: by sharing with the research community and clinicians our experiential knowledge of the consequences of myositis on everyday life. That way we can contribute to more meaningful research. We can only go forward if we do this together! That is why we are very ambitious in our efforts to contribute to myositis research. Here we list our collaborative efforts: * In 2019 the Myositis Network Netherlands of clinicians and researchers with expertise in IIM was established in which the DMWG is representing the patient perspective by a member on the board. * In OMERACT Myositis Working Group a member of the DMWG is one of the two Patient Research Partners and as such an equal partner of this study aiming to define a set of core patient reported domains with regard to the quality of life and respective instruments for use in IIM. The involvement of the DMWG has led to the opportunity for Dutch patients to participate in Delphi surveys and to an opportunity for Dutch myositis clinics to collaborate in the longitudinal study that emerged from this. * The IMACS network is an important part of our international network. One of our DMWG members is member of the Executive Committee of the Exercise & Rehabilitation Group and as such can facilitate for Dutch patients to become involved in the current study with the ultimate objective to develop recommendations for exercise in all types of IIM. * Members of the DMWG participated in several ENMC workshops on IIM as patient representatives and will continue to do so in the future. * Through a PARE membership in EULAR and membership of the study group of 'collaborative research' the DMWG hopes to raise awareness of myositis within the influential EULAR community and to speak up on behalf of the patients in Europe living with IIM. * One of our members is member of the GCOM committee responsible for the patient program of GCOM and shares the ambitions of this GCOM committee to increase the involvement of patients in this very important IIM conference. * One DMWG member joined the ERN- Neuromuscular Disease group and as such represents the people with IIM living throughout Europe. * DMWG has ambitions to empower people living with IIM and to connect with them, crossing borders by doing so. We have close and amicable relationships with patient organisations in Australia, Czech Republic, Germany, Sweden, UK and USA. * Empowering patients is one of our goals and we accomplished this for instance in Sweden. On invitation by prof. dr. Ingrid Lundberg our chair visited the Karolinska Institute, spent a week with their myositis team and in return was one of the speakers on the annual patient meeting and helped the Swedish patients establish their own myositis working group.

A coordinated health policy in response to COVID-19: A case of Botswana.

INTRODUCTION: This paper documents policy decisions and transformations in response to the COVID-19 pandemic in Botswana and utilizes the multi-sectoral approach (MSA) in providing this analysis. METHOD: A desk review of the different government gazette documents was conducted to trace health policy evolutionary developments and their impact on the general lives of the people of Botswana. FINDINGS: Revealed the actors, roles in this policy transformation and the conditions that enhanced the smooth implementation of the policies are discussed. CONCLUSION: The paper concludes by making some recommendations for the country's preparedness and anticipatory guidance for any other pandemic or disaster that may arise. CLINICAL RELEVANCE: This paper highlights the importance of the multisectoral approach in addressing crises such as pandemics. It also demonstrates the need for countries to have well-defined guidelines to allow decision making in the delivery of efficient health services to the general population during pandemics.

Long COVID Frameworks: Examining Individual- and Population-Level Models to Assess and Improve Patient Care.

Post-COVID conditions, commonly referred to as long COVID, has become of increasing interest to biomedical and public health communities. Patient advocacy is critical to not only advancing quality care outcomes for patients affected by prolonged effects of the disease, but also to contribute to our understanding of lived perspectives to ensure individual voices inform future care approaches. While there has not been a "one-size fits all" approach to standardized care, disease heterogeneity has challenged previous views that the virus is limited to single organ system infections. Stakeholders at all levels have the most impact when they collaborate and organize strategies to offer assessment and treatment in multidisciplinary settings. I propose a framework in which population- and individual-level models are integrated to enable quality outcomes while helping frontline practitioners improve their decision-making in all environments.

Current Changes in Europe Impacting Orphan Drugs Access: A Focus on the Latest Reforms in the EU4 and England

Objectives: Post-COVID, healthcare systems are balancing the need to improve health outcomes with expenditure. Healthcare and payer policy reforms have been approved or are being discussed in major European markets: France, Germany, Italy, Spain and England. This study outlines the evolving access landscape and potential implications on orphan drug (OD) access. Method(s): For each market, qualitative and quantitative analyses were performed on information collected via targeted literature review of publicly available sources: national authority websites, peer-reviewed articles, and grey literature. Findings were segmented into policy scope, policy occurrence likelihood and OD access impact. Policy scope comprised 4 categories: access, pricing, assessing methodology and wider healthcare policy. Occurrence likelihood was subdivided into occurred, expected, and discussed reforms. The OD access impact (positive/negative) was categorised as high, intermediate, and low. Analysing the relationship between likelihood of occurrence and impact to OD access pinpointed reforms most likely to change the OD payer landscape. Result(s): This research identified 29 reforms: 8 in England, 7 in Italy, 6 in France, 4 in Spain and Germany. Our analysis framework identified highly significant reforms: 3 in France and Germany, 2 in Italy, and 1 in England and Spain. England and Italy have the highest number of reforms encouraging OD launch. France showed a relative increase in attractiveness, while Spain lagged. Draft legislation in Germany appeared the most punitive for OD access to patients. Conclusion(s): Overall, attractiveness for OD launch is increasing in Europe. Due to the size of market potential, cost containment measures in Germany may hinder European launch strategy and mitigate the positive impact of increased OD patient access in other markets. Monitoring of upcoming changes alongside working closely with physician and patient advocacy groups to maintain OD funding priorities will ensure rare disease patients are not left behind and can continue to access therapies. Copyright © 2022

Impact of the initial wave of COVID-19 pandemic in Taiwan on local HIV services: Results from a cross-sectional online survey.

PURPOSE: This study aimed to investigate the impact of the initial wave of the COVID-19 pandemic on HIV services in Taiwan. METHODS: An online, cross-sectional survey was conducted among people living with HIV (PLWH), individuals at risk of HIV infection (IAR), and service prescribers between 20th October and 30th November, 2020. Representatives from patient advocacy groups were interviewed. RESULTS: In total, 66 PLWH, 104 IAR, and 32 prescribers from Taiwan completed the survey. Mild to moderate disruptions to HIV-related services (including medical consultation, HIV-related testing, and medications) were found by the survey, with IAR appearing more affected than PLWH. Nine (13.6%) PLWH and 31 (29.8%) IAR reported disruptions in hospital/clinic visits and two (3.0%) PLWH and 25 (24.0%) IAR reported decreased frequency of HIV testing. Similar observations were also made by four patient advocacy group representatives interviewed. Telehealth services were received by only limited proportions of PLWH and IAR who participated in the survey. CONCLUSION: HIV services in Taiwan were not severely affected by the initial wave of COVID-19, but notable disruptions were still observed in HIV screening and prevention services. Multi-pronged strategies, including telehealth services, are warranted to overcome new challenges in HIV care in the COVID-19 era.

A Renewed Charter: Key Principles to Improve Patient Care in Severe Asthma.

Asthma is a heterogenous respiratory disease, usually associated with chronic airway inflammation and hyper-responsiveness, which affects an estimated 339 million people worldwide. Severe asthma affects approximately 5-10% of patients with asthma, approximately 17-34 million people globally, more than half of whom have uncontrolled disease. Severe asthma carries a substantial burden of disease, including unpredictable symptoms and potentially life-threatening flare-ups. Furthermore, severe asthma has a substantial burden on health care systems and economies worldwide. In 2018, a group of experts from the clinical community, patient support groups, and professional organisations joined together to develop the Severe Asthma Patient Charter, which set out six principles to define what patients should expect for the management of their severe asthma and what should constitute a basic standard of care. Since the publication of that original Charter in 2018, several important changes have occurred, including an improved understanding of asthma and effective asthma management; several new therapies have become available; and finally, the COVID-19 pandemic has placed a spotlight on respiratory conditions, the workforces that treat them, and the fundamental importance of health care system resilience. With those developments in mind, we, representatives of the academic, clinical, and patient advocacy group communities, have updated the Charter to Improve Patient Care in Severe Asthma with a focus on six principles: (1) I deserve a timely, comprehensive assessment of my asthma and its severity; (2) I deserve a timely, straightforward referral to an appropriate specialist for my asthma when it is not well controlled; (3) I deserve to understand what makes my asthma worse; (4) I deserve access to treatment and care that reduces the impact of asthma on my daily life; (5) I deserve not to be reliant on systemic corticosteroids; (6) I deserve to be involved in decisions about my treatment and care.

ILLUMINATING THE PATIENT PERSPECTIVE: WHAT HEALTH CARE PROFESSIONALS SHOULD KNOW ABOUT LIVING WITH NONTUBERCULOUS MYCOBACTERIAL LUNG DISEASE

SESSION TITLE: Outcomes in Pneumonia and NTM SESSION TYPE: Rapid Fire Original Inv PRESENTED ON: 10/17/2022 12:15 pm - 1:15 pm PURPOSE: The purpose of this survey was to summarize demographic and clinical characteristics and identify patient perspectives in a cohort of people living with nontuberculous mycobacterial (NTM) lung disease to get a better understanding and perspectives of the patient population living with this disease. METHODS: In partnership with the patient advocacy group NTM Info & Research, we created and disseminated a survey to adult patients living with NTM lung disease. Survey participation was voluntary, and no incentives were offered. The survey focused on key clinical characteristics related to NTM lung disease and qualification and ranking of symptoms. Respondents were asked which HCP(s) they accessed for their NTM lung disease care, how their care could be improved, and to what degree the COVID-19 pandemic has affected their care. RESULTS: Respondents’ (N=90) mean age was 61.5 years, and 91.6% identified as female. Two thirds resided in North America. Most 86.4% identified mycobacterium avium complex (MAC) as the strain with which they were infected. Coughing (81.7%) and fatigue (80%) were most frequently reported. Fatigue was ranked as the most bothersome symptom, followed closely by coughing. These data are consistent with previous studies and patient reports, FDA-led Patient-Focused Drug Development (PFDD) meeting data, and a survey conducted by NTM Info & Research in 2019. Nearly all respondents (96.5%) reported accessing a specialty provider for their NTM lung disease. Close to one third (31.6%) accessed care from a respiratory specialist. Approximately half (53.6%) reported that the COVID-19 pandemic did not significantly affect their NTM care. Respondents reported on aspects of their NTM lung disease care that they would like to see improved. Common themes included earlier diagnosis, sooner evaluation by a specialist, shorter treatment courses, and more education about the disease and its treatments. CONCLUSIONS: Fatigue and coughing are both the most reported and the most bothersome symptoms reported by patients with NTM lung disease. Respondents would like to have been diagnosed earlier, have access to more efficacious and shorter courses of treatments, and be provided more information about their disease and the therapies used to treat it. Respondents would also like HCPs to be better educated on these same topics. These data illuminate the patient perspective so that HCPs who treat patients with NTM lung disease may be better informed of patients’ experiences. CLINICAL IMPLICATIONS: Improved understanding of demographics, risk factors, and clinical manifestations of those with NTM lung disease assist in population surveillance and timelier diagnosis of the disease. A better understanding of patients’ concerns, fears, and hopes is crucial for effective shared decision making and creation of individualized care plans. The ultimate goal is to improve outcomes for patients with NTM lung disease. DISCLOSURES: Stock owner relationship with Abbott Laboratories Please note: 10/2018-10/2020 Added 03/16/2022 by Sarah Anderson, value=Stock owner Stock owner relationship with Johnson & Johnson Please note: 10/2018-10/2020 Added 03/16/2022 by Sarah Anderson, value=Stock owner Stock owner relationship with Merck Please note: 10/2018-10/2020 Added 03/16/2022 by Sarah Anderson, value=Stock owner Advisory Committee Member relationship with MannKind Corporation Please note: Feb 2022-Present Added 04/18/2022 by Amy Leitman, value=Travel

EXPLORING the IMPACT of the PANDEMIC on ITALIAN RHEUMATIC DISEASE PATIENTS: A PATIENT ADVOCACY SURVEY

Background: Patients with rheumatic diseases show impaired quality of life (QoL): disease activity, comorbidities and treatment-related side effects contributes to decrease physical, emotional, and social functioning. The COVID-19 pandemic challenged health care systems and patients with chronic conditions: hospital and outpatient clinics delayed access, unpredictable changes like non-adherence to medication, but also negative emotions, psychological conditions recognized as risk factors for the poor QoL [1-2]. Objectives: To describe the impact of the COVID-19 pandemic on the Qol of Italian patients with rheumatic diseases in the frst period of the national lockdown. Methods: APMARR (Associazione Nazionale Persone con Malattie Reu-matolgoche e Rare) invited Italian patients and caregivers to participate to an online structured survey in the period March-June 2020. Informed consent was retrieved. The questionnaire, promoted by the patient advocacy website, collected demographics, emotional and healthcare pattern information. Results: 87,44% (N=215) complete questionnaires were retrieved (96.3% patients;3.7% caregivers;87.77% female;2% aged <18 years, 16% aged 18-30 years, 82% aged >31 years;0.53% missing data). Respondents were illustrative of the Patient Advocacy regional distribution (Puglia Region predominant). Rheumatoid arthritis regarded 29% of respondents, ankylosing spondylitis 17%, psoriasis 16%, remaining 38% other rheumatoid diseases. 96% of respondents were employed, 39% of which had to discontinue/change working activity during pandemics. 60% of respondents reported being worried about their disease. The deriving sense of fragility was the main cause of anxiety, which was not controlled even by compliance to the treatment plan (88%). 30% of respondents was worried about virus infection. Irritability, appetite and sleep disorders were also reported: anxiety had effects on irritability (46% sometimes more irritable) and sleep quality (38% always disturbed). GPs visits access was limited (40% considered it absent and 76% had to postpone it). Only 32% of specialist centers provided facilitated patterns of care. Respondents suggested possible solutions to improve QoL during COVID-19 pandemic and over it. Psychologist support was suggested as useful by 44% of respondents to manage therapies and by 56% to effort daily life. The home infusion would be of support according to 44% of respondents (18% already got access to it). Patient Advocacy had a main role in the new care and life context: 80% would consider it useful to participate to and 81% was satisfed by the prompt and continuous support received during pandemics. Conclusion: Similar results as far as distress were reported by Italian and foreign evidence, which demonstrated considerable COVID-19 related psychosocial burden in rheumatic disease patients [3-5]. Potential solutions are also with EULAR recent guidelines, which underlined how psychological interventions were found to reduce pain and fatigue even in difficult-to-treat patients [6] and how mental health needs should be periodically assessed, due to the link between better emotional well-being and better self-management [7]. Besides, the reported picture of reorganised care during pandemic corresponded to the real-world experience of the Italian Regions [8]. New approaches of care like home infusions and telemedicine supported by patient organisations should become routinary and may therefore beneft patients.

ENABLING REMOTE RESEARCH THROUGH DECENTRALIZED RANDOMIZED CONTROLLED CLINICAL TRIALS

BACKGROUND: Traditionally, randomized clinical trials have relied on physical research centers to support subject recruitment and participation. The COVID-19 pandemic has highlighted the need to interact with subjects who are unable to physically visit research centers. By leveraging remote technology, such clinical trials may reach subjects in isolation and broaden geographical reach. We describe a fully remote, multisite randomized controlled clinical trial of outpatient COVID-19 treatments using a technologyenabled, decentralized approach. METHODS: We conducted a remote double-blind, randomized placebocontrolled trial (COVID-OUT). We identified subjects through medical records, patient advocacy groups, testing facilities, and multiformat advertising. They were recruited via brochure, electronic message, telephone outreach, and self-referral. Research staff across sites used the Research Electronic Data Capture (REDCap) system to manage local and central enrollment and were reallocated dynamically based on trial needs. Subjects were screened by phone, consented and randomized electronically and delivered study medication by courier or same-day mail. They were followed via their preferred communication method (phone, video, text, or email) to determine the study endpoints. RESULTS: 1195 non-hospitalized adults aged 30-85 years with laboratory confirmed infection with SARS-CoV-2 were enrolled into the COVID-OUT trial through January 6, 2022 over a span of 7 months. Initially starting as a 2- arm trial with 7 sites, the study expanded to a 6-arm trial with recruitment at 8 sites. To date, 9600 subjects have been screened with an enrollment rate of 12% from 822 zip codes. 25 research coordinators are involved across 8 sites, and the rate of study completion is 90%. CONCLUSIONS: Decentralized remote studies offer an efficient, low-touch way of performing research in the COVID-19 era. Our decentralized study design enables research with infectious, isolated subjects in widespread geographies, while maintaining safety of subjects, the research team, and public atlarge. Coordination across sites via RED Cap enabled programmatic efficiencies, including the ability to redistribute staffing support across enrollment sites for study drug distribution, follow-up calls, recruitment, and event reporting. We decreased overall costs by less need for physical research space. The decentralized infrastructure enabled nimble adaptations of the protocol, including increasing follow-up periods to assess long-COVID symptoms and adding study arms for additional outpatient treatments. Given the widespread availability of mobile phones and remote communication, decentralized trials show promise for improving reach and efficiency in both pandemic and nonpandemic times.

Predicting Vaccine Confidence: Patient Self-advocacy and Fear of COVID-19 in the United States

This study investigated the extent to which patient self-advocacy predicted COVID-19 vaccine hesitance in the U.S. (n = 1987). Results indicated being vaccinated, increasing age, and higher education were each positively related to increased vaccine non-hesitance. Also, those who sought more information and who were more assertive about their health had more non-hesitance toward the vaccine. Higher vaccine hesitance was found among participants who were Black and from other ethnic minorities, those who voted for Donald Trump or who did not vote in the 2020 election, and those who were more prone to nonadherence to health provider instructions. Targeted communication campaigns to connect to people with these specific characteristics and levels of advocacy can decrease vaccine non-hesitance. [ FROM AUTHOR] Copyright of Southern Communication Journal is the property of Southern States Communication Association and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full . (Copyright applies to all s.)

Interventions, Participative Role, Barriers, and Facilitators for Involvement in eHealth Communication for People Undergoing Hemodialysis: Protocol for a Scoping Review.

BACKGROUND: eHealth interventions have been shown to offer people living with chronic kidney disease the opportunity of embracing dialysis therapies with greater confidence, the potential to obtain better clinical outcomes and increased quality of life, and diverse and flexible designs and delivery options. eHealth interventions or solutions can offer one-way information without the possibility for dialogue, as with most mobile apps. eHealth interventions intending to enable two-way communication between patients undergoing hemodialysis and health professionals are the focus of this review. eHealth communication interventions that enable two-way communication between patients undergoing hemodialysis and health professionals is an emerging field, but issues relating to participation in eHealth communication for patients undergoing hemodialysis are scarcely described. The current conceptualization of this issue is too scattered to inform the development of future interventions. In this scoping review, we want to assemble and examine this scattered knowledge on participation in two-way eHealth communication for patients undergoing hemodialysis. OBJECTIVE: We want to understand the participative role of people living with chronic kidney disease undergoing hemodialysis in available communicative eHealth interventions and to understand which barriers and facilitators exist for patient involvement in eHealth communication with health professionals. METHODS: A scoping review methodology is guiding this study. Peer-reviewed primary studies, including quantitative, qualitative, and mixed methods study designs will be included. A systematic search for published studies, dissertations, and theses at the doctoral level in the English language will be conducted in five databases (MEDLINE, Embase, CINAHL, Scopus, and ProQuest Dissertations and Theses). The included literature will focus on adult (18 years or older) patients undergoing hemodialysis who are involved in eHealth communication with health professionals. Data on the type of eHealth communication interventions, the participative role, and barriers and facilitators for the involvement in eHealth communication for people undergoing hemodialysis will be extracted independently by two reviewers. The extracted data will be collected in a draft charting table prepared for the study. Any discrepancies between the reviewers will be solved through discussion or with a third reviewer. RESULTS: Results are anticipated by the spring of 2023 and will be presented in tabular format along with a narrative summary. The anticipated results will be presented in alignment with the objectives of the study, presenting findings on the participative role of patients undergoing hemodialysis in eHealth communication interventions. CONCLUSIONS: We anticipate that this study will inform on eHealth communication interventions and the level of patient participation in eHealth communication for patients undergoing hemodialysis. The systematized overview will possibly identify research gaps and motivate further development of eHealth communication to ensure patient participation. The findings will be of interest to key stakeholders in clinical care, research, development, policy, and patient advocacy. INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): PRR1-10.2196/38615.