The US National Library of Medicine

Kittrie, Elizabeth

doi:10.1145/3209542.3209546

Cited by 4 publications

(3 citation statements)

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“…They allow researchers to link data across different research and therefore have an advantage in data interoperation and integration. The National Institute of Health (NIH) and National Library of Medicine(NLM) have established the importance of CDEs in their strategic plans [8], [9]. However, it is not straightforward to apply CDEs for research data collection in RADx-rad projects.…”

Section: Discussionmentioning

confidence: 99%

IMI-CDE: an interactive interface for collaborative mapping of study variables to common data elements

Tao

Chou

et al. 2022

2022 IEEE 10th International Conference on Healthcare Informatics (ICHI)

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The National Institute of Health (NIH) launches the RADx Radical research collaboratives (RADx-rad) to advance new, non-traditional approaches for COVID-19 testing. RADxrad projects are required to adopt common data elements (CDEs) to collect data to increase data interoperability. To overcome the challenges in finding appropriate CDEs for a wide range of study variables, we create a web application -IMI-CDE to ease the burden of mapping study variables to CDEs from researchers. IMI-CDE can automatically recommend CDE candidates for a study variable based on its name and description. Together with interactive mapping interfaces, IMI-CDE allows researchers to perform variable-CDE mapping with one mouse click. In addition, the IMI-CDE application supports users with multiple roles to work collaboratively on the mapping tasks. We have piloted the IMI-CDE with RADx-rad projects. 22 researchers from 8 different projects have started to use the IMI-CDE system for variable-CDE mappings. The beta-testing evaluators reported the system is intuitive, effective, and easy to use.

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Section: Discussionmentioning

confidence: 99%

IMI-CDE: an interactive interface for collaborative mapping of study variables to common data elements

Tao

Chou

et al. 2022

2022 IEEE 10th International Conference on Healthcare Informatics (ICHI)

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“…Clinical trial information, including design characteristics, number of study participants, and outcomes assessed by the FDA and the EMA to review the safety and efficacy of gene therapy products, was extracted from the US National Library of Medicine ClinicalTrials.gov and the EU Clinical Trials Register. 19,20 The agency recommended therapeutic indications, route of administration, therapeutic class, and boxed warnings of gene therapy drugs data were collected from the FDA-approved label and the EMA-approved summary of product characteristics. Orphan designation status expedited review processes used to review gene therapies, and the dates of the investigational new drug application submission, biological license application submission and authorization, and market withdrawal were extracted from the FDA and EMA websites.…”

Section: Methodsmentioning

confidence: 99%

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

et al. 2023

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Background: Gene therapy, altering the genes inside human cells, has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies.Objective: This study assessed the characteristics of the clinical trials, authorizations, and prices of gene therapies in the United States and the European Union.Research Design: We collected regulatory information from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and manufacturer-listed prices from the United States, UK, and Germany. Descriptive statistics and t tests were conducted in the study.Results: As of January 1, 2022, the FDA and EMA authorized 8 and 10 gene therapies, respectively. The FDA and EMA granted orphan designation to all gene therapies except talimogene laherparepvec. Pivotal clinical trials were nonrandomized, open level, uncontrolled, phase I-III, and included a limited number of patients. Study primary outcomes were mainly surrogate endpoints without demonstration of direct patient benefit. The price of gene therapies at market entry ranged from $200,064 to $2,125,000 million.Conclusions: Gene therapy is used to treat incurable diseases that affect only a small number of patients (orphan diseases). Based on this, they are approved by the EMA and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost.

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“…German national study with 10-15 participating study sites (academic hospitals and community clinics). List of participating sites can be obtained from continuously updated entries found in the registry on clinicaltrials.gov [28].…”

Section: Study Setting {9}mentioning

confidence: 99%

Liver-HERO: hepatorenal syndrome-acute kidney injury (HRS-AKI) treatment with transjugular intrahepatic portosystemic shunt in patients with cirrhosis—a randomized controlled trial

Ripoll

Franken

Aschenbach

et al. 2023

Trials

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Background Patients with cirrhosis and ascites (and portal hypertension) are at risk of developing acute kidney injury (AKI). Although many etiologies exist, hepatorenal AKI (HRS-AKI) remains a frequent and difficult-to-treat cause, with a very high mortality when left untreated. The standard of care is the use of terlipressin and albumin. This can lead to reversal of AKI, which is associated to survival. Nevertheless, only approximately half of the patients achieve this reversal and even after reversal patients remains at risk for new episodes of HRS-AKI. TIPS is accepted for use in patients with variceal bleeding and refractory ascites, which leads to a reduction in portal pressure. Although preliminary data suggest it may be useful in HRS-AKI, its use in this setting is controversial and caution is recommended given the fact that HRS-AKI is associated to cardiac alterations and acute-on-chronic liver failure (ACLF) which represent relative contraindications for transjugular intrahepatic portosystemic shunt (TIPS). In the last decades, with the new definition of renal failure in patients with cirrhosis, patients are identified at an earlier stage. These patients are less sick and therefore more likely to not have contraindications for TIPS. We hypothesize that TIPS could be superior to the standard of care in patients with HRS-AKI. Methods This study is a prospective, multicenter, open, 1:1-randomized, controlled parallel-group trial. The main end-point is to compare the 12-month liver transplant-free survival in patients assigned to TIPS compared to the standard of care (terlipressin and albumin). Secondary end-point include reversal of HRS-AKI, health-related Quality of Life (HrQoL), and incidence of further decompensation among others. Once patients are diagnosed with HRS-AKI, they will be randomized to TIPS or Standard of Care (SOC). TIPS should be placed within 72 h. Until TIPS placement, TIPS patients will be treated with terlipressin and albumin. Once TIPS is placed, terlipressin and albumin should be weaned off according to the attending physician. Discussion If the trial were to show a survival advantage for patients who undergo TIPS placement, this could be incorporated in routine clinical practice in the management of patients with HRS-AKI. Trial registration Clinicaltrials.gov NCT05346393. Released to the public on 01 April 2022.

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The US National Library of Medicine

Cited by 4 publications

References 0 publications

IMI-CDE: an interactive interface for collaborative mapping of study variables to common data elements

IMI-CDE: an interactive interface for collaborative mapping of study variables to common data elements

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

Liver-HERO: hepatorenal syndrome-acute kidney injury (HRS-AKI) treatment with transjugular intrahepatic portosystemic shunt in patients with cirrhosis—a randomized controlled trial

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