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2022 ◽  
Author(s):  
Huiling Zhao ◽  
humaira Rasheed ◽  
Therese Haugdahl Nost ◽  
Yoonsu Cho ◽  
Yi Liu ◽  
...  

Proteome-wide Mendelian randomization (MR) shows value in prioritizing drug targets in Europeans, but limited data has made identification of causal proteins in other ancestries challenging. Here we present a multi-ancestry proteome-wide MR analysis pipeline based on cross-population data from the Global Biobank Meta-analysis Initiative (GBMI). We estimated the causal effects of 1,545 proteins on eight complex diseases in up to 32,658 individuals of African ancestries and 1.22 million individuals of European ancestries. We identified 45 and seven protein-disease pairs with MR and genetic colocalization evidence in the two ancestries respectively. 15 protein-disease pairs showed evidence of differential effects between males and females. A multi-ancestry MR comparison identified two protein-disease pairs with MR evidence of an effect in both ancestries, seven pairs with European-specific effects and seven with African-specific effects. Integrating these MR signals with observational and clinical trial evidence, we were able to evaluate the efficacy of one existing drug, identify seven drug repurposing opportunities and predict seven novel effects of proteins on diseases. Our results highlight the value of proteome-wide MR in informing the generalisability of drug targets across ancestries and illustrate the value of multi-cohort and biobank meta-analysis of genetic data for drug development.


Author(s):  
Mario Silva ◽  
Gianluca Milanese ◽  
Roberta E Ledda ◽  
Sundeep M Nayak ◽  
Ugo Pastorino ◽  
...  

Lung cancer screening (LCS) by low-dose computed tomography is a strategy for secondary prevention of lung cancer. In the last two decades, LCS trials showed several options to practice secondary prevention in association with primary prevention, however, the translation from trial to practice is everything but simple. In 2020, the European Society of Radiology and European Respiratory Society published their joint statement paper on LCS. This commentary aims to provide the readership with detailed description about hurdles and potential solutions that could be encountered in the practice of LCS.


2022 ◽  
Vol 11 ◽  
Author(s):  
Aaron C. Tan ◽  
Drexell H. Boggs ◽  
Eudocia Q. Lee ◽  
Michelle M. Kim ◽  
Minesh P. Mehta ◽  
...  

Brain metastases cause significant morbidity and mortality in patients with advanced cancer. In the era of precision oncology and immunotherapy, there are rapidly evolving systemic treatment options. These novel therapies may have variable intracranial efficacy, and patients with brain metastases remain a population of special interest. Typically, only patients with stable, asymptomatic and/or treated brain metastases are enrolled in clinical trials, or may be excluded altogether, particularly in the setting of leptomeningeal carcinomatosis. Consequently, this leads to significant concerns on the external validity of clinical trial evidence to real-world clinical practice. Here we describe the current trends in cancer clinical trial eligibility for patients with brain metastases in both early and late phase trials, with a focus on targeted and immunotherapies. We evaluate recent newly FDA approved therapies and the clinical trial evidence base leading to approval. This includes analysis of inclusion and exclusion criteria, requirements for baseline screening for brain metastases, surveillance cerebral imaging and incorporation of trial endpoints for patients with brain metastases. Finally, the use of alternative sources of data such as real-world evidence with registries and collaborative studies will be discussed.


2021 ◽  
Author(s):  
Sabina Gall ◽  
Uta Kiltz ◽  
Tanja Kobylinski ◽  
Ioana Andreica ◽  
Kristina Vaupel ◽  
...  

Abstract The aim of this project was to understand patients’ knowledge and opinion about biosimilars and to evaluate patient satisfaction with care after education on multiswitching of biosimilars (bsDMARDs) by rheumatologists in comparison to nurse specialists. Adult patients with rheumatoid arthritis, axial spondyloarthritis or psoriatic arthritis who underwent a non-medical switch to the adalimumab biosimilar MSB 11022 were randomized into a group in which information about multiswitching of bsDMARDs was provided by a nurse specialist or a rheumatologist. Validated outcome tools and standardized parameters for assessment of disease activity and function were used at baseline and 12 weeks after switching. Patients’ satisfaction with care was assessed by the Leeds Satisfaction Questionnaire. A structured questionnaire was used to assess patient’s knowledge. A total of 102 patients was randomized, 40 were seen by the rheumatologist (39.2%) and 62 by the nurse (60.8%). Fifty patients (49%) had already undergone one and 52 multiple switches (51%). Less than one third of patients was able to correctly answer questions on manufacturing, effectiveness, clinical trial evidence and cost of bsDMARDs. Patients were generally satisfied with care irrespective of whether the information had been given by the nurse or the rheumatologist. No difference in outcomes was seen.Patient satisfaction and outcomes after education about bsDMARDs and switching by nurses and rheumatologists were similarly good. The number of switches did not have a negative impact on patient satisfaction.


2021 ◽  
Vol 8 (3) ◽  
pp. 339
Author(s):  
Peni Rinda

The purpose of this research is to examine the provisions of civil procedural law in settling a lawsuit, to know the form of simple, fast, and low-cost concretization in simple lawsuit settlement. The research method uses the research object of simple, fast, and low-cost concretization in the settlement of a simple claim, normative juridical approach, the type of data used in this study is secondary data, which is sourced from the literature. Secondary data consists of primary legal materials, secondary legal materials, legal material collection techniques carried out by literature and the internet, while the data analysis method is analyzed descriptively qualitatively. The results show that the provisions of civil procedural law in resolving claims in court so far have been using the basis of HIR, which do not differentiate between complicated and simple case examinations so that the time for settlement is the same. The process starting from submitting/registering a lawsuit, determining the day of trial by the head of the panel of judges, the parties being summoned appropriately, the trial, evidence to the verdict took a long time, namely 6 months (SUPREME COURT CIRCULAR No. 6 of 1992), so the Supreme Court issued SUPREME COURT CIRCULAR No. 2 of 2014 which provides a time limit for completing the case of five (5) months. The simple, fast, and low-cost principles of concretizing a small claim court can be seen in the settlement stage. Settlement of a simple lawsuit is divided into 4 (four) stages, namely: 1), preliminary stage, 2) stage of case examination, 3). The objection request stage and 4), the simple action decision stage. This simple lawsuit settlement process should take a maximum of 25 (twenty-five) days. The simple principle is embodied in the shortened settlement process mechanism, the fast principle is realized within 25 days of completion, a peace that does not use the provisions of Supreme Court rules No.1 of 2016, while the principle of low cost is realized from a simple settlement mechanism, will be affected by the cost of the case.


2021 ◽  
Author(s):  
D. James Surmeier ◽  
Jack T. Nguyen ◽  
Nicola Lancki ◽  
Charles S. Venuto ◽  
David Oakes ◽  
...  

Trials ◽  
2021 ◽  
Vol 22 (1) ◽  
Author(s):  
Noah A. Haber ◽  
Sarah E. Wieten ◽  
Emily R. Smith ◽  
David Nunan

AbstractNon-pharmaceutical interventions (NPI) for infectious diseases such as COVID-19 are particularly challenging given the complexities of what is both practical and ethical to randomize. We are often faced with the difficult decision between having weak trials or not having a trial at all. In a recent article, Dr. Atle Fretheim argues that statistically underpowered studies are still valuable, particularly in conjunction with other similar studies in meta-analysis in the context of the DANMASK-19 trial, asking “Surely, some trial evidence must be better than no trial evidence?” However, informative trials are not always feasible, and feasible trials are not always informative. In some cases, even a well-conducted but weakly designed and/or underpowered trial such as DANMASK-19 may be uninformative or worse, both individually and in a body of literature. Meta-analysis, for example, can only resolve issues of statistical power if there is a reasonable expectation of compatible well-designed trials. Uninformative designs may also invite misinformation. Here, we make the case that—when considering informativeness, ethics, and opportunity costs in addition to statistical power—“nothing” is often the better choice.


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