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Innovative Drugs

Eva Ornstova, Monika Sebestianova, Tomas Mlcoch, Klara Lamblova, Tomas Dolezal
BACKGROUND: Highly innovative drugs (HIDs) can be granted 2 to 3years of temporary reimbursement (TR) to provide timely patient access and to collect real-world evidence through registries in the Czech Republic. A TR applicant does not need to comply with cost-effectiveness (CE) requirements and the willingness-to-pay threshold. It is only when mandatory transition to permanent reimbursement (PR) status occurs does the drug need to comply with CE and willingness-to-pay requirements. OBJECTIVES: To describe and evaluate the HID program in the Czech Republic by analyzing the pharmacoeconomic results when a drug starts with TR status and transitions to PR status...
October 10, 2018: Value in Health Regional Issues
Vickie R Driver, Lisa J Gould, Peggy Dotson, Latricia L Allen, Marissa J Carter, Laura L Bolton
Patients with wounds bear significant clinical, personal, and economic burdens yet complete wound healing is the only United States Food and Drug Administration (FDA) recognized primary clinical trial endpoint. The overall goal of this project is to work with FDA to expand the list of acceptable primary endpoints, recognizing that new and innovative treatments, devices and drugs may not have complete healing as the focus. Part 1 of the project surveyed 628 wound care experts who identified and content-validated 15 endpoints most relevant to clinical practice and benefitting patients' lives as primary outcomes in clinical trials...
October 13, 2018: Wound Repair and Regeneration
Qian An, Ruiguang Song, Teresa J Finlayson, Catlainn Sionean, Cyprian Wejnert
We present a simple, comprehensive method for assessing similarity between sex partners of a participant and demonstrate its application using data collected in 2015 as part of CDC's National HIV behavioral surveillance (NHBS) among persons who inject drugs (PWID). We found that the pairwise similarity between sex partners of a survey participant was high. The similarity between second-to-last and third-to-last partners in the past 3 months was significantly higher than that between last and second-to-last partner in partner type, frequency of sex acts, and the contextual characteristics of sex behavior at last sexual encounter...
October 12, 2018: AIDS and Behavior
Jeanette M Bowles, Stephen E Lankenau
Fatal opioid overdoses can be prevented by opioid overdose prevention programs (OOPPs). The present study qualitatively examined the diffusion process of an OOPP among 30 persons who inject drugs (PWIDs) in an opioid-saturated community. Purposive sampling was used to recruit participants into three groups based on familiarity with the OOPP. Findings revealed that participants often adopted the OOPP, which was offered by a local harm reduction organization, if first exposed by staff hosting and implementing it...
October 12, 2018: Qualitative Health Research
Debra Rickwood, Marie Paraskakis, Diana Quin, Nathan Hobbs, Vikki Ryall, Jason Trethowan, Patrick McGorry
AIM: headspace is Australia's innovation in youth mental healthcare and comprises the largest national network of enhanced primary care, youth mental health centres world-wide. headspace centres aim to intervene early in the development of mental ill-health for young people aged 12 to 25 years by breaking down the barriers to service access experienced by adolescents and emerging adults and providing holistic healthcare. Centres have been progressively implemented over the past 12 years and are expected to apply a consistent model of integrated youth healthcare...
October 12, 2018: Early Intervention in Psychiatry
Shuichi Yanai, Shogo Endo
As the population of older individuals grows worldwide, researchers have increasingly focused their attention on identifying key molecular targets of age-related cognitive impairments, with the aim of developing possible therapeutic interventions. Two such molecules are the intracellular cyclic nucleotides, cAMP and cGMP. These second messengers mediate fundamental aspects of brain function relevant to memory, learning, and cognitive function. Consequently, phosphodiesterases (PDEs), which hydrolyze cAMP and cGMP, are promising targets for the development of cognition-enhancing drugs...
October 11, 2018: Molecular Neurobiology
Arthur G Roberts, Morgan E Gibbs
As a result of an increasing aging population, the number of individuals taking multiple medications simultaneously has grown considerably. For these individuals, taking multiple medications has increased the risk of undesirable drug-drug interactions (DDIs), which can cause serious and debilitating adverse drug reactions (ADRs). A comprehensive understanding of DDIs is needed to combat these deleterious outcomes. This review provides a synopsis of the pharmacokinetic (PK) and pharmacodynamic (PD) mechanisms that underlie DDIs...
2018: Clinical Pharmacology: Advances and Applications
Sudeep Pushpakom, Francesco Iorio, Patrick A Eyers, K Jane Escott, Shirley Hopper, Andrew Wells, Andrew Doig, Tim Guilliams, Joanna Latimer, Christine McNamee, Alan Norris, Philippe Sanseau, David Cavalla, Munir Pirmohamed
Given the high attrition rates, substantial costs and slow pace of new drug discovery and development, repurposing of 'old' drugs to treat both common and rare diseases is increasingly becoming an attractive proposition because it involves the use of de-risked compounds, with potentially lower overall development costs and shorter development timelines. Various data-driven and experimental approaches have been suggested for the identification of repurposable drug candidates; however, there are also major technological and regulatory challenges that need to be addressed...
October 12, 2018: Nature Reviews. Drug Discovery
Douglas M Silverstein, Scott O Trerotola, Timothy Clark, Garth James, Wing Ng, Amy Dwyer, Marius C Florescu, Roman Shingarev, Stephen R Ash
Central venous catheters remain a vital option for access for patients receiving maintenance hemodialysis. There are many important and evolving clinical and regulatory considerations for all stakeholders for these devices. Innovation and transparent and comprehensive regulatory review of these devices is essential to stimulate innovation to help promote better outcomes for patients receiving maintenance hemodialysis. A workgroup that included representatives from academia, industry, and the US Food and Drug Administration was convened to identify the major design considerations and clinical and regulatory challenges of central venous catheters for hemodialysis...
October 11, 2018: Clinical Journal of the American Society of Nephrology: CJASN
Xiaodong Ma, Mengqi Hu, Hao Wang, Jiaming Li
P-glycoprotein (P-gp)-mediated multi-drug resistance (MDR) is a well-documented and predominant phenotype hampering patients' response to cancer chemotherapy. Although the past several decades have witnessed the development of three generations of P-gp inhibitors, they have not lived up to the high expectations owing to their drawbacks, as exemplified by limited efficacy, drug-drug interactions (DDIs) and severe untoward reactions. The discovery of artemisinin is a testimony of the importance of traditional Chinese medicine (TCM) in innovative drug discovery...
September 26, 2018: European Journal of Medicinal Chemistry
Eric P Brass, Randy I Burnham, Kate M Reynolds
BACKGROUND: Overdoses due to therapeutic misuse result when the maximum dose of a drug is exceeded while using it for its intended purpose, due to either intentionally exceeding the label dose, misunderstanding the label or use of more than one product with the same ingredient. Nonprescription acetaminophen-containing combination products have been hypothesized to be a risk for therapeutic misuse. This study assessed the contribution of nonprescription acetaminophen-containing products to Poison Center exposures and the time trend in these exposures since public attention was brought to their potential risks...
October 11, 2018: Clinical Toxicology
Sanjeev Kumar, Suneeta Agarwal, Ranvijay
Genomic data nowadays is playing a vital role in number of fields such as personalized medicine, forensic, drug discovery, sequence alignment and agriculture, etc. With the advancements and reduction in the cost of next-generation sequencing (NGS) technology, these data are growing exponentially. NGS data are being generated more rapidly than they could be significantly analyzed. Thus, there is much scope for developing novel data compression algorithms to facilitate data analysis along with data transfer and storage directly...
June 28, 2018: Journal of Bioinformatics and Computational Biology
Yunzi Liu, Yifan Shi, Rong Ren, Jingyuan Xie, Weiming Wang, Nan Chen
Focal segmental glomerulosclerosis (FSGS) is a glomerulonephritis with podocyte injury. The renal prognosis of FSGS is relative poor. The overall remission rate of the FSGS patients with nephrotic syndrome to immunosuppressive treatments was reported as 47-66%, highlighting its therapeutic challenge-lacking in sufficient evidence-based interventions. In first-line treatment of nephrotic syndrome, daily oral prednisolone is a commonly used drug, whereas optimal treatment strategies, like indications and duration, remain controversial...
October 2018: Nephrology
Fen He, Nachuan Wen, Daipeng Xiao, Jianhua Yan, Hongjie Xiong, Shundong Cai, Zhenbao Liu, Yanfei Liu
Aptamers are single-stranded DNA or RNA with 20-100 nucleotides in length that can specifically bind to target molecules via formed three-dimensional structures. These innovative targeting molecules have attracted an increasing interest in biomedical field. Compared to traditional protein antibodies, aptamers have several advantages, such as small size, high binding affinity, specificity, good biocompatibility, high stability and low immunogenicity, which all contribute to their wide application in biomedical field...
October 8, 2018: Current Medicinal Chemistry
Hiddo J L Heerspink, James List, Vlado Perkovic
Despite advances in pharmacotherapy, diabetic kidney disease (DKD) remains associated with a high burden of micro- and macrovascular complications often leading to premature mortality. New therapies are highly desirable to mitigate the burden of this disease. However, there are a number of barriers that hamper drug development in DKD. These include, amongst others, the lengthy and complex clinical trials required to prove drug efficacy and safety, inefficiencies in clinical trial conduct, and the high costs associated with these development programs...
October 2018: Diabetes, Obesity & Metabolism
Peter G M Mol, Aliza Thompson, Hiddo J L Heerspink, Hubert G M Leufkens
Over the past 15 years, three new classes of drugs, glucagon-like peptide-1 (GLP-1) receptor agonists, dipeptidyl peptidase 4 (DPP-4) inhibitors and sodium glucose cotransporter-2 (SGLT-2) inhibitors have been approved to treat type 2 diabetes based on effects on glycemic control. Although large randomized controlled trials have played an important role in characterizing the efficacy and safety of these agents on a population level, questions remain about how best to individualize therapy and target the "right" medicine to the "right" patient...
October 2018: Diabetes, Obesity & Metabolism
Mirco Ponzoni, Flavio Curnis, Chiara Brignole, Silvia Bruno, Daniela Guarnieri, Leopoldo Sitia, Roberto Marotta, Angelina Sacchi, Matteo Bauckneht, Ambra Buschiazzo, Andrea Rossi, Daniela Di Paolo, Patrizia Perri, Alessandro Gori, Angela R Sementa, Laura Emionite, Michele Cilli, Roberto Tamma, Domenico Ribatti, Pier Paolo Pompa, Cecilia Marini, Gianmario Sambuceti, Angelo Corti, Fabio Pastorino
Targeted delivery of anticancer drugs with nanocarriers can reduce side effects and ameliorate therapeutic efficacy. However, poorly perfused and dysfunctional tumor vessels limit the transport of the payload into solid tumors. The use of tumor-penetrating nanocarriers might enhance tumor uptake and antitumor effects. A peptide containing a tissue-penetrating (TP) consensus motif, capable of recognizing neuropilin-1, is here fused to a neuroblastoma-targeting peptide (pep) previously developed. Neuroblastoma cell lines and cells derived from both xenografts and high-risk neuroblastoma patients show overexpression of neuropilin-1...
October 7, 2018: Small
Oriane Rabesandratana, Olivier Goureau, Gaël Orieux
Sight is a major sense for human and visual impairment profoundly affects quality of life, especially retinal degenerative diseases which are the leading cause of irreversible blindness worldwide. As for other neurodegenerative disorders, almost all retinal dystrophies are characterized by the specific loss of one or two cell types, such as retinal ganglion cells, photoreceptor cells, or retinal pigmented epithelial cells. This feature is a critical point when dealing with cell replacement strategies considering that the preservation of other cell types and retinal circuitry is a prerequisite...
2018: Frontiers in Neuroscience
Erica Dugnani, Valeria Sordi, Silvia Pellegrini, Raniero Chimienti, Ilaria Marzinotto, Valentina Pasquale, Daniela Liberati, Gianpaolo Balzano, Claudio Doglioni, Michele Reni, Alessandra Gandolfi, Massimo Falconi, Vito Lampasona, Lorenzo Piemonti
BACKGROUND: Despite the recent introduction of new drugs and the development of innovative multi-target treatments, the prognosis of pancreatic ductal adenocarcinoma (PDAC) remains very poor. Even when PDAC is resectable, the rate of local or widespread disease recurrence remains particularly high. Currently, reliable prognostic biomarkers of recurrence are lacking. We decided to explore the potential usefulness of pancreatic developmental regulators as biomarkers of PDAC relapse. METHODS: We analyzed by quantitative real-time PCR the mRNA of selected factors involved either in pancreatic organogenesis (ISL1, NEUROD1, NGN3, NKX2...
September 25, 2018: Pancreatology: Official Journal of the International Association of Pancreatology (IAP) ... [et Al.]
Caryn L Weiss, Mark R Fairchild, Brian W Stanton, Bertil S Nshime, Paul D Parkanzky
Background: Dexpanthenol is a widely used humectant in hair care products, especially anti-hairfall products. The hair care industry is highly regulated in East Asia and treats products containing the combination of dexpanthenol, zinc pyrithione, and nicotinamide (vitamin B₃) as quasi-drugs. Objective: Because dexpanthenol lacks a UV chromophore, existing methodologies for analysis in finished products include pretreatments and/or HPLC-UV analysis at low wavelengths at which poor signal-to-noise is observed...
October 5, 2018: Journal of AOAC International
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