Evaluating health policy and legal responses : how to reduce barriers and improve access to orphan drugs for rare diseases in Canada

Abstract : Rare diseases are debilitating conditions often leading to severe clinical manifestations for affected patients. Orphan drugs have been developed to treat these rare diseases affecting a small number of individuals. Incentives in the legal framework aimed to recoup the research and development cost of orphan drugs for pharmaceutical companies have been implemented in the United States and the European Union. At the present time, Canada is still lacking a legal and policy framework for orphan drugs. Several problems at the federal and provincial levels remain: lack of research funds for rare diseases, discrepancies on orphan drug policies between provinces, difficulties to access and reimburse these high price drugs. Recommendations and measures are proposed, such as a pan-Canadian (national) scientific committee to establish evidence-based guidelines for patients to access orphan drugs uniformly in all provinces with a disease specific registry, a formal agreement for a centralized Canadian public funding reimbursement procedure, and increasing the role of “guardian” for prices by the Patented Medicines Review Board in Canada. These recommendations and measures will be beneficial for the implementation of a policy framework for orphan drugs in Canada.

DIY Archives: Enhancing Access to Collections via Free, Open-Source Platforms

Presentation from the MARAC conference in Boston, MA on March 18-21, 2015. S. 24 - DIY Archives: Enhancing Access to Collections via Free, Open-Source Platforms

A contribution to knowledge of the biodiversity of Syrphidae (Diptera) in Spain

Data are provided on two hoverfly species new to the Iberian Peninsula, Brachyopa grunewaldensis Kassebeer and Criorhina floccosa (Meigen), and one new to Spain, Eumerus consimilis Šimić & Vujić. New habitat and breeding data are presented.

The importance of organizational commitment to knowledge management

In recent years, Knowledge Management (KM) has assumed great importance in the literature on business and management. However, we still have so little understanding of the human issues in KM processes. Thus, this research aims to contribute to analysing the importance of Organizational Commitment (OC) to KM. First, we used the Cardoso (2003) Knowledge Management Questionnaire (QGC) that embraces all organizational activities around knowledge processes and distinguishes four dimensions of KM. Secondly we applied the Quijano, Masip, Navarro and Aubert (1997) questionnaire (ASH-ICI) that distinguishes two types of commitment (personal and instrumental) into four dimensions. These two questionnaires were applied with 300 employees in the Portuguese industrial ceramics sector. Through multiple regression analysis we found that levels of organizational commitment are statistically important to KM dimensions. Furthermore, our analysis indicates that personal commitment is more important than need commitment. These results are discussed and Organizational Behaviour specialists and Work and Organizational psychologists are challenged to assume more responsibility and an active role in KM studies and practices and to explore human issues in this field.

Impact of dental caries on quality of life of adolescents according to access to oral health services: a cross sectional study

Aim: This study presents the prevalence of dental caries and its relation to the quality of life of adolescents according to the access to dental health services. Methods: Two hundred and fifty-six adolescents between 15 and 19 years of age participated in the study; they were all enrolled in public schools in a countryside municipality of the São Paulo State. Data related to dental caries were evaluated by the DMFT Index, and OHIP-14 was used for evaluating the quality of life. Mann Whitney and Spearmann correlation tests were also used (p<0.05). Results: A DMFT of 3.09 (±3.30) was found with a higher prevalence among the adolescents who used public dental services (3.43±3.34) compared with those who used private services (2.94±3.28). A statistically significant relationship between the decay component of DMFT with physical pain (0.020), physical disability (0.002) and quality of life (0.017) was verified. Conclusions: A low prevalence of dental caries was observed, and it was higher in adolescents who used public oral health services rather than private ones, evidencing the low influence of oral health on the quality of life of the participants.

Intersubjectivity and sentiment:from language to knowledge

Intersubjectivity is an important concept in psychology and sociology. It refers to sharing conceptualizations through social interactions in a community and using such shared conceptualization as a resource to interpret things that happen in everyday life. In this work, we make use of intersubjectivity as the basis to model shared stance and subjectivity for sentiment analysis. We construct an intersubjectivity network which links review writers, terms they used, as well as the polarities of the terms. Based on this network model, we propose a method to learn writer embeddings which are subsequently incorporated into a convolutional neural network for sentiment analysis. Evaluations on the IMDB, Yelp 2013 and Yelp 2014 datasets show that the proposed approach has achieved the state-of-the-art performance.

Adenine as an organocatalyst for the ring-opening polymerization of lactide: scope, mechanism and access to adenine-functionalized polylactide

Nucleobase-functionalized polymers are widely used in the fields of supramolecular chemistry and self-assembly, and their development for biomedical applications is also an area of interest. They are usually synthesized by tedious multistep procedures. In this study, we assess adenine as an organoinitiator/ organocatalyst for the ring-opening polymerization of lactide. L-Lactide can be quantitatively polymerized in the presence of adenine. Reaction conditions involving short reaction times and relatively low temperatures enable the access to adenine end-capped polylactide in a simple one-step procedure, in bulk, without additional catalyst. DFT calculations show that the polymerization occurs via hydrogen bond catalysis. The mechanism involves (i) a hydrogen bond between the NH9 of adenine and the carbonyl moiety of lactide, leading to an electron deficient carbon atom, and (ii) a second hydrogen bond between the N3 of adenine and the NH2 of a second adenine molecule, followed by a nucleophilic attack of the latter activated amine on the former electron deficient carbon on the monomer. For longer reaction times and higher temperatures, macrocyclic species are formed, and a mechanism involving the imidazole ring of adenine is proposed based on literature studies. Depending on the reaction conditions, adenine can thus be considered as an organoinitiator or an organocatalyst for the ring-opening polymerization of lactide.

Adenine as an organocatalyst for the ring-opening polymerization of lactide: scope, mechanism and access to adenine-functionalized polylactide

Nucleobase-functionalized polymers are widely used in the fields of supramolecular chemistry and self-assembly, and their development for biomedical applications is also an area of interest. They are usually synthesized by tedious multistep procedures. In this study, we assess adenine as an organoinitiator/organocatalyst for the ring-opening polymerization of lactide. L-Lactide can be quantitatively polymerized in the presence of adenine. Reaction conditions involving short reaction times and relatively low temperatures enable the access to adenine end-capped polylactide in a simple one-step procedure, in bulk, without additional catalyst. DFT calculations show that the polymerization occurs via hydrogen bond catalysis. The mechanism involves (i) a hydrogen bond between the NH9 of adenine and the carbonyl moiety of lactide, leading to an electron deficient carbon atom, and (ii) a second hydrogen bond between the N3 of adenine and the NH2 of a second adenine molecule, followed by a nucleophilic attack of the latter activated amine on the former electron deficient carbon on the monomer. For longer reaction times and higher temperatures, macrocyclic species are formed, and a mechanism involving the imidazole ring of adenine is proposed based on literature studies. Depending on the reaction conditions, adenine can thus be considered as an organoinitiator or an organocatalyst for the ring-opening polymerization of lactide.

Developing capabilities for ongoing learning and change in intermediary programs

In dynamic environments, firms seek to build capabilities which will permit them to become innovation and change ready. Programs offered by intermediaries, while varying greatly in content and format, are designed to support those firms wishing to enhance their competitiveness. Firms which participate in intermediary programs have displayed their willingness to overcome deficiencies or barriers to competitiveness through acquiring knowledge which is external to the firm. This paper reports on interviews with 24 firms who were involved in a MAP or TAP program offered by QMI Solutions. The findings of the research suggest that knowledge intermediaries serve to disrupt organisational paths and in so doing establish mechanisms for ongoing learning and change. They do this first by disrupting the firm with a positive learning experience and also by establishing processes for developing new relationships and access to knowledge which are critical for learning and change. It is the experience of learning through knowledge exchange which can trigger the pursuit of new paths and it is the processes involving new relations and knowledge processing that provides the micro-foundations for ongoing learning and change. This suggests that the role of intermediaries goes well beyond merely knowledge transfer to include longer term effects on the capability of organisations to innovate, which is critical to economic competitiveness and the survival rate of firms.

A University Support Model for eResearch

Queensland University of Technology (QUT) is faced with a rapidly growing research agenda built upon a strategic research capacity-building program. This presentation will outline the results of a project that has recently investigated QUT’s research support requirements and which has developed a model for the support of eResearch across the university. QUT’s research building strategy has produced growth at the faculty level and within its research institutes. This increased research activity is pushing the need for university-wide eResearch platforms capable of providing infrastructure and support in areas such as collaboration, data, networking, authentication and authorisation, workflows and the grid. One of the driving forces behind the investigation is data-centric nature of modern research. It is now critical that researchers have access to supported infrastructure that allows the collection, analysis, aggregation and sharing of large data volumes for exploration and mining in order to gain new insights and to generate new knowledge. However, recent surveys into current research data management practices by the Australian Partnership for Sustainable Repositories (APSR) and by QUT itself, has revealed serious shortcomings in areas such as research data management, especially its long term maintenance for reuse and authoritative evidence of research findings. While these internal university pressures are building, at the same time there are external pressures that are magnifying them. For example, recent compliance guidelines from bodies such as the ARC, and NHMRC and Universities Australia indicate that institutions need to provide facilities for the safe and secure storage of research data along with a surrounding set of policies, on its retention, ownership and accessibility. The newly formed Australian National Data Service (ANDS) is developing strategies and guidelines for research data management and research institutions are a central focus, responsible for managing and storing institutional data on platforms that can be federated nationally and internationally for wider use. For some time QUT has recognised the importance of eResearch and has been active in a number of related areas: ePrints to digitally publish research papers, grid computing portals and workflows, institutional-wide provisioning and authentication systems, and legal protocols for copyright management. QUT also has two widely recognised centres focused on fundamental research into eResearch itself: The OAK LAW project (Open Access to Knowledge) which focuses upon legal issues relating eResearch and the Microsoft QUT eResearch Centre whose goal is to accelerate scientific research discovery, through new smart software. In order to better harness all of these resources and improve research outcomes, the university recently established a project to investigate how it might better organise the support of eResearch. This presentation will outline the project outcomes, which include a flexible and sustainable eResearch support service model addressing short and longer term research needs, identification of resource requirements required to establish and sustain the service, and the development of research data management policies and implementation plans.

Survey of eResearch practices and skills at QUT, Australia

Queensland University of Technology (QUT) is a multidisciplinary university in Brisbane, Queensland, Australia, and has 40,000 students and 1,700 researchers. Notable eResearch infrastructure includes the QUT ePrints repository, Microsoft QUT Research Centre, the OAK (Open Access to Knowledge) Law Project, Cambia and leading research institutes. ---------- The Australian Government, via the Australian National Data Service (ANDS), is funding institutions to identify and describe their research datasets, to develop and populate data repositories and collaborative infrastructure, and to seed the Australian Research Data Commons. QUT is currently broadening its range of research support services, including those to support the management of research data, in recognition of the value of these datasets as products of the research process, and in order to maximize the potential for reuse. QUT is integrating Library and High Performance Computing (HPC) services to achieve its research support goals. ---------- The Library and HPC released an online survey using Key Survey to 1,700 researchers in September 2009. A comprehensive range of eResearch practices and skills was presented for response, and grouped into areas of scholarly communication and open access publishing, using collaborative technologies, data management, data collection and management, computation and visualization tools. Researchers were asked to rate their skill level on each practice. 254 responses were received over two weeks. Eight focus groups were also held with 35 higher degree research (HDR) students and staff to provide additional qualitative feedback. A similar survey was released to 100 support staff and 73 responses were received.---------- Preliminary results from the researcher survey and focus groups indicate a gap between current eResearch practices, and the potential for researchers to engage in eResearch practices. Researchers are more likely to seek advice from their peers, than from support staff. HDR students are more positive about eResearch practices and are more willing to learn new ways of conducting research. An account of the survey methodology, the results obtained, and proposed strategies to embed eResearch practices and skills across and within the research disciplines will be provided.

Towards a relational theory of copyright law

This research provides a systematic and theoretical analysis of the digital challenges to the established exclusive regime of the economic rights enjoyed by authors (and related rightholders) under the law of copyright. Accordingly, this research has developed a relational theory of authorship and a relational approach to copyright, contending that the regulatory emphasis of copyright law should focus on the facilitation of the dynamic relations between the culture, the creators, the future creators, the users and the public, rather than the allocation of resources in a static world. In this networked digital world, the creative works and contents have become increasingly vital for people to engage in creativity and cultural innovation, and for the evolution of the economy. Hence, it is argued that today copyright owners, as content holders, have certain obligations to make their works accessible and available to the public under fair conditions. This research sets forward a number of recommendations for the reform of the current copyright system.