Despite dramatic advances in the molecular pathogenesis of disease, translation of basic biomedical research into safe and effective clinical applications remains a slow, expensive, and failure-prone ...endeavor. To pursue opportunities for disruptive translational innovation, the U.S. National Institutes of Health (NIH) intends to establish a new entity, the National Center for Advancing Translational Sciences (NCATS). The mission of NCATS is to catalyze the generation of innovative methods and technologies that will enhance the development, testing, and implementation of diagnostics and therapeutics across a wide range of diseases and conditions. The new center's activities will complement, and not compete with, translational research being carried out at NIH and elsewhere in the public and private sectors.
TRIPOD provides guidance on the key items to report when describing studies developing, evaluating (or validating), or updating clinical prediction models.10,11 Although TRIPOD aims primarily to ...improve reporting, it also leads to more comprehensive understanding, conduct, and analysis of prediction model studies, ensuring that prediction models can be picked up by subsequent researchers and users to be studied further and used to guide health care, thus encouraging reproducible research and reduce research waste. ...concerns have been raised that artificial intelligence in clinical medicine is overhyped and, if not used with proper guidance, knowledge, or expertise, has methodological shortcomings, poor transparency, and poor reproducibility.12 Methodological concerns include an often incorrect focus on classification over prediction, overfitting (whereby too many predictors or features are included for the sample size), lack of robust assessment of predictive accuracy when used with other data than those from which they were developed (validation), weak and unbiased comparison with simpler modelling approaches, and lack of transparency of the artificial intelligence and machine learning algorithm, which limits independent evaluation. Clearly, the consequences of making a wrong or inaccurate prediction are substantial for the clinical application of a machine learning prediction model, such as the deep learning models for detection of stroke or wrist fractures approved by the US Food and Drug Administration.13 Therefore, the clinical community must not get mesmerised by the artificial intelligence and machine learning revolution, and artificial intelligence and machine learning prediction models must be appropriately developed, evaluated, and—if needed—tailored to different situations before they are used in daily medical practice.
In this Viewpoint directors of the NIH and NIDDK summarize the agency's 2020-2030 Strategic Plan for NIH Nutrition Research, a roadmap to establish a science of nutrition as a means to prevent and ...treat illness and to develop actionable, evidence-based dietary recommendations across cultures and settings.
Prediction models are developed to aid health care providers in estimating the probability or risk that a specific disease or condition is present (diagnostic models) or that a specific event will ...occur in the future (prognostic models), to inform their decision making. However, the overwhelming evidence shows that the quality of reporting of prediction model studies is poor. Only with full and clear reporting of information on all aspects of a prediction model can risk of bias and potential usefulness of prediction models be adequately assessed. The Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) Initiative developed a set of recommendations for the reporting of studies developing, validating, or updating a prediction model, whether for diagnostic or prognostic purposes. This article describes how the TRIPOD Statement was developed. An extensive list of items based on a review of the literature was created, which was reduced after a Web based survey and revised during a three day meeting in June 2011 with methodologists, health care professionals, and journal editors. The list was refined during several meetings of the steering group and in e-mail discussions with the wider group of TRIPOD contributors. The resulting TRIPOD Statement is a checklist of 22 items, deemed essential for transparent reporting of a prediction model study. The TRIPOD Statement aims to improve the transparency of the reporting of a prediction model study regardless of the study methods used. The TRIPOD Statement is best used in conjunction with the TRIPOD explanation and elaboration document. To aid the editorial process and readers of prediction model studies, it is recommended that authors include a completed checklist in their submission (also available at www.tripod-statement.org).To encourage dissemination of the TRIPOD Statement, this article is freely accessible on the Annals of Internal Medicine Web site (www.annals.org) and will be also published in BJOG, British Journal of Cancer, British Journal of Surgery, BMC Medicine, The BMJ, Circulation, Diabetic Medicine, European Journal of Clinical Investigation, European Urology, and Journal of Clinical Epidemiology. The authors jointly hold the copyright of this article. An accompanying explanation and elaboration article is freely available only on www.annals.org; Annals of Internal Medicine holds copyright for that article.
A strategic approach to COVID-19 vaccine R&D Corey, Lawrence; Mascola, John R; Fauci, Anthony S ...
Science (American Association for the Advancement of Science),
05/2020, Letnik:
368, Številka:
6494
Journal Article
Recenzirano
Odprti dostop
A public-private partnership and platform for harmonized clinical trials aims to accelerate licensure and distribution
There is an unprecedented need to manufacture and distribute enough safe and ...effective vaccine to immunize an extraordinarily large number of individuals in order to protect the entire global community from the continued threat of morbidity and mortality from severe acute respiratory syndrome–coronavirus 2 (SARS-CoV-2). The global need for vaccine and the wide geographic diversity of the pandemic require more than one effective vaccine approach. Collaboration will be essential among biotechnology and pharmaceutical companies, many of which are bringing forward a variety of vaccine approaches (
1
). The full development pathway for an effective vaccine for SARS-CoV-2 will require that industry, government, and academia collaborate in unprecedented ways, each adding their individual strengths. We discuss one such collaborative program that has recently emerged: the ACTIV (Accelerating COVID-19 Therapeutic Interventions and Vaccines) public-private partnership. Spearheaded by the U.S. National Institutes of Health (NIH), this effort brings together the strengths of all sectors at this time of global urgency. We further discuss a collaborative platform for conducting harmonized, randomized controlled vaccine efficacy trials. This mechanism aims to generate essential safety and efficacy data for several candidate vaccines in parallel, so as to accelerate the licensure and distribution of multiple vaccine platforms and vaccines to protect against COVID-19 (coronavirus disease 2019).
As gene therapy continues to change, so must the federal framework set up to oversee it. As new biotechnologies continue to emerge, the NIH and the FDA are proposing reductions in duplicative ...oversight and changes to the role of the Recombinant DNA Advisory Committee.
Causal directed acyclic graphs (cDAGs) have become popular tools for researchers to better examine biases related to causal questions. DAGs comprise a series of arrows connecting nodes that represent ...variables and in doing so can demonstrate the causal relation between different variables. cDAGs can provide researchers with a blueprint of the exposure and outcome relation and the other variables that play a role in that causal question. cDAGs can be helpful in the design and interpretation of observational studies in pulmonary, critical care, sleep, and cardiovascular medicine. They can also help clinicians and researchers to better identify the structure of different biases that can affect the validity of observational studies. Most of the available literature on cDAGs and their function use language that might be unfamiliar to clinicians. This article explains cDAG terminology and the principles behind how they work. We use cDAGs and clinical examples that are mostly focused in the area of pulmonary medicine to describe the structure of confounding, selection bias, overadjustment bias, and detection bias. These principles are then applied to a more complex published case study on the use of statins and COPD mortality. We also introduce readers to other resources for a more in-depth discussion of causal inference principles.
The diary entry of an 8-year-old girl with cystic fibrosis indicates that Aug. 25, 1989, was an important day for her (Figure 1). That was the day the research teams at the University of Michigan and ...the Hospital for Sick Children, Toronto, announced the discovery of the cystic fibrosis gene and the most common mutation, a three-base deletion that results in a missing phenylalanine in codon 508 (denoted the Phe508del
CFTR
mutation). We hoped that the gene discovery would someday lead to effective treatments for children and adults with cystic fibrosis, but we knew that would be a long road.
2,3
. . .
Prediction models are developed to aid health care providers in estimating the probability or risk that a specific disease or condition is present (diagnostic models) or that a specific event will ...occur in the future (prognostic models), to inform their decision making. However, the overwhelming evidence shows that the quality of reporting of prediction model studies is poor. Only with full and clear reporting of information on all aspects of a prediction model can risk of bias and potential usefulness of prediction models be adequately assessed. The Transparent Reporting of a multivariable prediction model for Individual Prognosis Or Diagnosis (TRIPOD) Initiative developed a set of recommendations for the reporting of studies developing, validating, or updating a prediction model, whether for diagnostic or prognostic purposes. This article describes how the TRIPOD Statement was developed. An extensive list of items based on a review of the literature was created, which was reduced after a Web-based survey and revised during a 3-day meeting in June 2011 with methodologists, health care professionals, and journal editors. The list was refined during several meetings of the steering group and in e-mail discussions with the wider group of TRIPOD contributors. The resulting TRIPOD Statement is a checklist of 22 items, deemed essential for transparent reporting of a prediction model study. The TRIPOD Statement aims to improve the transparency of the reporting of a prediction model study regardless of the study methods used. The TRIPOD Statement is best used in conjunction with the TRIPOD explanation and elaboration document. To aid the editorial process and readers of prediction model studies, it is recommended that authors include a completed checklist in their submission (also available at www.tripod-statement.org).
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