Right now, in the right way: U. S. Food and Drug Administration’s expanded access program and patient rights

Holbein, Monika; Weatherwax, Kevin; Gravelin, Misty; Hutchinson, Raymond J.; Mashour, George A.

doi:10.1017/cts.2018.318

Cited by 7 publications

(7 citation statements)

References 10 publications

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“…While toxicity was common, even as assessed through our composite endpoint, it was an uncommon reason for discontinuing therapy, Whether the most appropriate means of accessing experimental medications outside of a clinical trial includes the FDA's Expanded Access Program versus the Right To Try Act has been an ongoing debate in the literature and lay press. 3,11,12 Much of the criticism of the Expanded Access Program stems from concerns that it is burdensome and prohibitive to patients receiving experimental, but potentially effective, medications. We present data from the FDA over the last two years (the only time period in our study in which aggregate national pediatric data were available) of this study Unplanned Clinic visits for toxicity, ED visits for toxicity, and/or admissions/ transfers to ICU for toxicity.…”

Section: Discussionmentioning

confidence: 99%

Retrospective evaluation of single patient investigational new drug (IND) requests in pediatric oncology

et al. 2021

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Section: Discussionmentioning

confidence: 99%

Retrospective evaluation of single patient investigational new drug (IND) requests in pediatric oncology

et al. 2021

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“…We believed that "compassionate use drug" can not only meet the special needs of patients but also carry out clinical effectiveness observation, research and analysis, so as to improve the research efficiency and benefit patients [38][39][40][41][42] . We believed that "compassionate use drug" can not only meet the special needs of patients but also carry out clinical effectiveness observation, research and analysis, so as to improve the research efficiency and benefit patients [38][39][40][41][42] .…”

Section: Discussionmentioning

confidence: 99%

“…For these patients, we suggested that: based on the “compassionate use drug” principle, with safe and obvious antiviral potential drugs, to conduct a staged small batch and single-arm clinical trials is feasible. We believed that “compassionate use drug” can not only meet the special needs of patients but also perform clinical effectiveness observation, research and analysis, so as to enhance the efficiency of research and benefit the patients [38-42]. Also, given a large number of clinical cases have accumulated information, and using available existing data for statistics and analysis with the help of new statistical methods such as clinical data-mining [43-45] and real-world study [46-48], etc., can help in quickly obtaining some very valuable information and save research time.…”

Section: Discussionmentioning

confidence: 99%

Systematic Review of the Registered Clinical Trials of Coronavirus Disease 2019 (COVID-19)

Zhu

Gao

Robert

et al. 2020

Preprint

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Background: Since the outbreak of coronavirus diseases 2019 (COVID-19), many researchers in China have immediately carried out clinical research scheme of the COVID-19. But, there is still a lack of systematic review of registered clinical trials. Therefore, we made the first systematic review of the clinical trials of COVID-19 in order to provide evidence for the control of the COVID-19. Methods: The database from the Chinese Clinical Registration Center and the ClinicalTrials.gov were searched to collect the registered clinical trials of COVID-19. The retrieval inception date is February 9, 2020. Two evaluators independently selected literature, extracted data and evaluated the risk of bias. This study is based on the recommendations of PRISMA in Cochrane handbook. Results: A total of 75 COVID-19 registered clinical trials (63 interventional studies and 12 observational studies) were obtained. 97.3% of clinical trials were initiated by Chinese organizations. Only 11 trials have begun to recruit patients, and all registered clinical trials have not been completed. Most of the trials are early clinical exploratory trials or in pre-experiment stage (only two trials of Remdesivir in Ⅲ stage), and the sample size of subjects recruited is small. The main intervention methods include traditional Chinese medicine treatment, western medicine treatment and integrated Chinese and Western medicine treatment. The subjects were mainly non severe adult patients (≥ 18 years old). The main outcomes were clinical observation and examination. The duration of most trials was more than 5 months, and the median of the intervention study was 180 d (95% CI: 146.3 -328.9 d); the median of the observation period was 334 d (95% CI: 166.6 -363.4 d). Overall, both the methodology quality of intervention register trials and observational trials are low. Conclusions: Disorderly and intensive clinical trialsof COVID-19 using traditional Chinese medicine and Western medicine are ongoing or will be carried out in China. However, based on the poor quality and small sample size and long completion period, we will not be able to obtain reliable, high-quality clinical evidence about COVID-19 treatment for quite a long time in the future. Improving the quality of study design, prioritizing promising drugs, and using different designs and statistical methods are worth advocating and recommending for the clinical trials of COVID-19 in China.

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“…Further funding was obtained in 2018 through a supplementary award from NCATS for Transforming Expanded Access to Maximize Support and Study (TEAMSS), a multi-site effort aimed at developing a federated, national consortium for EA interventions to advance clinical care and translational research by improving patient access to experimental therapies. At this point, key stakeholders in the EA program advocated for the use of the FDA's EA pathway (as opposed to the Right to Try Act) to provide critically ill patients with investigational medical products through a system that makes patient safety and care paramount [16].…”

Section: The Establishment Of Michr's Ea Programmentioning

confidence: 99%

Adapting an Expanded Access program to enable investigational treatments for COVID-19

Samuels

Champagne

Gravelin

et al. 2022

J. Clin. Trans. Sci.

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Retrospective case studies of initiatives supported by the National Institutes of Health's Clinical and Translational Science Award (CTSA) hubs can be used to identify facilitators and barriers of translational science. This case study investigates how a CTSA Expanded Access program adapted to changing FDA guidance issued in 2020 to support clinicians' treatment of COVID-19 patients in Michigan. We studied how this program changed throughout the pandemic to support physicians' requests for remdesivir, convalescent plasma, and other uses of unapproved drugs and novel medical devices. A protocol for retrospective translational science case studies of health interventions developed by CTSA evaluators was used for this case study. Data collection methods included seven interviews and a review of institutional data, peerreviewed publications, news stories, and other public records. The barriers identified include evolving guidance, misalignment of organizational operations, and the complexity of the research infrastructure. The facilitators of translation include collaboration between research and care teams, increasing engagement with a broad network of supporters, and ongoing professional development for research staff. The findings of this case study can be used to inform future investigations of the principles underlying the translational process.

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Right now, in the right way: U. S. Food and Drug Administration’s expanded access program and patient rights

Cited by 7 publications

References 10 publications

Retrospective evaluation of single patient investigational new drug (IND) requests in pediatric oncology

Retrospective evaluation of single patient investigational new drug (IND) requests in pediatric oncology

Systematic Review of the Registered Clinical Trials of Coronavirus Disease 2019 (COVID-19)

Adapting an Expanded Access program to enable investigational treatments for COVID-19

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