Lessons learned about harmonizing survey measures for the CSER consortium

AbstractIntroduction:Implementation of genome-scale sequencing in clinical care has significant challenges: the technology is highly dimensional with many kinds of potential results, results interpretation and delivery require expertise and coordination across multiple medical specialties, clinical utility may be uncertain, and there may be broader familial or societal implications beyond the individual participant. Transdisciplinary consortia and collaborative team science are well poised to address these challenges. However, understanding the complex web of organizational, institutional, physical, environmental, technologic, and other political and societal factors that influence the effectiveness of consortia is understudied. We describe our experience working in the Clinical Sequencing Evidence-Generating Research (CSER) consortium, a multi-institutional translational genomics consortium.Methods:A key aspect of the CSER consortium was the juxtaposition of site-specific measures with the need to identify consensus measures related to clinical utility and to create a core set of harmonized measures. During this harmonization process, we sought to minimize participant burden, accommodate project-specific choices, and use validated measures that allow data sharing.Results:Identifying platforms to ensure swift communication between teams and management of materials and data were essential to our harmonization efforts. Funding agencies can help consortia by clarifying key study design elements across projects during the proposal preparation phase and by providing a framework for data sharing data across participating projects.Conclusions:In summary, time and resources must be devoted to developing and implementing collaborative practices as preparatory work at the beginning of project timelines to improve the effectiveness of research consortia.

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Biomarker-Driven Oncology Clinical Trials: Key Design Elements, Types, Features, and Practical Considerations

JCO Precision Oncology ◽

10.1200/po.19.00086 ◽

2019 ◽

pp. 1-12

Author(s):

Chen Hu ◽

James J. Dignam

Keyword(s):

Clinical Trials ◽

Adaptive Design ◽

Lessons Learned ◽

Precision Oncology ◽

Design Elements ◽

Individual Patient Level ◽

Oncology Clinical Trials ◽

Multi Agent ◽

The Individual ◽

Design Options

In this precision oncology era, where molecular profiling at the individual patient level becomes increasingly accessible and affordable, more and more clinical trials are now driven by biomarkers, with an overarching objective to optimize and personalize disease management. As compared with the conventional clinical development paradigms, where the key is to evaluate treatment effects in histology-defined populations, the choices of biomarker-driven clinical trial designs and analysis plans require additional considerations that are heavily dependent on the nature of biomarkers (eg, prognostic or predictive, integral or integrated) and the credential of biomarkers’ performance and clinical utility. Most recently, another major paradigm change in biomarker-driven trials is to conduct multi-agent and/or multihistology master protocols or platform trials. These trials, although they may enjoy substantial infrastructure and logistical advantages, also face unique operational and conduct challenges. Here we provide a concise overview of design options for both the setting of single-biomarker/single-disease and the setting of multiple-biomarker/multiple-disease types. We focus on explaining the trial design and practical considerations and rationale of when to use which designs, as well as how to incorporate various adaptive design components to provide additional flexibility, enhance logistical efficiency, and optimize resource allocation. Lessons learned from real trials are also presented for illustration.

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Timely access to trial data in the context of a pandemic: the time is now

BMJ Open ◽

10.1136/bmjopen-2020-039326 ◽

2020 ◽

Vol 10 (10) ◽

pp. e039326

Author(s):

Rebecca Li ◽

Julie Wood ◽

Amrutha Baskaran ◽

Stanley Neumann ◽

Elizabeth Graham ◽

...

Keyword(s):

Data Sharing ◽

Scientific Progress ◽

Clinical Trial Data ◽

Trial Data ◽

Lessons Learned ◽

Trial Participant ◽

Non Profit ◽

Timely Access ◽

Individual Participant ◽

Data Tables

ObjectiveClinical trial data sharing has the potential to accelerate scientific progress, answer new lines of scientific inquiry, support reproducibility and prevent redundancy. Vivli, a non-profit organisation, operates a global platform for sharing of individual participant-level trial data and associated documents. Sharing of these data collected from each trial participant enables combining of these data to drive new scientific insights or assess reproducibility—not possible with the aggregate or summary data tables historically made available. We report on our initial experience including key metrics, lessons learned and how we see our role in the data sharing ecosystem. We also describe how Vivli is addressing the needs of the COVID-19 challenge through a new dedicated portal that provides a direct search function for COVID-19 studies, availability for fast-tracked request review and data sharing.Data summaryThe Vivli platform was established in 2018 and has partnered with 28 diverse members from industry, academic institutions, government platforms and non-profit foundations. Currently, 5400 trials representing 3.6 million participants are shared on the platform. From July 2018 to September 2020, Vivli received 201 requests. To date, 106 of 201 requests received approval, 5 have been declined, 27 withdrew and 27 are in the revision stage.ConclusionsThe pandemic has only magnified the necessity for data sharing. If most data are shared and in a manner that allows interoperability, then we have hope of moving towards a cohesive scientific understanding more quickly not only for COVID-19 but also for all diseases. Conversely, if only isolated pockets of data are shared then society loses the opportunity to close vital gaps in our understanding of this rapidly evolving epidemic. This current challenge serves to highlight the value of data sharing platforms—critical enablers that help researchers build on prior knowledge.

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Classification of processes involved in sharing individual participant data from clinical trials

F1000Research ◽

10.12688/f1000research.13789.2 ◽

2018 ◽

Vol 7 ◽

pp. 138 ◽

Cited By ~ 4

Author(s):

Christian Ohmann ◽

Steve Canham ◽

Rita Banzi ◽

Wolfgang Kuchinke ◽

Serena Battaglia

Keyword(s):

Clinical Trials ◽

Data Sharing ◽

Cultural Change ◽

Process Model ◽

Group Processes ◽

Major Type ◽

Individual Participant Data ◽

Consensus Document ◽

Individual Participant ◽

The Individual

Background: In recent years, a cultural change in the handling of research data has resulted in the promotion of a culture of openness and an increased sharing of data. In the area of clinical trials, sharing of individual participant data involves a complex set of processes and the interaction of many actors and actions. Individual services and tools to support data sharing are becoming available, but what is missing is a detailed, structured and comprehensive list of processes and subprocesses involved and the tools and services needed. Methods: Principles and recommendations from a published consensus document on data sharing were analysed in detail by a small expert group. Processes and subprocesses involved in data sharing were identified and linked to actors and possible supporting services and tools. Definitions adapted from the business process model and notation (BPMN) were applied in the analysis. Results: A detailed and comprehensive tabulation of individual processes and subprocesses involved in data sharing, structured according to 9 main processes, is provided. Possible tools and services to support these processes are identified and grouped according to the major type of support. Conclusions: The identification of the individual processes and subprocesses and supporting tools and services, is a first step towards development of a generic framework or architecture for the sharing of data from clinical trials. Such a framework is needed to provide an overview of how the various actors, research processes and services could interact to form a sustainable system for data sharing.

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Quality of Life for Children with Persistent Sinonasal Symptoms

Otolaryngology ◽

10.1067/mhn.2003.41 ◽

2003 ◽

Vol 128 (1) ◽

pp. 17-26 ◽

Cited By ~ 97

Author(s):

David J. Kay ◽

Richard M. Rosenfeld

Keyword(s):

Quality Of Life ◽

Clinical Care ◽

Longitudinal Change ◽

Good Test ◽

Routine Clinical Care ◽

Before And After ◽

The Mean ◽

The Individual ◽

Sinonasal Symptoms

OBJECTIVE: The goal was to validate the SN-5 survey as a measure of longitudinal change in health-related quality of life (HRQoL) for children with persistent sinonasal symptoms. DESIGN AND SETTING: We conducted a before and after study of 85 children aged 2 to 12 years in a metropolitan pediatric otolaryngology practice. Caregivers completed the SN-5 survey at entry and at least 4 weeks later. The survey included 5 symptom-cluster items covering the domains of sinus infection, nasal obstruction, allergy symptoms, emotional distress, and activity limitations. RESULTS: Good test-retest reliability ( R = 0.70) was obtained for the overall SN-5 score and the individual survey items ( R ≥ 0.58). The mean baseline SN-5 score was 3.8 (SD, 1.0) of a maximum of 7.0, with higher scores indicating poorer HRQoL. All SN-5 items had adequate correlation ( R ≥ 0.36) with external constructs. The mean change in SN-5 score after routine clinical care was 0.88 (SD, 1.19) with an effect size of 0.74 indicating good responsiveness to longitudinal change. The change scores correlated appropriately with changes in related external constructs ( R ≥ 0.42). CONCLUSIONS: The SN-5 is a valid, reliable, and responsive measure of HRQoL for children with persistent sinonasal symptoms, suitable for use in outcomes studies and routine clinical care.

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Neurodevelopmental Trajectories and Psychiatric Morbidity: Lessons Learned From the 22q11.2 Deletion Syndrome

Current Psychiatry Reports ◽

10.1007/s11920-021-01225-z ◽

2021 ◽

Vol 23 (3) ◽

Author(s):

Ania M. Fiksinski ◽

Maude Schneider ◽

Janneke Zinkstok ◽

Danielle Baribeau ◽

Samuel J. R. A. Chawner ◽

...

Keyword(s):

Genetic Variants ◽

Clinical Care ◽

Psychiatric Morbidity ◽

Phenotypic Expression ◽

22Q11.2 Deletion Syndrome ◽

Autism Spectrum ◽

Lessons Learned ◽

Deletion Syndrome ◽

22Q11.2 Deletion ◽

Variable Expression

AbstractPurpose of ReviewThe 22q11.2 deletion syndrome (22q11DS) is associated with a broad spectrum of neurodevelopmental phenotypes and is the strongest known single genetic risk factor for schizophrenia. Compared to other rare structural pathogenic genetic variants, 22q11DS is relatively common and one of the most extensively studied. This review provides a state-of-the-art overview of current insights regarding associated neurodevelopmental phenotypes and potential implications for 22q11DS and beyond.Recent FindingsWe will first discuss recent findings with respect to neurodevelopmental phenotypic expression associated with 22q11DS, including psychotic disorders, intellectual functioning, autism spectrum disorders, as well as their interactions. Second, we will address considerations that are important in interpreting these data and propose potential implications for both the clinical care for and the empirical study of individuals with 22q11DS. Third, we will highlight variable penetrance and pleiotropy with respect to neurodevelopmental phenotypes in 22q11DS. We will discuss how these phenomena are consistently observed in the context of virtually all rare pathogenic variants and that they pose substantial challenges from both a clinical and a research perspective.SummaryWe outline how 22q11DS could be viewed as a genetic model for studying neurodevelopmental phenotypes. In addition, we propose that 22q11DS research can help elucidate mechanisms underlying variable expression and pleiotropy of neurodevelopmental phenotypes, insights that are likely relevant for 22q11DS and beyond, including for individuals with other rare pathogenic genetic variants and for individuals with idiopathic neurodevelopmental conditions.

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Data Dentistry: How Data Are Changing Clinical Care and Research

Journal of Dental Research ◽

10.1177/00220345211020265 ◽

2021 ◽

pp. 002203452110202

Author(s):

F. Schwendicke ◽

J. Krois

Keyword(s):

Health Care ◽

Data Sharing ◽

Clinical Care ◽

Open Data ◽

User Interaction ◽

Data Availability ◽

Related Data ◽

Data User ◽

Regulatory Data ◽

Consumer Data

Data are a key resource for modern societies and expected to improve quality, accessibility, affordability, safety, and equity of health care. Dental care and research are currently transforming into what we term data dentistry, with 3 main applications: 1) medical data analysis uses deep learning, allowing one to master unprecedented amounts of data (language, speech, imagery) and put them to productive use. 2) Data-enriched clinical care integrates data from individual (e.g., demographic, social, clinical and omics data, consumer data), setting (e.g., geospatial, environmental, provider-related data), and systems level (payer or regulatory data to characterize input, throughput, output, and outcomes of health care) to provide a comprehensive and continuous real-time assessment of biologic perturbations, individual behaviors, and context. Such care may contribute to a deeper understanding of health and disease and a more precise, personalized, predictive, and preventive care. 3) Data for research include open research data and data sharing, allowing one to appraise, benchmark, pool, replicate, and reuse data. Concerns and confidence into data-driven applications, stakeholders’ and system’s capabilities, and lack of data standardization and harmonization currently limit the development and implementation of data dentistry. Aspects of bias and data-user interaction require attention. Action items for the dental community circle around increasing data availability, refinement, and usage; demonstrating safety, value, and usefulness of applications; educating the dental workforce and consumers; providing performant and standardized infrastructure and processes; and incentivizing and adopting open data and data sharing.

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Challenges and opportunities for ovarian cancer management in the epidemic of Covid-19: lessons learned from Wuhan, China

Journal of Ovarian Research ◽

10.1186/s13048-021-00784-2 ◽

2021 ◽

Vol 14 (1) ◽

Author(s):

Zhilan Chen ◽

Chun Zhang ◽

Jiu Yin ◽

Xin Xin ◽

Hemei Li ◽

...

Keyword(s):

Ovarian Cancer ◽

Large Scale ◽

Treatment Plan ◽

Clinical Care ◽

Gynecologic Cancer ◽

Treatment Phase ◽

Tumor Stage ◽

Current Treatment ◽

Lessons Learned ◽

Cancer Management

AbstractChina and the rest of the world are experiencing an outbreak of the 2019 novel coronavirus disease (COVID-19). Patients with cancer are more susceptible to viral infection and are more likely to develop severe complications, as compared to healthy individuals. The growing spread of COVID-19 presents challenges for the clinical care of patients with gynecological malignancies. Ovarian debulking surgery combined with the frequent need for chemotherapy is most likely why ovarian cancer was rated as the gynecologic cancer most affected by COVID-19. Therefore, ovarian cancer presents a particular challenging task. Concerning the ovarian cancer studies with confirmed COVID-19 reported from large-scale general hospitals in Wuhan, we hold that the treatment plan was adjusted appropriately and an individualized remedy was implemented. The recommendations discussed here were developed mainly based on the experience from Wuhan. We advise that the management strategy for ovarian cancer patients should be adjusted in the light of the local epidemic situation and formulated according to the pathological type, tumor stage and the current treatment phase. Online medical service is an effective and convenient communication platform during the pandemic.

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Considerations for the Management of Women with Breast Cancer during the COVID-19 Pandemic

Indian Journal of Cardiovascular Disease in Women WINCARS ◽

10.1055/s-0040-1716923 ◽

2020 ◽

Vol 5 (03) ◽

pp. 260-263

Author(s):

Monica Irukulla ◽

Palwai Vinitha Reddy

Keyword(s):

Breast Cancer ◽

Resource Allocation ◽

Patient Care ◽

Cancer Patients ◽

Cancer Care ◽

Clinical Care ◽

Current Availability ◽

The Individual

AbstractOutcomes in cancer patients are strongly influenced by timeliness and quality of multidisciplinary interventions. The COVID-19 pandemic has led to severe disruption in cancer care in many countries. This has necessitated several changes in clinical care and workflow, including resource allocation, team segregation and deferment of many elective procedures. Several international oncological societies have proposed guidelines for the care of patients afflicted with breast cancer during the pandemic with a view to optimize resource allocation and maximize risk versus benefit for the individual and society. Clinicians may utilize these recommendations to adapt patient care, based on the current availability of resources and severity of the COVID-19 pandemic in each region. This article discusses the guidelines for care of patients afflicted with breast cancer during the pandemic.

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Reopening Selves: Phenomenological Considerations on Psychiatric Spaces and the Therapeutic Stance

Psychopathology ◽

10.1159/000517888 ◽

2021 ◽

pp. 1-12

Author(s):

Samuel Thoma ◽

Isabelle Schwänzl ◽

Laura Galbusera

Keyword(s):

Social Factors ◽

Phenomenological Research ◽

Social World ◽

Societal Implications ◽

Structural Level ◽

Open Door ◽

Coercive Treatment ◽

Psychiatric Institutions ◽

The Individual

Classical and contemporary phenomenological approaches in psychiatry describe schizophrenia as a disorder of common sense and self-affection. Although taking into account intersubjectivity, this conceptualization still puts forward an individualistic view of the disorder, that is, the intersubjective deficit resides within the person. To overcome such individualism, in this article, we first propose that schizophrenic experience might be understood as arising from a dialectic relation between the self’s loss of openness to the world and the world’s loss of openness to the self. To show the relevance of social factors at the onset of schizophrenic experience, we propose a phenomenological analysis of trigger situations. In the second and main part of this article, we then focus on the implications of these phenomenological insights for the clinical practice: we argue that if schizophrenia is understood as a loss of openness between self and social world, psychiatric institutions should be transformed into spaces that enable a reopening of selves. We first describe enclosing phenomena such as coercive treatment to then, in contrast, present particular forms of open psychiatric spaces such as open door approaches and open dialogue. Besides the institutional-structural level, we also highlight aspects of openness at the intersubjective level of the individual agents, thus particularly emphasizing the role of an open therapeutic stance. We thus speak of (re)opening selves as we believe that the reopening of the patients’ self cannot but be related to and fostered by a reopening of the professionals’ self and stance. We thus argue that openness in the therapeutic stance is key to initiating the further process of recovery, which we describe as a reattunement of selves both at the bodily and narrative level. Last but not least, we sketch out possibilities for future phenomenological research on the question of psychiatric space and draw some broader societal implications.

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Development and acceptability of an educational video about a smoking cessation quitline for use in adult outpatient mental healthcare

Translational Behavioral Medicine ◽

10.1093/tbm/ibaa145 ◽

2021 ◽

Author(s):

Adina S Kazan ◽

Laura M Perry ◽

Wasef F Atiya ◽

Hallie M Voss ◽

Seowoo Kim ◽

...

Keyword(s):

Mental Health ◽

Tobacco Use ◽

Transtheoretical Model ◽

Mental Healthcare ◽

Advisory Board ◽

Future Research ◽

Educational Video ◽

Design Elements ◽

Outpatient Mental Health ◽

Preparation Phase

Abstract Tobacco use is a leading preventable cause of early mortality and is prevalent among adults with mental health diagnoses, especially in the southern USA. Increasing cessation resources in outpatient mental health care and targeting individuals most receptive to changing their behavior may improve cessation. Drawing on the transtheoretical model, our goals were to develop an educational video about the Louisiana Tobacco Quitline and evaluate its acceptability. We designed the video with knowledge derived from Louisiana-specific data (2016 Louisiana Adult Tobacco Survey, N = 6,469) and stakeholder feedback. Bivariate associations between demographic/tobacco-use characteristics and participants’ stage of quitting (preparation phase vs. nonpreparation phase) were conducted, which informed design elements of the video. Four stakeholder advisory board meetings involving current smokers, mental health clinicians, and public health advocates convened to provide iterative feedback on the intervention. Our stakeholder advisory board (n = 10) and external stakeholders (n = 20) evaluated intervention acceptability. We found that 17.9% of Louisiana adults were current smokers, with 46.9% of them in the preparation phase of quitting. Using insights from data and stakeholders, we succeeded in producing a 2-min video about the Louisiana Tobacco Quitline which incorporated three themes identified as important by stakeholders: positivity, relatability, and approachability. Supporting acceptability, 96.7% of stakeholders rated the video as helpful and engaging. This study demonstrates the acceptability of combining theory, existing data, and iterative stakeholder feedback to develop a quitline educational video. Future research should examine whether the video can be used to reduce tobacco use.

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