scholarly journals In the future simulations will replace clinical trials

Author(s):  
Martin A. Ebert ◽  
Val Gebski ◽  
Clive Baldock
Keyword(s):  
2016 ◽  
Vol 10 (2) ◽  
pp. 1 ◽  
Author(s):  
Melody Hermel ◽  
Rebecca Duffy ◽  
Alexander Orfanos ◽  
Isabelle Hack ◽  
Shayna McEnteggart ◽  
...  

Cardiac registries have filled many gaps in knowledge related to arrhythmogenic cardiovascular conditions. Despite the less robust level of evidence available in registries when compared with clinical trials, registries have contributed a range of clinically useful information. In this review, the authors discuss the role that registries have played – related to diagnosis, natural history, risk stratification, treatment, and genetics of arrhythmogenic cardiovascular conditions – in closing knowledge gaps, and their role in the future.


Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Ciska Verbaanderd ◽  
Ilse Rooman ◽  
Isabelle Huys

Abstract Background Finding new therapeutic uses for existing medicines could lead to safe, affordable and timely new treatment options for patients with high medical needs. However, due to a lack of economic incentives, pharmaceutical developers are rarely interested to invest in research with approved medicines, especially when they are out of basic patent or regulatory protection. Consequently, potential new uses for these medicines are mainly studied in independent clinical trials initiated and led by researchers from academia, research institutes, or collaborative groups. Yet, additional financial support is needed to conduct expensive phase III clinical trials to confirm the results from exploratory research. Methods In this study, scientific and grey literature was searched to identify and evaluate new mechanisms for funding clinical trials with repurposed medicines. Semi-structured interviews were conducted with 16 European stakeholders with expertise in clinical research, funding mechanisms and/or drug repurposing between November 2018 and February 2019 to consider the future perspectives of applying new funding mechanisms. Results Traditional grant funding awarded by government and philanthropic organisations or companies is well known and widely implemented in all research fields. In contrast, only little research has focused on the application potential of newer mechanisms to fund independent clinical research, such as social impact bonds, crowdfunding or public-private partnerships. Interviewees stated that there is a substantial need for additional financial support in health research, especially in areas where there is limited commercial interest. However, the implementation of new funding mechanisms is facing several practical and financial challenges, such as a lack of expertise and guidelines, high transaction costs and difficulties to measure health outcomes. Furthermore, interviewees highlighted the need for increased collaboration and centralisation at a European and international level to make clinical research more efficient and reduce the need for additional funding. Conclusions New funding mechanisms to support clinical research may become more important in the future but the unresolved issues identified in the current study warrant further exploration.


2005 ◽  
Vol 2 (1) ◽  
pp. 72-79 ◽  
Author(s):  
Jennifer Litchfield ◽  
Jenny Freeman ◽  
Henrik Schou ◽  
Mark Elsley ◽  
Robert Fuller ◽  
...  

2021 ◽  
Author(s):  
Samar Khalifa

Abstract Background: Frontotemporal dementia is a common type of dementia and is a group of progressive neurodegenerative syndromes usually caused by the accumulation of pathological tau or TDP-43 proteins. The review is identifying the clinical measures including neuropsychological scores and functional measures. Methods: A systematic review was conducted covering the clinical trials done to investigate the Frontotemporal Dementia. The sample was taken from Pubmed library. 28 results were found in a range of time from 2016 to 2021. The excluded papers were 17. Results: A total of 10,349 articles were identified at the first stage of papers selecting. All records were screened in order to include and exclude by title/abstract and then based on full text. After excluding articles by year and type of papers, a total of 28 articles were identified through the databases. Following this, the irrelevant papers from databases were removed from original articles, and finally 11 articles were included based on their title/abstract. Full articles were then sourced for about 600 references. It included 732 patients and 195 controls as a total. Conclusions: The review describes the clinical and RCT trials for FTD in the last five years so it can be very updated information for the researchers to cover information required for their researches in the future ones.


2018 ◽  
Vol 19 (12) ◽  
pp. 3770 ◽  
Author(s):  
Xianbin Zhang ◽  
Ahmed Abdelrahman ◽  
Brigitte Vollmar ◽  
Dietmar Zechner

Yes-associated protein, a core regulator of the Hippo-YAP signaling pathway, plays a vital role in inhibiting apoptosis. Thus, several studies and reviews suggest that yes-associated protein is a good target for treating cancer. Unfortunately, more and more evidence demonstrates that this protein is also an essential contributor of p73-mediated apoptosis. This questions the concept that yes-associated protein is always a good target for developing novel anti-cancer drugs. Thus, the aim of this review was to evaluate the clinical relevance of yes-associated protein for cancer pathophysiology. This review also summarized the molecules, processes and drugs, which regulate Hippo-YAP signaling and discusses their effect on apoptosis. In addition, issues are defined, which should be addressed in the future in order to provide a solid basis for targeting the Hippo-YAP signaling pathway in clinical trials.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13598-e13598
Author(s):  
Helen Winter ◽  
Joanna Willis ◽  
Stephen Lang ◽  
Kay Drury ◽  
Jonathan Heywood ◽  
...  

e13598 Background: The impact on cancer outcomes from the Covid-19 pandemic has yet to be determined. Concerns persist on screening, delays in diagnosis, treatment interruptions and outcomes of infection in the immunosuppressed. The need for agile working has been exemplified by establishment of Nightingale Hospitals, staff redeployment and sudden integration of virtual consultations into clinical working. With most cancer clinical trials halted, recruitment into COVID-19 research became essential and embedded into the everyday. Here we present how rapid implementation of COVID-19 randomised clinical trials within an NHS organisation during the pandemic was achieved. Methods: A COVID-19 senior facilitation committee was set up to provide oversight, maximise staff capacity and resource and prioritise studies. Specific strategies to maximise access and clinical trials recruitment for patients including children and those with solid tumours were designed. These included presence of a research nurse at clinical ward rounds and team meetings, the promotion of protocol and informed consent training to all including doctors in the acute settings and weekly research meetings to share-best practice. Reflecting on learnings from this time provide an opportunity to consider how we adjust working for our patients in the future. Results: The integration of research into the everyday working of clinical teams looking after patients with COVID-19 has become the norm. The provision of protocol and informed consent training for all levels of staff and the consideration of all patients for trials during clinical ward rounds and multi-disciplinary meetings, have ensured access to trials has become embedded. The integration of research nurses working, upskilling and prompting clinical colleagues has ensured equity of access and provided a research presence and focus during the busy clinical day. The adoption of cross-disciplinary working, sharing best practice and a culture of commitment and support to the trials ensures no patient is denied the opportunity to participate. Three RTCs opened over 7 weeks. At one site 1904 patients were screened for one of the randomised-controlled trials and over 18% of these patients (351) were recruited and 175 patients declined. Conclusions: The pandemic has had a devastating impact across the UK. However, a coordinated and collaborative multi-disciplinary approach has supported high recruitment and equity of access for patients into COVID-19 trials. Learnings from this work may lead to embedding clinical trials and access to translational research for cancer patients in the future as we recover from the full impact of the pandemic. COVID-19 research has demonstrated how increased recruitment accelerates access and implementation of new innovations and novel drug combinations.The full impact of improved access to cancer research in the future during COVID recovery is worthy of more research.


2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 1527-1527
Author(s):  
Waqas Haque ◽  
Ann M. Geiger ◽  
Celette Sugg Skinner ◽  
Rasmi Nair ◽  
Simon Craddock Lee ◽  
...  

1527 Background: Patient accrual for cancer clinical trials is suboptimal. The complexity of applying eligibility criteria and enrolling patients may deter oncologists from recommending patients for a trial. As such, there is a need to understand how experience, training, and clinical decision support impact physician practices and intentions related to trial accrual. Methods: From May to September 2017, we conducted a survey on clinical trial accrual in a national sample of medical, surgical, and radiation oncologists. The 20-minute survey assessed barriers and facilitators to clinical trial accrual, including experience (e.g., “In the past 5 years, have you been a study or site PI of a trial?”), training (e.g., “Did you receive training about trial design and recruitment as part of medical school, residency, or fellowship? After fellowship?”), and clinical decision support (e.g., “What kind of clinical decision support has your practice implemented?). We used logistic regression to identify factors associated with frequency of discussing trials (with ≥25% of patients) and likelihood of recommending a trial to a patient (likely or very likely) in the future. Results: Survey respondents (n = 1,030) were mostly medical oncologists (59%), age 35-54 years (67%), male (74%), and not in academic practice (58%). About 18% of respondents (n = 183) reported discussing trials with ≥25% of their patients, and 80% reported being likely or very likely to recommend a trial to a patient in the future. Prior experience as principal investigator of a trial was associated with both frequency of discussing trials (OR 3.27, 95% CI 2.25, 4.75) and likelihood of recommending a trial in the future (OR 5.22, 95% CI 3.71, 7.34), as was receiving additional training in clinical trials after fellowship (discussion with patients: OR 2.48, 95% CI 1.80, 3.42; recommend in future: OR 1.92, 95% CI 1.37, 2.69). Implementing clinical decision support was not associated with discussing trials with ≥25% of patients (OR 1.12, 95% CI 0.76, 1.67), but was associated with being likely to recommend a trial in the future (OR 1.73, 95% CI 1.11, 2.71). Conclusions: In a national survey of oncologists, we observed differences in physician practices and intention related to clinical trial accrual. Whereas the vast majority (80%) reported being likely or very likely to recommend trials in the future, far fewer (20%) reported discussing trials with their patients within the past 5 years. Implementation of clinical decision support – electronic tools intended to optimize patient care and identification of patient eligibility – was not associated with frequency of past discussion of clinical trials but was associated with recommending a trial in the future. Given the stronger association between experience as a site Principal Investigator and recommending a trial, future research should explore how improving opportunities to lead a clinical trial impact trial accrual.


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