Trends driving clinical trials into large clinical care settings

2018 ◽  
Vol 17 (10) ◽  
pp. 703-704 ◽  
Author(s):  
Kenneth Getz
Keyword(s):  
Clinical Trials ◽  
Clinical Care

scholarly journals COVD-07. THE IMPACT OF COVID-19 ON PATIENTS AND CAREGIVERS AFFECTED BY BRAIN TUMORS: THE PATIENT NAVIGATOR PERSPECTIVE

2020 ◽  
Vol 22 (Supplement_2) ◽  
pp. ii22-ii22
Author(s):  
Kyle Walsh
Keyword(s):  
Clinical Trials ◽  
Brain Tumor ◽  
Clinical Care ◽  
Structured Interview ◽  
Patient Navigator ◽  
Structured Interviews ◽  
Social Distancing ◽  
Tumor Patient ◽  
Patient Navigators ◽  
The Impact

Abstract BACKGROUND Preliminary evidence indicates that glioma patients are at higher risk for COVID-19 complications due to systemic immunosuppression. Interruptions in cancer care may exacerbate patient and caregiver anxiety, but surveying patients/caregivers about their COVID-19 experiences is often limited by attainable sample sizes and over-reliance upon single-institution experiences. METHODS To explore how COVID-19 is impacting brain tumor patients/caregivers across the U.S., we performed semi-structured interviews with brain tumor patient navigators employed by two different 501(c)3 nonprofit organizations. A semi-structured interview guide was used, utilizing prompts and open-ended questions to facilitate dialogue. A core set of COVID-19 topics were covered, including: financial issues, coping strategies, geographic variability, variability by tumor grade/histology, disruptions in care continuity, accessing clinical trials, psychosocial issues, and end-of-life care. Interviews were audio-recorded, transcribed, and organized by discussion topic to identify emerging themes. Inductive sub-coding was completed using the constant comparison method, within and between transcripts. RESULTS/CONCLUSIONS Ten patient navigators were interviewed between April 15th and May 8th, with interviews lasting approximately one hour (range 38-77minutes). Navigators reported having contact with 183 unique brain tumor families during the pandemic (range 7–38 families per navigator). High concordance emerged across narratives, revealing important considerations for the neuro-oncology workforce. The most prominent theme was increased caregiver burden, attributed to maintaining social distancing by reducing visits from home-health aides and friends/family. A related theme that applied to both patients and caregivers was increased social isolation due to social distancing, suspension of in-person support groups, and church/temple closures. Accessing clinical trials was a recurrent issue, exacerbated by patients’ increasing unwillingness to travel. Glioblastoma patients, especially those with recurrent tumors, expressed greater reluctance to travel. Access to standard-of-care treatment was rarely interrupted, but reduced access to supportive services – especially physical and occupational therapy – was identified as an emerging COVID-related deficiency in clinical care.

scholarly journals The European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC): experiences from a successful ERS Clinical Research Collaboration

Breathe ◽  
2017 ◽  
Vol 13 (3) ◽  
pp. 180-192 ◽  
Author(s):  
James D. Chalmers ◽  
Megan Crichton ◽  
Pieter C. Goeminne ◽  
Michael R. Loebinger ◽  
Charles Haworth ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Research Collaboration ◽  
Clinical Care ◽  
Research Priorities ◽  
Chronic Obstructive ◽  
List Type ◽  
European Respiratory Society ◽  
Obstructive Pulmonary Disease ◽  
Research Education

In contrast to airway diseases like chronic obstructive pulmonary disease or asthma, and rare diseases such as cystic fibrosis, there has been little research and few clinical trials in bronchiectasis. Guidelines are primarily based on expert opinion and treatment is challenging because of the heterogeneous nature of the disease.In an effort to address decades of underinvestment in bronchiectasis research, education and clinical care, the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) was established in 2012 as a collaborative pan-European network to bring together bronchiectasis researchers. The European Respiratory Society officially funded EMBARC in 2013 as a Clinical Research Collaboration, providing support and infrastructure to allow the project to grow.EMBARC has now established an international bronchiectasis registry that is active in more than 30 countries both within and outside Europe. Beyond the registry, the network participates in designing and facilitating clinical trials, has set international research priorities, promotes education and has participated in producing the first international bronchiectasis guidelines. This manuscript article the development, structure and achievements of EMBARC from 2012 to 2017.Educational aimsTo understand the role of Clinical Research Collaborations as the major way in which the European Respiratory Society can stimulate clinical research in different disease areasTo understand some of the key features of successful disease registriesTo review key epidemiological, clinical and translational studies of bronchiectasis contributed by the European Multicentre Bronchiectasis Audit and Research Collaboration (EMBARC) project in the past 5 yearsTo understand the key research priorities identified by EMBARC for the next 5 years

scholarly journals Patterns and Predictors of Emicizumab Adherence in People with Hemophilia

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2178-2178
Author(s):  
Ang Li ◽  
Catlin Goodfriend ◽  
John Sokol ◽  
Rebecca Kruse-Jarres
Keyword(s):  
Clinical Trials ◽  
Half Life ◽  
Clinical Care ◽  
Multivariable Analysis ◽  
Skewed Distribution ◽  
Prior Exposure ◽  
Age Group ◽  
Active Inhibitor ◽  
Routine Prophylaxis

Introduction: Emicizumab is a subcutaneously administered, humanized bispecific monoclonal antibody that is recently approved for hemostatic prophylaxis in people with hemophilia A (PWH) with or without factor VIII inhibitors. We hypothesize that the new route and frequency of administration would lead to better treatment adherence compared to factor or bypass products in PWH outside of clinical trials. We performed the current study to test the hypothesis and to examine potential predictors of non-adherence associated with emicizumab treatment. Methods: We performed a retrospective cohort study at the Washington Center for Bleeding Disorders. Inclusion criteria included PWH with moderate to severe hereditary hemophilia (FVIII <5%), clinician recommendation for routine prophylaxis, receipt of clinical care and medication from a hemophilia treatment center (HTC), at least 12 months of prior exposure to factor or bypass products, and at least 3 months of emicizumab treatment. For patients who were previously enrolled in emicizumab clinical trials, adherence data were collected from the time of first commercial product administration. Adherence percentage (%) was estimated as days of drug dispensed / days elapsed x 100 and non-adherence % was determined as 100 - adherence %. Relevant patient, condition, treatment, and socioeconomic variable data were collected from review of medical records. To assess the difference in drug adherence, we compared the adherence % of emicizumab versus that of factor or bypass product in the same patients using the paired t test. To assess predictors of non-adherence %, we used a generalized linear model (GLM) with log link and gamma distribution to account for the right skewed distribution of the outcome. A multivariable GLM was built to incorporate the most significant predictors of non-adherence. Results: We identified 56 PWH that initiated commercial emicizumab from 1/2018 to 5/2019 at our HTC. Five patients were excluded for fewer than 3 months of follow-up on treatment and 3 patients were excluded for not having prior exposure to factor therapy. The remaining 48 patients had a median duration on emicizumab of 7 months (IQR 5-9) at the time of study. The most common dosing frequency was weekly administration (77%). The median age at treatment index date was 17 years (IQR 9-36), 65% were Caucasian, and 46% had Medicaid or Medicare insurance. The majority of patients had a diagnosis of severe hemophilia (90%), 42% had a history of inhibitor (15% active inhibitor), and 25% were previously enrolled in an emicizumab interventional trial. Prior to emicizumab initiation, 46% of patients had 5 or more self-reported annualized bleeds. The most common reason for emicizumab initiation was patient preference (35%), followed by breakthrough bleeding (33%), difficult venipuncture (21%), and shortened factor half-life (10%). Among 12 patients who previously received only on demand treatment, their adherence on emicizumab was 89%. Among 36 patients who previously received routine prophylaxis, their adherence was significantly higher on emicizumab (98%) than factor/bypass products (89%) (p=0.002). Specifically, 18 out of 48 patients (38%) had factor/bypass adherence <75% (or refused prophylaxis) and 2 out of 48 patients (4%) had emicizumab adherence <75%. Various factors were associated with increased emicizumab non-adherence (Table 1). Age group had the strongest association where young and older adult PWH had more non-adherence % than children and adolescents (Figure 1). On multivariable analysis, age group, active inhibitor, and prior factor/bypass agent non-adherence (episodic or <75% usage) were significantly associated with increased emicizumab non-adherence. Conclusions: In the current study, we found that PWH requiring routine prophylaxis were more likely to be adherent to emicizumab than previous factor or bypass agents. Age group (young adult), active inhibitor, and prior non-adherence to factor product were significant predictors for decreased emicizumab adherence but the differences were small. Given the long half-life of the drug, the significance of non-perfect adherence on bleeding outcomes needs to be studied prospectively with longer clinical follow-up. Disclosures No relevant conflicts of interest to declare.

Sensible use of observational clinical data

2011 ◽  
Vol 22 (1) ◽  
pp. 7-13 ◽  
Author(s):  
J Marc Overhage ◽  
Lauren M Overhage
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Medical Research ◽  
Observational Data ◽  
Clinical Data ◽  
Low Cost ◽  
Clinical Care ◽  
Data Sets ◽  
Large Size ◽  
Health Care Operations

Observational data sets offer many potential advantages for medical research including their low cost, large size and generalisability. Because they are collected for clinical care and health care operations purposes, observational data sets have some limitations that must be considered in order to perform useful analyses. Sensible use of observational data sets can yield valuable insights, particularly when clinical trials are impractical.

Evolution of natriuretic peptide biomarkers in heart failure: Implications for clinical care and clinical trials

2018 ◽  
Vol 254 ◽  
pp. 215-221 ◽  
Author(s):  
Nicolas Vodovar ◽  
Alexandre Mebazaa ◽  
James L. Januzzi ◽  
Gillian Murtagh ◽  
Wendy Gattis Stough ◽  
...  
Keyword(s):  
Heart Failure ◽  
Clinical Trials ◽  
Natriuretic Peptide ◽  
Clinical Care

scholarly journals Digital Health Applications for Pharmacogenetic Clinical Trials

Genes ◽  
2020 ◽  
Vol 11 (11) ◽  
pp. 1261
Author(s):  
Hetanshi Naik ◽  
Latha Palaniappan ◽  
Euan A. Ashley ◽  
Stuart A. Scott
Keyword(s):  
Clinical Trials ◽  
Trial Design ◽  
Digital Health ◽  
Clinical Care ◽  
Vital Signs ◽  
Medication Administration ◽  
Therapeutic Interventions ◽  
Participant Engagement ◽  
Patient Reported ◽  
Health Related

Digital health (DH) is the use of digital technologies and data analytics to understand health-related behaviors and enhance personalized clinical care. DH is increasingly being used in clinical trials, and an important field that could potentially benefit from incorporating DH into trial design is pharmacogenetics. Prospective pharmacogenetic trials typically compare a standard care arm to a pharmacogenetic-guided therapeutic arm. These trials often require large sample sizes, are challenging to recruit into, lack patient diversity, and can have complicated workflows to deliver therapeutic interventions to both investigators and patients. Importantly, the use of DH technologies could mitigate these challenges and improve pharmacogenetic trial design and operation. Some DH use cases include (1) automatic electronic health record-based patient screening and recruitment; (2) interactive websites for participant engagement; (3) home- and tele-health visits for patient convenience (e.g., samples for lab tests, physical exams, medication administration); (4) healthcare apps to collect patient-reported outcomes, adverse events and concomitant medications, and to deliver therapeutic information to patients; and (5) wearable devices to collect vital signs, electrocardiograms, sleep quality, and other discrete clinical variables. Given that pharmacogenetic trials are inherently challenging to conduct, future pharmacogenetic utility studies should consider implementing DH technologies and trial methodologies into their design and operation.

scholarly journals How to design and set up a clinical trial part 1: the research question

2019 ◽  
Vol 12 (3) ◽  
pp. 111-116
Author(s):  
Amarpreet Atwal ◽  
Philip E Benson
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Relevance ◽  
Clinical Care ◽  
Research Question ◽  
Relevant Information ◽  
Evidence Base ◽  
Human Participants ◽  
Set Up

Data from clinical trials involving human participants are essential in establishing an evidence base about the safety and effectiveness of our treatments. This first article describes the steps involved in designing and setting up a clinical trial, from establishing the research question(s) to searching the literature. Acquiring some knowledge about how to set up a clinical trial will allow the conscientious clinician to use the most relevant information to provide the highest possible standards of clinical care for his/her patients. CPD/Clinical Relevance: Even if a clinician is not, has never been, nor is ever planning to be involved in research, he/she should understand and be able to interpret the data from clinical trials.

scholarly journals Conducting clinical trials in heart failure during (and after) the COVID-19 pandemic: an Expert Consensus Position Paper from the Heart Failure Association (HFA) of the European Society of Cardiology (ESC)

2020 ◽  
Vol 41 (22) ◽  
pp. 2109-2117 ◽  
Author(s):  
Stefan D Anker ◽  
Javed Butler ◽  
Muhammad Shahzeb Khan ◽  
William T Abraham ◽  
Johann Bauersachs ◽  
...  
Keyword(s):  
Heart Failure ◽  
Clinical Trials ◽  
European Society ◽  
Clinical Care ◽  
Position Paper ◽  
Adverse Outcomes ◽  
Web Based ◽  
Effective Care ◽  
America South ◽  
European Society Of Cardiology

Abstract The coronavirus disease 2019 (COVID-19) pandemic, caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has important implications for the safety of participants in clinical trials and the research staff caring for them and, consequently, for the trials themselves. Patients with heart failure may be at greater risk of infection with COVID-19 and the consequences might also be more serious, but they are also at risk of adverse outcomes if their clinical care is compromised. As physicians and clinical trialists, it is our responsibility to ensure safe and effective care is delivered to trial participants without affecting the integrity of the trial. The social contract with our patients demands no less. Many regulatory authorities from different world regions have issued guidance statements regarding the conduct of clinical trials during this COVID-19 crisis. However, international trials may benefit from expert guidance from a global panel of experts to supplement local advice and regulations, thereby enhancing the safety of participants and the integrity of the trial. Accordingly, the Heart Failure Association of the European Society of Cardiology on 21 and 22 March 2020 conducted web-based meetings with expert clinical trialists in Europe, North America, South America, Australia, and Asia. The main objectives of this Expert Position Paper are to highlight the challenges that this pandemic poses for the conduct of clinical trials in heart failure and to offer advice on how they might be overcome, with some practical examples. While this panel of experts are focused on heart failure clinical trials, these discussions and recommendations may apply to clinical trials in other therapeutic areas.

scholarly journals Evaluation of Clinically Meaningful Changes in Measures of Frailty

2020 ◽  
Vol 75 (6) ◽  
pp. 1143-1147 ◽  
Author(s):  
Il-Young Jang ◽  
Hee-Won Jung ◽  
Hea Yon Lee ◽  
Hyungchul Park ◽  
Eunju Lee ◽  
...  
Keyword(s):  
Older Adults ◽  
Clinical Trials ◽  
Clinical Care ◽  
Frailty Index ◽  
Large Change ◽  
Community Dwelling ◽  
Deficit Accumulation ◽  
Frailty Phenotype ◽  
Euroqol 5D ◽  
Community Dwelling Older Adults

Abstract Background To determine the clinically meaningful changes and responsiveness of widely used frailty measures. Methods We analyzed data from a prospective cohort study of 1,135 community-dwelling older adults who underwent assessments of frailty and health-related quality of life using the EuroQol-5D at baseline and 1 year later. Frailty measures included deficit-accumulation frailty index (FI); frailty phenotype; Fatigue, Resistance, Ambulation, Illness, and Loss of Weight scale; and the Study of Osteoporotic Fracture (SOF) index. We determined the clinically meaningful changes by the distribution-based method and the anchor-based method using the EuroQol-5D score and responsiveness indices. Results Frailty measures were available in 925 participants at 1 year (81.5%). Based on the distribution-based method, small and large clinically meaningful changes were 0.019 and 0.057 for FI, 0.249 and 0.623 for frailty phenotype, 0.235 and 0.587 for FRAIL scale, and 0.116 and 0.289 for SOF index, respectively. The anchor-based estimates of small and large changes were 0.028 and 0.076 for FI, 0.097 and 0.607 for frailty phenotype, 0.269 and 0.368 for FRAIL scale, and 0.023 and 0.287 for SOF index, respectively. Based on the responsiveness index, per-group sample sizes to achieve 80% power in clinical trials, ranged from 51 (FI) to 7,272 (SOF index) for a small change and 9 (FI) to 133 (FRAIL scale) for a large change. Conclusions The estimates of clinically meaningful change of frailty measures can inform the choice of frailty measures to track longitudinal changes of frailty in clinical trials and clinical care of community-dwelling older adults.

Sign in / Sign up

Export Citation Format

Share Document