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https://www.readbyqxmd.com/read/28644734/high-throughput-phenotypic-screening-of-kinase-inhibitors-to-identify-drug-targets-for-polycystic-kidney-disease
#1
Tijmen H Booij, Hester Bange, Wouter N Leonhard, Kuan Yan, Michiel Fokkelman, Steven J Kunnen, Johannes G Dauwerse, Yu Qin, Bob van de Water, Gerard J P van Westen, Dorien J M Peters, Leo S Price
Polycystic kidney disease (PKD) is a prevalent disorder characterized by renal cysts that lead to kidney failure. Various signaling pathways have been targeted to stop disease progression, but most interventions still focus on alleviating PKD-associated symptoms. The mechanistic complexity of the disease, as well as the lack of functional in vitro assays for compound testing, has made drug discovery for PKD challenging. To identify modulators of PKD, Pkd1(-/-) kidney tubule epithelial cells were applied to a scalable and automated 3D cyst culture model for compound screening, followed by phenotypic profiling to determine compound efficacy...
June 1, 2017: SLAS Discovery
https://www.readbyqxmd.com/read/28642700/pricing-and-reimbursement-of-biosimilars-in-central-and-eastern-european-countries
#2
Paweł Kawalec, Ewa Stawowczyk, Tomas Tesar, Jana Skoupa, Adina Turcu-Stiolica, Maria Dimitrova, Guenka I Petrova, Zinta Rugaja, Agnes Männik, Andras Harsanyi, Pero Draganic
Objectives: The aim of this study was to review the requirements for the reimbursement of biosimilars and to compare the reimbursement status, market share, and reimbursement costs of biosimilars in selected Central and Eastern European (CEE) countries. Methods: A questionnaire-based survey was conducted between November 2016 and January 2017 among experts from the following CEE countries: Bulgaria, Czech Republic, Croatia, Estonia, Hungary, Latvia, Lithuania, Poland, Slovakia, and Romania. The requirements for the pricing and reimbursement of biosimilars were reviewed for each country...
2017: Frontiers in Pharmacology
https://www.readbyqxmd.com/read/28642295/drug-price-disparities-and-other-stories
#3
(no author information available yet)
No abstract text is available yet for this article.
June 22, 2017: BMJ: British Medical Journal
https://www.readbyqxmd.com/read/28639617/gene-therapy-for-spinomuscular-atrophy-a-biomedical-advance-a-missed-opportunity-for-more-equitable-drug-pricing
#4
T Friedmann
An experimental approach for gene therapy of spinomuscular atrophy has been reported to prevent development of the neuromuscular features of this lethal and previously untreatable disorder. The approach involves treatment of patients suffering from SMN1-associated infantile form of the disease with a splice-switching antisense oligonucleotide (ASO) that corrects aberrant splicing of the nearly identical SMN2 gene to allow the generation of functional SMN protein, thereby mitigating the development of the disease...
June 22, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28634131/cost-effectiveness-of-pre-versus-post-liver-transplant-hepatitis-c-treatment-with-direct-acting-antivirals
#5
Sumeyye Samur, Brian Kues, Turgay Ayer, Mark S Roberts, Fasiha Kanwal, Chin Hur, Drew Michael S Donnell, Raymond T Chung, Jagpreet Chhatwal
BACKGROUND & AIMS: Oral direct-acting antivirals (DAAs) for hepatitis C virus (HCV) treatment offer new hope to both pre- and post-liver transplant (LT) patients. However, whether to treat HCV patients pre- versus post-LT is not clear, as treatment can improve liver function but could reduce the chance of receiving a LT while on the waiting list. Our objective was to evaluate the cost-effectiveness of pre-LT versus post-LT HCV treatment with oral DAAs in decompensated cirrhotic patients on the LT waiting list...
June 17, 2017: Clinical Gastroenterology and Hepatology
https://www.readbyqxmd.com/read/28629392/do-investors-value-the-fda-orphan-drug-designation
#6
Kathleen L Miller
BACKGROUND: The Orphan Drug Act is an important piece of legislation that uses financial incentives to encourage the development of drugs that treat rare diseases. This analysis studies the effects of a portion of the Orphan Drug Act, the orphan drug designation. Specifically, it studies the value that investors place on the orphan drug designation, by investigating how investors react to companies' announcing that their product has received the designation. RESULTS: The results, on average, show that the stock price of a company increases by 3...
June 19, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28628853/willingness-to-pay-for-opioid-agonist-treatment-among-opioid-dependent-people-who-inject-drugs-in-ukraine
#7
Iuliia Makarenko, Alyona Mazhnaya, Ruthanne Marcus, Martha J Bojko, Lynn Madden, Sergii Filippovich, Sergii Dvoriak, Frederick L Altice
BACKGROUND: In the context of decreasing external and limited Ukrainian governmental funding for opioid agonist treatments (OAT) for opioid dependent people who inject drugs in Ukraine, information on sustainable financial models is needed. METHODS: Data on 855 opioid dependent people who inject drugs (PWID) were drawn from a cross-sectional nationwide survey of 1613 PWID. They comprised 434 participants who were receiving OAT and 421 who were on OAT in the past or have never been on OAT and were interested in receiving the treatment...
June 16, 2017: International Journal on Drug Policy
https://www.readbyqxmd.com/read/28627808/evaluation-of-a-pharmaceutical-risk-sharing-agreement-when-patients-are-screened-for-the-probability-of-success
#8
Reza Mahjoub, Fredrik Ødegaard, Gregory S Zaric
We analyze a game-theoretic model of a risk-sharing agreement between a payer and a pharmaceutical firm. The drug manufacturer chooses the price while the payer sets the rebate rate and decides which patients are eligible for treatment. The manufacturer provides the payer with a rebate for nonresponding patients. We generalize on the existing literature, by making both price and rebate rate decision variables, allowing the rebate rate to be different from 100%, and incorporating 2 types of administrative costs...
June 19, 2017: Health Economics
https://www.readbyqxmd.com/read/28625684/brand-medications-and-medicare-part-d-how-eye-care-providers-prescribing-patterns-influence-costs
#9
Paula Anne Newman-Casey, Maria A Woodward, Leslie M Niziol, Paul P Lee, Lindsey B De Lott
PURPOSE: To quantify costs of eye care providers' Medicare Part D prescribing patterns for ophthalmic medications and to estimate the potential savings of generic or therapeutic drug substitutions and price negotiation. DESIGN: Retrospective cross-sectional study. PARTICIPANTS: Eye care providers prescribing medications through Medicare Part D in 2013. METHODS: Medicare Part D 2013 prescriber public use file and summary file were used to calculate medication costs by physician specialty and drug...
June 16, 2017: Ophthalmology
https://www.readbyqxmd.com/read/28624978/preferences-for-breast-cancer-risk-reduction-among-brca1-brca2-mutation-carriers-a-discrete-choice-experiment
#10
Alexander Liede, Carol A Mansfield, Kelly A Metcalfe, Melanie A Price, Carrie Snyder, Henry T Lynch, Sue Friedman, Justyna Amelio, Joshua Posner, Steven A Narod, Geoffrey J Lindeman, D Gareth Evans
PURPOSE: Unaffected women who carry BRCA1 or BRCA2 mutations face difficult choices about reducing their breast cancer risk. Understanding their treatment preferences could help us improve patient counseling and inform drug trials. The objective was to explore preferences for various risk-reducing options among women with germline BRCA1/2 mutations using a discrete-choice experiment survey and to compare expressed preferences with actual behaviors. METHODS: A discrete-choice experiment survey was designed wherein women choose between hypothetical treatments to reduce breast cancer risk...
June 17, 2017: Breast Cancer Research and Treatment
https://www.readbyqxmd.com/read/28610626/social-accountability-in-primary-health-care-in-west-and-central-africa-exploring-the-role-of-health-facility-committees
#11
Elsbet Lodenstein, Eric Mafuta, Adolphe C Kpatchavi, Jean Servais, Marjolein Dieleman, Jacqueline E W Broerse, Alpha Amadou Bano Barry, Thérèse M N Mambu, Jurrien Toonen
BACKGROUND: Social accountability has been emphasised as an important strategy to increase the quality, equity, and responsiveness of health services. In many countries, health facility committees (HFCs) provide the accountability interface between health providers and citizens or users of health services. This article explores the social accountability practices facilitated by HFCs in Benin, Guinea and the Democratic Republic of Congo. METHODS: The paper is based on a cross-case comparison of 11 HFCs across the three countries...
June 13, 2017: BMC Health Services Research
https://www.readbyqxmd.com/read/28606975/drivers-of-expenditure-on-primary-care-prescription-drugs-in-10-high-income-countries-with-universal-health-coverage
#12
Steven G Morgan, Christine Leopold, Anita K Wagner
BACKGROUND: Managing expenditures on pharmaceuticals is important for health systems to sustain universal access to necessary medicines. We sought to estimate the size and sources of differences in expenditures on primary care medications among high-income countries with universal health care systems. METHODS: We compared data on the 2015 volume and cost per day of primary care prescription drug therapies purchased in 10 high-income countries with various systems of universal health care coverage (7 from Europe, in addition to Australia, Canada and New Zealand)...
June 12, 2017: CMAJ: Canadian Medical Association Journal, Journal de L'Association Medicale Canadienne
https://www.readbyqxmd.com/read/28606974/drug-prices-how-do-we-get-to-a-better-place
#13
Joel Lexchin
No abstract text is available yet for this article.
June 12, 2017: CMAJ: Canadian Medical Association Journal, Journal de L'Association Medicale Canadienne
https://www.readbyqxmd.com/read/28605615/cancer-drugs-an-international-comparison-of-postlicensing-price-inflation
#14
Philip Savage, Sarah Mahmoud, Yogin Patel, Hagop Kantarjian
PURPOSE: The cost of cancer drugs forms a rising proportion of health care budgets worldwide. A number of studies have examined international comparisons of initial cost, but there is little work on postlicensing price increases. To examine this, we compared cancer drug prices at initial sale and subsequent price inflation in the United States and United Kingdom and also reviewed relevant price control mechanisms. METHODS: The 10 top-selling cancer drugs were selected, and their prices at initial launch and in 2015 were compared...
June 2017: Journal of Oncology Practice
https://www.readbyqxmd.com/read/28603636/make-up-a-missed-lesson-new-policy-to-ensure-the-interchangeability-of-generic-drugs-in-china
#15
Baobin Huang, Sarah L Barber, Mingzhe Xu, Shuanghong Cheng
Generic drugs should be interchangeable with originators in terms of quality and efficacy. With relative lower prices, generic drugs are playing an important role in controlling health expenditures and ensuring access. However, the widespread understanding of "cheap price equals low quality" has a negative impact on the acceptance of generic drugs. In China, medical doctors doubt the efficacy and quality of generic drugs manufactured domestically. To address these concerns, the Chinese State Council released a policy in 2016 to ensure the interchangeability by re-evaluating the quality and efficacy of generic drugs...
June 2017: Pharmacology Research & Perspectives
https://www.readbyqxmd.com/read/28600618/application-of-omics-in-predicting-anti-tnf-efficacy-in-rheumatoid-arthritis
#16
Xi Xie, Fen Li, Shu Li, Jing Tian, Jin-Wei Chen, Jin-Feng Du, Ni Mao, Jian Chen
Rheumatoid arthritis (RA) is a systemic autoimmune disease characterized by progressive joint erosion. Tumor necrosis factor (TNF) antagonists are the most widely used biological disease-modifying anti-rheumatic drug in RA. However, there continue to be one third of RA patients who have poor or no response to TNF antagonists. Following consideration of the uncertainty of therapeutic effects and the high price of TNF antagonists, it is worthy to predict the treatment responses before anti-TNF therapy. According to the comparisons between the responders and non-responders to TNF antagonists by omic technologies, such as genomics, transcriptomics, proteomics, and metabolomics, rheumatologists are eager to find significant biomarkers to predict the effect of TNF antagonists in order to optimize the personalized treatment in RA...
June 10, 2017: Clinical Rheumatology
https://www.readbyqxmd.com/read/28599300/cancer-and-the-kidney-dangereoux-liasons-or-price-paid-for-the-progress-in-medicine
#17
REVIEW
Jolanta Małyszko, Leszek Kozlowski, Klaudia Kozłowska, Maciej Małyszko, Jacek Małyszko
A long time ago, the links between renal disease and malignancy were observed, however, quite recently, their importance was recognized and 'new' subspecialty in nephrology, namely 'onconephrology' was established. In the XXI century, patients with malignancy make up the most growing number of the subjects seen for nephrology consult and/or critical care nephrology services. A plethora of renal problems may be found in patients with malignancy. They may influence not only their short-term outcomes but also the adequate therapy of the underlying oncological problem...
May 23, 2017: Oncotarget
https://www.readbyqxmd.com/read/28597173/problems-interventions-and-their-outcomes-during-the-routine-work-of-hospital-pharmacists-in-bosnia-and-herzegovina
#18
Gordana Ljubojević, Branislava Miljković, Tatjana Bućma, Milica Ćulafić, Milica Prostran, Sandra Vezmar Kovačević
Background In the last 30 years, activities of hospital pharmacists have gone through significant changes. Pharmacists are increasingly involved in patient care. Objectives To explore drug-related and logistic problems, interventions, and their outcomes during routine everyday work of hospital pharmacists. Setting Institute for physical medicine and rehabilitation, Banja Luka, Bosnia and Herzegovina. Methods In the period of January 2013-October 2015 a prospective observational study was performed. Medical doctors, nurses, therapists, and patients addressed pharmacists, face-to-face or by telephone, with drug-related problems (DRPs) and/or logistic issues...
June 9, 2017: International Journal of Clinical Pharmacy
https://www.readbyqxmd.com/read/28596826/field-evaluation-of-the-efficacy-of-common-anthelmintics-used-in-the-control-of-gastrointestinal-nematodes-of-sheep-in-dabat-district-northwest-ethiopia
#19
Zewdu Seyoum, Yitayew Demessie, Basazinew Bogale, Achenef Melaku
BACKGROUND: Gastrointestinal nematode (GIN) infections are the main impediments that restrict the welfare and productivity of small ruminant in the world. Effective management of GINs in grazing sheep relies heavily on the use of highly efficacious anthelmintic drugs. However, anthelmintic resistance is becoming a significant concern in the world, and this phenomenon severely threatens the potential utilisation of this control strategy. Therefore, this study was conducted 1) to evaluate the efficacy of commonly used anthelmintic on GINs in naturally infected sheep and 2) to assess the farmers' perception on anthelmintics utilisation practices in Dabat district, Northwest Ethiopia...
2017: Irish Veterinary Journal
https://www.readbyqxmd.com/read/28592621/bevacizumab-for-metastatic-colorectal-cancer-a-global-cost-effectiveness-analysis
#20
Daniel A Goldstein, Qiushi Chen, Turgay Ayer, Kelvin K W Chan, Kiran Virik, Ariel Hammerman, Baruch Brenner, Christopher R Flowers, Peter S Hall
BACKGROUND: In the U.S., the addition of bevacizumab to first-line chemotherapy in metastatic colorectal cancer (mCRC) has been demonstrated to provide 0.10 quality-adjusted life years (QALYs) at an incremental cost-effectiveness ratio (ICER) of $571,000/QALY. Due to variability in pricing, value for money may be different in other countries. Our objective was to establish the cost-effectiveness of bevacizumab in mCRC in the U.S., U.K., Canada, Australia, and Israel. METHODS: We performed the analysis using a previously established Markov model for mCRC...
June 7, 2017: Oncologist
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