scholarly journals The United States’ Regulatory Environment Is Evolving to Accommodate a Coming Boom in Gene Therapy Research

2019 ◽  
Vol 24 (3) ◽  
pp. 147-152 ◽  
Author(s):  
Daniel Eisenman
Keyword(s):  
United States ◽  
Gene Therapy ◽  
Clinical Trials ◽  
The United States ◽  
Regulatory Environment ◽  
Evidence Based ◽  
Regulatory Structure ◽  
Regulatory Changes ◽  
Current State ◽  
Therapy Field

Introduction: A dramatic increase in the number of clinical trials involving gene-modified cell therapy and gene therapy is taking place. The field is on the verge of a boom, and the regulatory environment is evolving to accommodate the growth. Discussion: This commentary summarizes the current state of the field, including an overview of the growth. The United States (US) regulatory structure for gene therapy will be summarized, and the evolution of the oversight structure will be explained. Conclusion: The gene therapy field has recently produced its first FDA-approved therapeutics and has a pipeline of other investigational products in the final stages of clinical trials before they can be evaluated by the FDA as safe and effective therapeutics. As research continues to evolve, so must the oversight structure. Biosafety professionals and IBCs have always played key roles in contributing to the safe, evidence-based advancement of gene therapy research. With the recent regulatory changes and current surge in gene therapy research, the importance of those roles has increased dramatically.

Trends in Clinical Trials Reports in Common Cancers Between 1989 and 2000

2003 ◽  
Vol 21 (9) ◽  
pp. 1850-1858 ◽  
Author(s):  
Bruce E. Hillner
Keyword(s):  
United States ◽  
Clinical Trials ◽  
Sample Size ◽  
Financial Incentives ◽  
Cancer Biology ◽  
Pilot Trial ◽  
The United States ◽  
Evidence Based ◽  
Pilot Trials ◽  
Female Genital

Purpose: Cancer clinical trials can be dichotomized into pilot (phase I and phase II) and randomized controlled clinical trials (RCTs). The best data source for evidence-based medicine is from RCTs. However, many patients prefer to enroll onto pilot trials, and many investigators prefer to conduct or refer their patients to pilot trials. This exploratory study sought to describe the epidemiologic patterns of clinical trial reports in common cancers. Methods: A structured review was conducted of MEDLINE citations of all English clinical trials reports published between 1989 and 2000 in breast, lung, colorectal, prostate, and female genital cancers, plus leukemias and lymphomas. Each report was classified by design (RCT, pilot, or other) and country. The abstracts of RCTs were reviewed for sample size. Reports addressing screening or prevention were excluded. Results: A total of 12,035 reports, of which 3,560 were from RCTs, were found. The annual growth in RCT reports in breast, colorectal, and prostate cancer was significant (range, 4.8% to 8.5% per year) but was insignificant in leukemias, lymphomas, and female genital and lung cancers (range, 0.1% to 4.3% per year). Within each cancer, the average sample size per report did not change during the 12 years. Nonrandomized trial reports increased on average 15.1% per year (range, 10.1% to 23.2%). The United States accounted for 30% of all RCT reports and 45% of pilot trial reports. Conclusion: The faster growth in nonrandomized compared with RCT reports may reflect rapid advances in cancer biology or different structural, commercial, and financial incentives, especially in the United States compared with Europe. Unless additional studies show evidence of an increase in their quality, the modest growth in RCT reports may limit future evidence-based cancer care.

scholarly journals Melatonin Use in Pediatrics: Evaluating the Discrepancy in Evidence Based on Country and Regulations Regarding Production

2021 ◽  
Vol 26 (1) ◽  
pp. 4-20
Author(s):  
Michelle Skrzelowski ◽  
Amanda Brookhaus ◽  
Leticia A. Shea ◽  
Daniel J. Berlau
Keyword(s):  
United States ◽  
Autism Spectrum Disorder ◽  
Clinical Trials ◽  
Pediatric Population ◽  
The United States ◽  
Autism Spectrum ◽  
Evidence Based ◽  
Hyperactivity Disorder ◽  
Safety Regulations ◽  
The World

Melatonin manufacturers in the United States have begun producing melatonin products specifically targeted for use in the pediatric population. This paper aims to critically evaluate the evidence available regarding the use of melatonin in children based on where the clinical trials are performed and the regulations regarding the production of melatonin in that country. Melatonin is regulated differently around the world with the least amount of regulation placed on OTC supplements in the United States. The majority of studies evaluating melatonin use in the pediatric population are conducted with children who have comorbidities, such as autism spectrum disorder or attention-deficit/hyperactivity disorder. Evidence supporting the use of US formulations of melatonin in the otherwise healthy pediatric population is non-existent. Based on the lack of safety regulations in place in the United States and the lack of evidence regarding US melatonin products, they should be used sparingly in the otherwise healthy pediatric population, if they are used at all.

scholarly journals Effective buprenorphine use and tapering strategies: Endorsements and insights by people in recovery from opioid use disorder on a Reddit forum

2019 ◽  
Author(s):  
Rachel L Graves ◽  
Abeed Sarker ◽  
Mohammed Ali Al-Garadi ◽  
Yuan-chi Yang ◽  
Jennifer S Love ◽  
...  
Keyword(s):  
United States ◽  
Clinical Trials ◽  
Social Network ◽  
Language Processing ◽  
Recovery Process ◽  
The United States ◽  
Opioid Use Disorder ◽  
Evidence Based ◽  
Opioid Use ◽  
Successful Recovery

AbstractOpioid use disorder (OUD) is a public health emergency in the United States. Over 47,000 overdose-related deaths in 2017 involved opioids. Medication-assisted treatment (MAT), in particular, buprenorphine and buprenorphine combination products such as Suboxone®, is the most effective, evidence-based treatment for OUD. However, there are a limited number of conclusive scientific studies that provide guidance to medical professionals about strategies for using buprenorphine to achieve stable recovery. In this study, we used data-driven natural language processing methods to mine a total of 16,146 posts about buprenorphine from 1933 unique users on the anonymous social network Reddit. Analysis of a sample of these posts showed that 74% of the posts described users’ personal experiences and that the top three topics included advice on using Suboxone® (55.0%), Suboxone® dosage information (35.5%) and information about Suboxone® tapering (32.0%). Based on two models, one that incorporated ‘upvoting’ by other members and one that did not, we found that Reddit users reported more successful recovery with longer tapering schedules, particularly from 2.0 mg to 0.0 mg (median: 93 days; mean: 95 days), as compared to shorter tapering schedules investigated in past clinical trials. Diarrhea, insomnia, restlessness, and fatigue were commonly reported adverse events. Physical exercise, clonidine, and Imodium® were frequently reported to help during the recovery process. Due to the difficulties of conducting longer-term clinical trials involving patients with OUD, clinicians should consider other information sources including peer discussions from the abundant, real-time information available on Reddit.Significance StatementOpioid use disorder (OUD) is a national crisis in the United States and buprenorphine is one of the most effective evidence-based treatments. However, few studies have explored successful strategies for using and tapering buprenorphine to achieve stable recovery, particularly due to the difficulties of conducting long-term studies involving patients with OUD. In this study, we show that discussions on the anonymous social network Reddit may be leveraged, via automatic text mining methods, to discover successful buprenorphine use and tapering strategies. We discovered that longer tapering schedules, compared to those investigated in past clinical trials, may lead to (self-reported) sustained recovery. Furthermore, Reddit posts also provide key information regarding buprenorphine withdrawal, cravings, adjunct medications for withdrawal symptoms and relapse prevention strategies.

440: Barriers to the Practice of Evidence Based Urology in the United States

2007 ◽  
Vol 177 (4S) ◽  
pp. 147-148
Author(s):  
Philipp Dahm ◽  
Hubert R. Kuebler ◽  
Susan F. Fesperman ◽  
Roger L. Sur ◽  
Charles D. Scales ◽  
...  
Keyword(s):  
United States ◽  
The United States ◽  
Evidence Based

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

scholarly journals Ice-Breaking Fleets of the United States and Canada: Assessing the Current State of Affairs and Future Plans

2021 ◽  
Vol 13 (2) ◽  
pp. 703
Author(s):  
Megan Drewniak ◽  
Dimitrios Dalaklis ◽  
Anastasia Christodoulou ◽  
Rebecca Sheehan
Keyword(s):  
United States ◽  
The United States ◽  
Maritime Transportation ◽  
The Arctic ◽  
Polar Ice ◽  
Northwest Passage ◽  
Ice Cap ◽  
Ice Coverage ◽  
Current State ◽  
State Of Affairs

In recent years, a continuous decline of ice-coverage in the Arctic has been recorded, but these high latitudes are still dominated by earth’s polar ice cap. Therefore, safe and sustainable shipping operations in this still frozen region have as a precondition the availability of ice-breaking support. The analysis in hand provides an assessment of the United States’ and Canada’s polar ice-breaking program with the purpose of examining to what extent these countries’ relevant resources are able to meet the facilitated growth of industrial interests in the High North. This assessment will specifically focus on the maritime transportation sector along the Northwest Passage and consists of four main sections. The first provides a very brief description of the main Arctic passages. The second section specifically explores the current situation of the Northwest Passage, including the relevant navigational challenges, lack of infrastructure, available routes that may be used for transit, potential choke points, and current state of vessel activity along these routes. The third one examines the economic viability of the Northwest Passage compared to that of the Panama Canal; the fourth and final section is investigating the current and future capabilities of the United States’ and Canada’s ice-breaking fleet. Unfortunately, both countries were found to be lacking the necessary assets with ice-breaking capabilities and will need to accelerate their efforts in order to effectively respond to the growing needs of the Arctic. The total number of available ice-breaking assets is impacting negatively the level of support by the marine transportation system of both the United States and Canada; these two countries are facing the possibility to be unable to effectively meet the expected future needs because of the lengthy acquisition and production process required for new ice-breaking fleets.

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 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< 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.

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