BACKGROUND:Confirmatory phase III trials aim to provide decisive evidence about a medical product's safety and efficacy. Although these trials are planned and conducted based on accumulated knowledge, they are not without risk or uncertainty. A trial prematurely concluding contributes to great loss in both financial and human research resources.METHODS:We categorized and evaluated trials concluded prematurely after recruitment had begun, as registered in Clinical Trials.gov between January 2013 and August 2017.RESULTS:We found 9828 registered interventional phase III trials; of those, 320 were concluded prematurely. Many clinical trials were concluded prematurely for reasons related to reducing participant risk, such as interim stopping for safety, efficacy, or futility. Yet, 70% trials were halted for other reasons, such as insufficient recruitment (the most often cited reason) or unspecified business decisions. Of all prematurely concluded trials, 102 trials evaluated 72 different novel therapeutics; in 66.7% of these trials, the clinical development program was stopped entirely. Most of the prematurely concluded trials (78%) had not provided results to ClinicalTrials.gov at the time of this analysis.CONCLUSIONS:Evaluation of the factors that influence premature conclusion could inform solutions for improving research participation and help ensure trial completion. Registering and reporting results acknowledges the voluntary contribution and consent expectations of research participants.

Electronic health records (EHRs) have changed how medical information is captured, and they have the potential to be a rich source of information to improve drug development and clinical care. Accelerated in USA by the Health Information Technology for Economic and Clinical Health (HITECH) Act of 2009, the majority of US healthcare providers use EHRs in practice. Although the Office of the National Coordinator for Health Information Technology within the US Department of Health and Human Services (HHS) set forth standards with the intention to create EHRs that could be used to advance healthcare processes,1–3 there is still much room for improvement to adequately capture patient-provided and clinically relevant data needed to support learning healthcare systems that adapt as new clinical knowledge is gained.4 Currently, stakeholders within regulatory agencies, professional organisations, the pharmaceutical industry and payer groups are exploring how data collected during the delivery of routine healthcare or data related to a patient’s health status, defined as real-world data (RWD), can be analysed to generate clinical evidence, known as real-world evidence, to enhance healthcare and medical product development.5 6 This shared interest has led workshops exploring the incentives that are important to each stakeholder group.6–8 Multistakeholder collaborations have formed to empower RWD projects, such as the work being done by the Food and Drug Administration (FDA) Oncology Center of Excellence, healthcare technology company, Flatiron Health, and the American Society of Clinical Oncology CancerLinQ, which will focus on determining the characteristics and clinical outcomes associated with patients with advanced cancer using RWD collected from the EHR.9 The RWD effort is supported with legislative action. The 21st Century Cures Act requires the US FDA to explore and produce guidance on how RWD can inform decision-making, including label expansion for approved products and postmarket commitments.10 Investigating RWD applications is also a …

AimsFDA-approved drug labels are an important source of information for clinicians who prescribe medications for treatment of diabetes. We reviewed drug labels to (1) understand the landscape of classes of medications approved for type 1 diabetes mellitus (T1DM) and type 2 diabetes mellitus (T2DM), (2) explore the indications and safety information and (3) examine their cardiovascular safety.MethodsWe searched four public references and reviewed all FDA-approved labels for “indication and usage,” “adverse effects,” “warnings and precautions,” and “cardiovascular outcomes” from October 1982 to July 2016. We also reviewed FDA drug-safety communications from January 2015 to May 2017.ResultsThe labels reveal 12 classes of medications approved for T2DM with only 2 classes approved for T1DM. There is emerging evidence about cardiovascular safety and risk reduction from diabetes medications which is now being incorporated in drug labels.ConclusionsAll currently available diabetes medications are approved for adults with T2DM with a remarkably limited number for adults with T1DM and children with T1DM or T2DM. The incorporation of emerging data on cardiovascular outcomes in FDA drug labels is expected to influence the way physicians treat patients with diabetes.

Our website uses cookies to enhance your experience. By continuing to use our site, or clicking "Continue," you are agreeing to our Cookie Policy | Continue JAMA Neurology HomeNew OnlineCurrent IssueFor Authors Podcast Publications JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry (1919-1959) JN Learning / CMESubscribeJobsInstitutions / LibrariansReprints & Permissions Terms of Use | Privacy Policy | Accessibility Statement 2023 American Medical Association. All Rights Reserved Search All JAMA JAMA Network Open JAMA Cardiology JAMA Dermatology JAMA Forum Archive JAMA Health Forum JAMA Internal Medicine JAMA Neurology JAMA Oncology JAMA Ophthalmology JAMA Otolaryngology–Head & Neck Surgery JAMA Pediatrics JAMA Psychiatry JAMA Surgery Archives of Neurology & Psychiatry Input Search Term Sign In Individual Sign In Sign inCreate an Account Access through your institution Sign In Purchase Options: Buy this article Rent this article Subscribe to the JAMA Neurology journal

Regulatory science, a complex field which draws on science, law, and policy, is a growing discipline in medical-related applications. Competencies help define both a discipline and the criteria to measure high-quality learning experiences. This paper identifies competencies for regulatory science, how they were developed, and broader recommendations to enhance education and training in this burgeoning field, including a multifaceted training approach.

Data sharing is a key biomedical research theme for the 21st century. Biomedical data sharing is the exchange of data among (non)affiliated parties under mutually agreeable terms to promote scientific advancement and the development of safe and effective medical products. Wide sharing of research data is important for scientific discovery, medical product development, and public health. Data sharing enables improvements in development of medical products, more attention to rare diseases, and cost-efficiencies in biomedical research. We interviewed 11 participants about their attitudes and beliefs about data sharing. Using a qualitative, thematic analysis approach, our analysis revealed a number of themes including: experiences, approaches, perceived challenges, and opportunities for sharing data.

The generation, dissemination, and sharing of research data are key ingredients in contributing to scientific progress and the public good. Data sharing has been encouraged to facilitate open science within the clinical research enterprise, improve the development of drugs and devices, and benefit public health.1 But sharing data is complex. Investigators generally tend to guard research data to retain ownership and property rights, avoid competition, protect confidentiality and privacy, or avoid misuse by unqualified persons. Sharing research data also comes at a cost to the sharer. This Viewpoint examines some approaches and cost considerations involved in sharing participant-level clinical research data.

The generation, dissemination, and sharing of research data are key ingredients in contributing to scientific progress and the public good. Data sharing has been encouraged to facilitate open science within the clinical research enterprise, improve the development of drugs and devices, and benefit public health. To date, the subject has received considerable attention in the media and scientific literature, however focused mainly on philosophical arguments. Of the empirical research that exists, much of it has focused on data sharing among academic investigators in the field of life science, including biomedical research, medicine, and genetics. A number of commentaries and editorials have dealt with the ethical repercussions when researchers choose not to share data; others have discussed real or perceived legal barriers to data sharing. These barriers include (1) compromised intellectual property rights and unfair advantages to competitors, (2) risk of increased product liability, (3) issues of collusion under antitrust laws, and (4) risks to research participant privacy.

BACKGROUND: According to a wide variety of analyses and projections, the potential effects of global climate change on human health are large and diverse. The U.S. National Institutes of Health (NIH), through its basic, clinical, and population research portfolio of grants, has been increasing efforts to understand how the complex interrelationships among humans, ecosystems, climate, climate variability, and climate change affect domestic and global health.OBJECTIVES: In this commentary we present a systematic review and categorization of the fiscal year (FY) 2008 NIH climate and health research portfolio.METHODS: A list of candidate climate and health projects funded from FY 2008 budget appropriations were identified and characterized based on their relevance to climate change and health and based on climate pathway, health impact, study type, and objective.RESULTS: This analysis identified seven FY 2008 projects focused on climate change, 85 climate-related projects, and 706 projects that focused on disease areas associated with climate change but did not study those associations. Of the nearly 53,000 awards that NIH made in 2008, approximately 0.17% focused on or were related to climate.CONCLUSIONS: Given the nature and scale of the potential effects of climate change on human health and the degree of uncertainty that we have about these effects, we think that it is helpful for the NIH to engage in open discussions with science and policy communities about government-wide needs and opportunities in climate and health, and about how NIH's strengths in human health research can contribute to understanding the health implications of global climate change. This internal review has been used to inform more recent initiatives by the NIH in climate and health.

Traumatic brain injury (TBI) may affect 10 million people worldwide. It is considered the signature wound of the conflicts in Iraq and Afghanistan. These injuries result from a bump or blow to the head, or from external forces that cause the brain to move within the head, such as whiplash or exposure to blasts. TBI can cause an array of physical and mental health concerns and is a growing problem, particularly among soldiers and veterans because of repeated exposure to violent environments. One form of treatment for TBI is cognitive rehabilitation therapy (CRT), a patient-specific, goal-oriented approach to help patients increase their ability to process and interpret information. The Department of Defense asked the IOM to conduct a study to determine the effectiveness of CRT for treatment of TBI.