Abstract 11144: Demographics of Study Participants in Clinical Trials for Cardiovascular Drugs Approved at FDA CDER From 2013 to 2014

Circulation ◽  
2015 ◽  
Vol 132 (suppl_3) ◽  
Author(s):  
Emmanuel O Fadiran ◽  
Hawi Itana ◽  
Merina Elahi ◽  
Ayomide Igun ◽  
Greg Soon ◽  
...  
Keyword(s):  
Clinical Trials ◽  
The United States ◽  
Time Frame ◽  
Disease Prevalence ◽  
Female Participation ◽  
Participation Of Women ◽  
Us Fda ◽  
Demographic Subgroup ◽  
Study Participants ◽  
Percent Female

Introduction: Cardiovascular disease (CVD) is the leading cause of death for women in the United States. To assess the safety and efficacy of CVD drugs in the diverse population of patients who will use them post-approval, the US FDA has implemented guidance and regulation to encourage the inclusion of women and racial/ethnic minorities in drug clinical trials (CTs). This study assessed the participation of women and minorities in CTs for CVD indications for New Drug Applications (NDAs) approved at FDA CDER from 2013-2014. Methods: The sex, race/ethnicity, and age of subjects who participated in CVD drug CTs were assessed from final clinical study reports accessed via internal FDA databases, and were evaluated for the presence of data analysis by sex. The ratio of the percent of women in the CT population was also compared to those in the disease population. Results: Four novel CVD drugs were approved in this time frame, including 2 drugs indicated for pulmonary hypertension (PH) (riociguat, total CT population n=2691; macitentan, n=1884), and 1 drug each for prevention of thrombotic acute CV events (vorapaxar, n=41972) and treatment of neurogenic orthostatic hypotension (NOH) (droxidopa, n=1125). Demographic subgroup analysis indicated that percent female participation was 26% for thrombotic CV events, 40% for NOH, and 47% for PH. Female participation in pivotal trials only (in which benefit-risk was demonstrated) was 24% for thrombotic CV events, 33% for NOH, and 75% for PH. The majority of the trial participants were White (85%), and mean ages were 48 years (PH), 61 years (thrombotic CV events), and 64 years (NOH). Sex-based analysis for both efficacy and safety was presented for all drugs. Three of the NDAs with available disease prevalence data had a ratio of percent female participation to disease prevalence that was between 0.80 and 1.2. Conclusions: Percent female participation varied substantially by disease indication, with PH (a female predominant disease) having the highest female participation. Female participation in the prevention of thrombotic CV events was lower than anticipated based upon disease prevalence in the population. Further exploration of demographic subgroup participation in CTs for CVD drugs in a larger sample size is ongoing.

scholarly journals Bayesian estimation of SARS-CoV-2 prevalence in Indiana by random testing

2021 ◽  
Vol 118 (5) ◽  
pp. e2013906118
Author(s):  
Constantin T. Yiannoutsos ◽  
Paul K. Halverson ◽  
Nir Menachemi
Keyword(s):  
Test Performance ◽  
Randomized Study ◽  
False Negative ◽  
The United States ◽  
Disease Prevalence ◽  
Serological Tests ◽  
Demographic Groups ◽  
External Data ◽  
Study Participants ◽  
Available Information

From 25 to 29 April 2020, the state of Indiana undertook testing of 3,658 randomly chosen state residents for the novel severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) virus, the agent causing COVID-19 disease. This was the first statewide randomized study of COVID-19 testing in the United States. Both PCR and serological tests were administered to all study participants. This paper describes statistical methods used to address nonresponse among various demographic groups and to adjust for testing errors to reduce bias in the estimates of the overall disease prevalence in Indiana. These adjustments were implemented through Bayesian methods, which incorporated all available information on disease prevalence and test performance, along with external data obtained from census of the Indiana statewide population. Both adjustments appeared to have significant impact on the unadjusted estimates, mainly due to upweighting data in study participants of non-White races and Hispanic ethnicity and anticipated false-positive and false-negative test results among both the PCR and antibody tests utilized in the study.

Developing a Clinical Trials Infrastructure in the United States

2012 ◽  
Vol 2 (5) ◽  
Author(s):  
Paul Eisenberg ◽  
◽  
Petra Kaufmann ◽  
Ellen Sigal ◽  
Janet Woodcock ◽  
...  
Keyword(s):  
United States ◽  
Clinical Trials ◽  
The United States

The Use of Digital Clinical Trial Methods in the Evaluation of Digital Therapeutics: A Scoping Review (Preprint)

2019 ◽  
Author(s):  
Allison Hirsch ◽  
Mahip Grewal ◽  
Anthony James Martorell ◽  
Brian Michael Iacoviello
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Research ◽  
Scoping Review ◽  
Digital Technologies ◽  
Good Clinical Practice ◽  
The United States ◽  
Clinical Trial Research ◽  
Digital Methods ◽  
Clinical Trial Methods

BACKGROUND Digital Therapeutics (DTx) provide evidence based therapeutic health interventions that have been clinically validated to deliver therapeutic outcomes, such that the software is the treatment. Digital methodologies are increasingly adopted to conduct clinical trials due to advantages they provide including increases in efficiency and decreases in trial costs. Digital therapeutics are digital by design and can leverage the potential of digital and remote clinical trial methods. OBJECTIVE The principal purpose of this scoping review is to review the literature to determine whether digital technologies are being used in DTx clinical research, which type are being used and whether publications are noting any advantages to their use. As DTx development is an emerging field there are likely gaps in the knowledge base regarding DTx and clinical trials, and the purpose of this review is to illuminate those gaps. A secondary purpose is to consider questions which emerged during the review process including whether fully remote digital clinical research is appropriate for all health conditions and whether digital clinical trial methods are inline with the principles of Good Clinical Practice. METHODS 1,326 records were identified by searching research databases and 1,227 reviewed at the full-article level in order to determine if they were appropriate for inclusion. Confirmation of clinical trial status, use of digital clinical research methods and digital therapeutic status as well as inclusion and exclusion criteria were applied in order to determine relevant articles. Digital methods employed in DTx research were extracted from each article and these data were synthesized in order to determine which digital methods are currently used in clinical trial research. RESULTS After applying our criteria for scoping review inclusion, 11 articles were identified. All articles used at least one form of digital clinical research methodology enabling an element of remote research. The most commonly used digital methods are those related to recruitment, enrollment and the assessment of outcomes. A small number of articles reported using other methods such as online compensation (n = 3), or digital reminders for participants (n = 5). The majority of digital therapeutics clinical research using digital methods is conducted in the United States and increasing number of articles using digital methods are published each year. CONCLUSIONS Digital methods are used in clinical trial research evaluating DTx, though not frequently as evidenced by the low proportion of articles included in this review. Fully remote clinical trial research is not yet the standard, more frequently authors are using partially remote methods. Additionally, there is tremendous variability in the level of detail describing digital methods within the literature. As digital technologies continue to advance and the clinical research DTx literature matures, digital methods which facilitate remote research may be used more frequently.

Health and Service-related Impact of Sexual and Stalking Victimization During United States Military Service on LGBT Service Members

2020 ◽  
pp. 088626052097031
Author(s):  
Cary Leonard Klemmer ◽  
Ashley C. Schuyler ◽  
Mary Rose Mamey ◽  
Sheree M. Schrager ◽  
Carl Andrew Castro ◽  
...  
Keyword(s):  
United States ◽  
Military Service ◽  
The United States ◽  
Active Duty ◽  
Service Members ◽  
United States Military ◽  
Service Outcomes ◽  
Study Participants ◽  
The Military ◽  
Stalking Victimization

Prior research among military personnel has indicated that sexual harassment, stalking, and sexual assault during military service are related to negative health sequelae. However, research specific to LGBT U.S. service members is limited. The current study aimed to explore the health, service utilization, and service-related impact of stalking and sexual victimization experiences in a sample of active-duty LGBT U.S. service members ( N = 248). Respondent-driven sampling was used to recruit study participants. U.S. service members were eligible to participate if they were 18 years or older and active-duty members of the U.S. Army, U.S. Navy, U.S. Marine Corps, or U.S. Air Force. This study included a sizeable portion of transgender service members ( N = 58, 23.4%). Sociodemographic characteristics, characteristics of military service, health, and sexual and stalking victimization in the military were assessed. Regression was used to examine relationships between health and service outcomes and sexual and stalking victimization during military service. Final adjusted models showed that experiencing multiple forms of victimization in the military increased the odds of visiting a mental health clinician and having elevated somatic symptoms, posttraumatic stress disorder symptomatology, anxiety, and suicidality. Sexual and stalking victimization during U.S. military service was statistically significantly related to the mental and physical health of LGBT U.S. service members. Interventions to reduce victimization experiences and support LGBT U.S. service members who experience these types of violence are indicated. Research that examines the role of LGBT individuals’ experiences and organizational and peer factors, including social support, leadership characteristics, and institutional policies in the United States military is needed.

scholarly journals 690 Are participants in obstetrical randomized clinical trials representative of the United States pregnant population?

2021 ◽  
Vol 224 (2) ◽  
pp. S433
Author(s):  
Cynthia Coots ◽  
Stephen Wagner ◽  
Matthew J. Bicocca ◽  
Megha Gupta ◽  
Hector Mendez Figueroa ◽  
...  
Keyword(s):  
United States ◽  
Clinical Trials ◽  
Randomized Clinical Trials ◽  
The United States

scholarly journals Scaling Up: Multisite Open-Label Clinical Trials of MDMA-Assisted Therapy for Severe Posttraumatic Stress Disorder

2021 ◽  
pp. 002216782110236
Author(s):  
Julie B. Wang ◽  
Jessica Lin ◽  
Leah Bedrosian ◽  
Allison Coker ◽  
Ilsa Jerome ◽  
...  
Keyword(s):  
Posttraumatic Stress Disorder ◽  
Clinical Trials ◽  
Posttraumatic Stress ◽  
Clinical Supervision ◽  
Stress Disorder ◽  
The United States ◽  
Health Condition ◽  
Open Label ◽  
Adverse Health Outcomes ◽  
Ptsd Symptom Severity

Background: Posttraumatic stress disorder (PTSD) is a debilitating mental health condition associated with serious adverse health outcomes and functional impairment. Previous MDMA–assisted therapy (MDMA-AT) studies have shown promising results in single site studies. Two open-label studies tested this modality in multisite clinical trials to assess the feasibility of scaling this manualized therapy across 14 North American sites. Method: Cotherapist dyads were trained in the manualized MDMA-AT protocol and administered three experimental sessions 3 to 5 weeks apart among participants with severe PTSD. Cotherapist dyads were provided clinical supervision and evaluated for protocol adherence by centralized raters. Clinician-Administered PTSD Scale for DSM-5 (CAPS-5) assessed change in symptoms severity. Results: Adherence rating scores were high across cotherapist dyads ( M = 95.08%, SD = 3.70%) and sites ( M = 95.23%, SD = 2.20%). CAPS-5 scores decreased following 3 MDMA-AT sessions at 18 weeks post baseline (Δ M = −29.99, Δ SD = 13.45, p < .0001, n = 37, Cohen’s d = 2.2, confidence interval [1.97, 2.47]). MDMA was well tolerated. Conclusions: These findings corroborate previous results that MDMA-AT can achieve significant improvements in PTSD symptom severity and demonstrate scalability of manualized therapy across clinic sites in the United States and Canada.

scholarly journals Multicenter point prevalence evaluation of the utilization and safety of drug therapies for COVID-19 at the onset of the pandemic timeline in the United States

2021 ◽  
Author(s):  
Nathaniel J Rhodes ◽  
Atheer Dairem ◽  
William J Moore ◽  
Anooj Shah ◽  
Michael J Postelnick ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Drug Therapy ◽  
Supportive Care ◽  
The United States ◽  
Primary Objective ◽  
Point Prevalence ◽  
Medical Centers ◽  
Academic Medical ◽  
History Of ◽  
Secondary Objective

Abstract Disclaimer In an effort to expedite the publication of articles related to the COVID-19 pandemic, AJHP is posting these manuscripts online as soon as possible after acceptance. Accepted manuscripts have been peer-reviewed and copyedited, but are posted online before technical formatting and author proofing. These manuscripts are not the final version of record and will be replaced with the final article (formatted per AJHP style and proofed by the authors) at a later time. Purpose There are currently no FDA-approved medications for the treatment of coronavirus disease 2019 (COVID-19). At the onset of the pandemic, off-label medication use was supported by limited or no clinical data. We sought to characterize experimental COVID-19 therapies and identify safety signals during this period. Methods We conducted a non-interventional, multicenter, point prevalence study of patients hospitalized with suspected/confirmed COVID-19. Clinical and treatment characteristics within a 24-hour window were evaluated in a random sample of up to 30 patients per site. The primary objective was to describe COVID-19–targeted therapies. The secondary objective was to describe adverse drug reactions (ADRs). Results A total of 352 patients treated for COVID-19 at 15 US hospitals From April 18 to May 8, 2020, were included in the study. Most patients were treated at academic medical centers (53.4%) or community hospitals (42.6%). Sixty-seven patients (19%) were receiving drug therapy in addition to supportive care. Drug therapies used included hydroxychloroquine (69%), remdesivir (10%), and interleukin-6 antagonists (9%). Five patients (7.5%) were receiving combination therapy. The rate of use of COVID-19–directed drug therapy was higher in patients with vs patients without a history of asthma (14.9% vs 7%, P = 0.037) and in patients enrolled in clinical trials (26.9% vs 3.2%, P &lt; 0.001). Among those receiving drug therapy, 8 patients (12%) experienced an ADR, and ADRs were recognized at a higher rate in patients enrolled in clinical trials (62.5% vs 22%; odds ratio, 5.9; P = 0.028). Conclusion While we observed high rates of supportive care for patients with COVID-19, we also found that ADRs were common among patients receiving drug therapy, including those enrolled in clinical trials. Comprehensive systems are needed to identify and mitigate ADRs associated with experimental COVID-19 treatments.

scholarly journals EPID-07. ACCESS TO GLIOBLASTOMA CLINICAL TRIALS FOR RURAL PATIENTS IN THE UNITED STATES FROM 2010–2020

2020 ◽  
Vol 22 (Supplement_2) ◽  
pp. ii79-ii79
Author(s):  
Kathryn Nevel ◽  
Samuel Capouch ◽  
Lisa Arnold ◽  
Katherine Peters ◽  
Nimish Mohile ◽  
...  
Keyword(s):  
United States ◽  
Clinical Trials ◽  
Rural Communities ◽  
The United States ◽  
Interventional Treatment ◽  
Phase Ii Trials ◽  
Treatment Trials ◽  
Rural Sites ◽  
Rural Patients ◽  
Urban Sites

Abstract BACKGROUND Patients in rural communities have less access to optimal cancer care and clinical trials. For GBM, access to experimental therapies, and consideration of a clinical trial is embedded in national guidelines. Still, the availability of clinical trials to rural communities, representing 20% of the US population, has not been described. METHODS We queried ClinicalTrials.gov for glioblastoma interventional treatment trials opened between 1/2010 and 1/2020 in the United States. We created a Structured Query Language database and leveraged Google application programming interfaces (API) Places to find name and street addresses for the sites, and Google’s Geocode API to determine the county location. Counties were classified by US Department of Agriculture Rural-Urban Continuum Codes (RUCC 1–3 = urban and RUCC 4–9 = rural). We used z-ratios for rural-urban statistical comparisons. RESULTS We identified 406 interventional treatment trials for GBM at 1491 unique sites. 8.7% of unique sites were in rural settings. Rural sites opened an average of 1.7 trials/site and urban sites 2.8 trials/site from 1/2010–1/2020. Rural sites offered more phase II trials (63% vs 57%, p= 0.03) and fewer phase I trials (22% vs 28%, p= 0.01) than urban sites. Rural locations were more likely to offer federally-sponsored trials (p&lt; 0.002). There were no investigator-initiated or single-institution trials offered at rural locations, and only 1% of industry trials were offered rurally. DISCUSSION Clinical trials for GBM were rarely open in rural areas, and were more dependent on federal funding. Clinical trials are likely difficult to access for rural patients, and this has important implications for the generalizability of research as well as how we engage the field of neuro-oncology and patient advocacy groups in improving patient access to trials. Increasing the number of clinical trials in rural locations may enable more rural patients to access and enroll in GBM studies.

scholarly journals Recent Findings on Cell-Based Therapies for COVID19-Related Pulmonary Fibrosis

2021 ◽  
Vol 30 ◽  
pp. 096368972199621
Author(s):  
Hong-Meng Chuang ◽  
Li-Ing Ho ◽  
Horng-Jyh Harn ◽  
Ching-Ann Liu
Keyword(s):  
Stem Cells ◽  
Clinical Trials ◽  
Pulmonary Fibrosis ◽  
Tissue Repair ◽  
Immune Modulation ◽  
Distress Syndrome ◽  
Clinical Symptoms ◽  
The United States ◽  
Past Experiences ◽  
Respiratory Tract Inflammation

COVID-19 has spread worldwide, including the United States, United Kingdom, and Italy, along with its site of origin in China, since 2020. The virus was first found in the Wuhan seafood market at the end of 2019, with a controversial source. The clinical symptoms of COVID-19 include fever, cough, and respiratory tract inflammation, with some severe patients developing an acute and chronic lung injury, such as acute respiratory distress syndrome (ARDS) and pulmonary fibrosis (PF). It has already claimed approximately 300 thousand human lives and the number is still on the rise; the only way to prevent the infection is to be safe till vaccines and reliable treatments develop. In previous studies, the use of mesenchymal stem cells (MSCs) in clinical trials had been proven to be effective in immune modulation and tissue repair promotion; however, their efficacy in treating COVID-19 remains underestimated. Here, we report the findings from past experiences of SARS and MSCs, and how SARS could also induce PF. Such studies may help to understand the rationale for the recent cell-based therapies for COVID-19.

scholarly journals Enabling patient-reported outcome measures in clinical trials, exemplified by cardiovascular trials

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Theresa M. Coles ◽  
Adrian F. Hernandez ◽  
Bryce B. Reeve ◽  
Karon Cook ◽  
Michael C. Edwards ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Outcome Measures ◽  
Contextual Factors ◽  
The United States ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Think Tank ◽  
Level Of Evidence ◽  
Patient Reported ◽  
Evidentiary Standards

Abstract Objectives There has been limited success in achieving integration of patient-reported outcomes (PROs) in clinical trials. We describe how stakeholders envision a solution to this challenge. Methods Stakeholders from academia, industry, non-profits, insurers, clinicians, and the Food and Drug Administration convened at a Think Tank meeting funded by the Duke Clinical Research Institute to discuss the challenges of incorporating PROs into clinical trials and how to address those challenges. Using examples from cardiovascular trials, this article describes a potential path forward with a focus on applications in the United States. Results Think Tank members identified one key challenge: a common understanding of the level of evidence that is necessary to support patient-reported outcome measures (PROMs) in trials. Think Tank participants discussed the possibility of creating general evidentiary standards depending upon contextual factors, but such guidelines could not be feasibly developed because many contextual factors are at play. The attendees posited that a more informative approach to PROM evidentiary standards would be to develop validity arguments akin to courtroom briefs, which would emphasize a compelling rationale (interpretation/use argument) to support a PROM within a specific context. Participants envisioned a future in which validity arguments would be publicly available via a repository, which would be indexed by contextual factors, clinical populations, and types of claims. Conclusions A publicly available repository would help stakeholders better understand what a community believes constitutes compelling support for a specific PROM in a trial. Our proposed strategy is expected to facilitate the incorporation of PROMs into cardiovascular clinical trials and trials in general.

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