Thomsenconnell9385

Alternatively, submitting multiple parallel variations with their own corresponding EU-RMPs can result in very complicated procedural wrap-up activities and very short-lived approved versions. In this article, we describe an approach to the management of multiple Type II variations, which is now in line with the recently revised European Medicines Agency (EMA) frequently asked questions (FAQ) guidance on how to manage grouped Type II variation applications, whereby four parallel Type II variation procedures were successfully initiated simultaneously with a single EU-RMP.BACKGROUND Growth in development, approvals, and revenue of drugs treating rare diseases (orphan drugs) has been increasing over the last four decades, which has drawn substantial attention to these products. Much of this growth has been attributed to the incentives created by the Orphan Drug Act, which includes a seven-year exclusivity period for the approval of rare disease indications. OBJECTIVE This study aims to compare the effective market exclusivity period of small molecule new molecular entities (NMEs) for rare (orphan) and non-rare (non-orphan) diseases approved by the U.S. Food and Drug Administration (FDA) from 2001-2012. While the overall length of a drug's effective market exclusivity period has been explored previously, there is little empirical research evaluating the differences in its duration between drugs for rare and non-rare diseases. METHODS Data sources utilized in this analysis included the NME Drug and New Biologic Approvals Reports, Orange Book, Orphan Drug Product Designation Datab in comparison with non-orphan NMEs. Only NMEs that were approved for the treatment of both orphan and non-orphan diseases experience lower hazard of generic entry and longer exclusivity periods compared with non-orphan drugs with a single indication.The correct name of the second author should be "Moritz Fehrle", and not "Mortiz Fehrle" as given in the original publication of the article.BACKGROUND Rare diseases (defined as affecting  less then  1 in 2000 Europeans) may collectively affect up to approximately 8% of the population. The low prevalence of individual diseases limits patient studies and data collection is a key challenge; international rare disease patient registries are essential for optimal data collection and research. Registry data achieves value when research conducted on them are published-this is termed evidence generation. OBJECTIVE The aim of this study was to examine selected factors and their association with evidence generation, via scientific publication, from international rare disease patient registry data. METHODS All international rare disease patient registries listed in the Orphanet 2018 report were analysed. Rates of scientific publications were compared by funding stream, disease area and registry size using multivariable regression analyses. Publication characteristics, such as novelty of findings, were also compared by registry funding stream, disease area and duration of operation. RESULTS Privately funded registries had approximately two to four times higher rates of scientific publication compared with publically funded registries, with adjusted rate ratios of 1.85 (95% confidence interval [CI] 1.07-3.22) and 4.18 (95% CI 2.54-6.87) for private not-for-profit and private for-profit funding, respectively. The inclusion of outcomes, use of pharmaceutical medicines, novel findings and citation rate for publications generated from patient registries with any private funding was not significantly different from those attributed to only publicly funded registries. E7766 nmr CONCLUSION The results of this study indicate that privately funded international rare disease patient registries produce significantly more evidence than their publicly funded counterparts. Examination of the quality indicators of these publications showed they were of the same high quality as those generated from publicly funded patient registry data.INTRODUCTION Methods for assessing the quality of herbal medicine preparations have advanced significantly in recent years in conjunction with increases in herbal medicine use and reports of adulteration and contamination. OBJECTIVE This study examined the quality of analgesic and anti-inflammatory herbal medicine preparations available on the Australian market by detecting the presence of listed ingredients, adulterants and contaminants. METHODS Forty-nine analgesic and anti-inflammatory herbal medicine preparations were randomly sourced from Australian capital cities. They were audited using a dual approach of liquid chromatography-mass spectrometry (LC-MS) combined with next-generation DNA sequencing. Once screened, a comparison of listed ingredients with verified ingredients was conducted to determine the accuracy of labelling, and the extent of adulteration and contamination. RESULTS Twenty-six of 49 (53%) herbal medicines were adulterated or contaminated with undeclared ingredients. LC-MS revealed the pan dispensaries supports the need for more stringent pharmacovigilance measures in Australia and abroad.The Taguchi method and metAFLP analysis were used to optimise barley regenerants towards maximum and minimum levels of tissue culture-induced variation. The subtle effects of symmetric and asymmetric methylation changes in regenerants were identified. Plant tissue cultures (PTCs) provide researchers with unique materials that accelerate the development of new breeding cultivars and facilitate studies on off-type regenerants. The emerging variability of regenerants derived from PTCs may have both genetic and epigenetic origins, and may be desirable or degrade the value of regenerated plants. Thus, it is crucial to determine how the PTC variation level can be controlled. The easiest way to manipulate total tissue culture-induced variation (TTCIV) is to utilise appropriate stress factors and suitable medium components. This study describes the optimisation of in vitro tissue culture-induced variation in plant regenerants derived from barley anther culture, and maximizes and minimizes regenerant variation compared with the source explants.