Journal Journal of Medicines Development Sciences

29 articles
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Franck Atienzar, Annie Delaunois, Frédéric Brouta, Miranda Cornet, Renaud Fleurance, Helga Gerets, Stephanie Glineur, Catrin Hasselgren, Andrea Kiessling, Andre Nogueira Da Costa, et al.
Journal of Medicines Development Sciences, Volume 2, pp 2-29; doi:10.18063/jmds.2016.01.001

Abstract:Understanding and reducing attrition rate remains a key challenge in drug development. Preclinical and clinical safety issues still represent about 40% of drug discontinuation, of which cardiac and liver toxicities are the leading reasons. Reducing attrition rate can be achieved by various means, starting with a comprehensive evaluation of the potential safety issues associated to the primary target followed by an evaluation of undesirable secondary targets. To address these risks, a risk mitigation plan should be built at very early development stages, using a panel of in silico, in vitro, and in vivo models. While most pharmaceutical companies have developed robust safety strategies to de-risk genotoxicity and cardiotoxicity issues, partly driven by regulatory requirements; safety issues affecting other organs or systems, such as the central nervous system, liver, kidney, or gastro-intestinal system are less commonly addressed during early drug development. This paper proposes some de-risking strategies that can be applied to these target organ systems, including the use of novel biomarkers that can be easily integrated in both preclinical and clinical studies. Experiments to understand the mechanisms’ underlying toxicity are also important. Two examples are provided to demonstrate how such mechanistic studies can impact drug development. Novel trends in investigative safety are reviewed, such as computational modeling, mitochondrial toxicity assessment, and imaging technologies. Ultimately, understanding the predictive value of non-clinical safety testing and its translatability to humans will enable to optimize assays in order to address the key objectives of the drug discovery process, i.e., hazard identification, risk assessment, and mitigation.
Anita Aperia, Jorgen Dirach, Mike Hardman, Christa Janko, Jeff Kipling, Rosan Kreeftmeijer-Vegter, Rebecca Ludwig, Lena Scott, Armel Stockis
Journal of Medicines Development Sciences, Volume 3; doi:10.18063/jmds.v3i1.163

Abstract:“No research without trained researchers” has become the mantra of the EU-funded Innovative Medicines Initiative (IMI) education and training projects. However, it is often hard to determine the type of training required at different stages of a scientist’s career. The situation is further complicated by the constantly changing environment, e.g. the growth of disruptive technologies, societal expectations of biomedical sciences, the greater need for multi-disciplinary collaborations, and conservative or changing regulatory requirements. This article summarises the experience from a series of five EMTRAIN Public Private Partnership PhD workshops that included both scientific and transferrable skill training. This is followed by an example of a recently developed training programme, including a competency profile, for translational research and medicines development; the C-COMEND teaching programme. The emphasis is on competencies as a new currency for continuing professional development. Finally, this paper describes what we consider to be the next steps required by the scientific community to address solutions to the current training challenges so that society can benefit from the innovations that only science can provide.
Victoria Elegant
Journal of Medicines Development Sciences, Volume 2, pp 43-50; doi:10.18063/jmds.2016.01.004

Abstract:The Asia Pacific region is an extremely diverse region, characterized by heterogeneity from a number of aspects, including culture, religion, economics, landscapes, and languages. This also applies to the standard of medical care and the regulatory requirements for approval of drugs in the region. Developed economies such as Japan and Australia have requirements which are not dissimilar to those of the EU and USA, but still have their own unique requirements. The developing economies all have their own requirements. In the ASEAN region there is harmonization of the dossier format, but each country still has local requirements. The region has seen significant growth in clinical trial activity, both to satisfy local registration and safety requirements and to help accelerate global trial patient recruitment. There is a clear need for training in all aspects of medical, regulatory, clinical and safety aspects of medicines development, which is being addressed through several organizations and at different locations in the region.
Dipak Kalra, Mats Sundgren, Brecht Claerhout, Pierre Meulien, Bart Vannieuwenhuyse, Peter Singleton, Terje Peetso, Veli Stroetmann, Petra Wilson, Mary Baker, et al.
Journal of Medicines Development Sciences, Volume 3; doi:10.18063/jmds.v3i1.165

Abstract:The European Institute for Innovation through Health Data (i~HD) has been formed as one of the sustainable entities arising from the Electronic Health Records for Clinical Research (EHR4CR) and SemanticHealthNet projects, in collaboration with other European Commission projects and initiatives. The vision of i~HD is to become the European organisation of reference for guiding and catalysing the best, most efficient and trustworthy uses of health data and interoperability, for optimizing health and knowledge discovery.i~HD has been established in recognition that there is a need to tackle areas of challenge in the successful scaling up of innovations that rely on high-quality and interoperable health data, to sustain and propagate the results of eHealth research, and to address current-day obstacles to using health data. i~HD was launched at an inaugural conference in Paris, in March 2016. This was attended by over 200 European clinicians, healthcare providers and researchers, representatives of the pharma industry, patient associations, health professional associations, the health ICT industry and standards bodies. The event showcased issues and approaches, that are presented in this paper to highlight the activities that i~HD intends to pursue as enablers of the better uses of health data, for care and research.
Dorothée Cammarata, Abduelhakem Ben Addi, Eva D’Amico, Bernard Robaye
Journal of Medicines Development Sciences, Volume 2, pp 30-37; doi:10.18063/jmds.2016.01.002

Abstract:Numerous studies have demonstrated the role of uridine diphosphate (UDP) and its P2Y6 receptor in the inflammatory reaction and innate immunity. However, the importance of the P2Y6 receptor in the adaptive immune response remains unclear. In this study, we demonstrate that the P2Y6 receptor is functionally expressed in murine bone marrow dendritic cells (BMDC). UDP induced a Ca2+ transient in these cells that was decreased in P2Y6-deficient mice. UDP also increased the endocytosis of fluorescein isothiocyanate-dextran (FITC-dextran) and amplified the secretion of interleukin 12-p70 (IL-12p70) induced by CpG; these responses were abolished in P2Y6-deficient mice. In vivo experiments showed that the serum level of specific IgG2c after immunisation with ovalbumin was decreased in P2Y6-deficient mice, while the level of specific IgG1 was unchanged. These data suggest that the P2Y6-mediated effects of UDP on myeloid dendritic cells play a role in the in vivo Th1 skewing of the immune response.
Alan Boyd, Peter Stonier, Konrad Obiora, Ben Cottam
Journal of Medicines Development Sciences, Volume 3; doi:10.18063/jmds.v3i1.193

Abstract:The Faculty of Pharmaceutical Medicine was established in the United Kingdom (UK) in 1989 to develop and maintain competence, ethics and the highest professional standards of practice in the discipline. This article charts the founding principles and history of the Faculty, details its composition and membership, and outlines the major functions and achievements of the organisation in the context of the development of the discipline of pharmaceutical medicine. The recognition of pharmaceutical medicine as a medical specialty in the UK is described together with the associated certification programme Pharmaceutical Medicine Specialty Training, which enables pharmaceutical physicians to become certified practitioners in the specialty with the UK General Medical Council (UK GMC).
Ana Paula Ruenis, João Massud Filho
Journal of Medicines Development Sciences, Volume 2, pp 51-54; doi:10.18063/jmds.2016.01.005

Abstract:Brazil is one of the world´s largest economies and pharmaceutical markets, having the Brazilian government as an important purchaser. There are strong local companies that have grown sustainably after the introduction of generics and are investing in both incremental and radical innovation. However, research and development (R&D) expenditures are still modest; this could be explained by a combination of economic and political uncertainty in the past few years and a bureaucratic, complex regulatory framework. New regulations, efforts to reduce ethical and regulatory review timelines, and a Senate bill aimed to accomplish that goal should constitute the definitive regulatory landmark for boosting clinical research. In addition to government investments they have given a breath of relief in the market, as Brazil is trying to, once again, gain momentum as a “must-go” country for clinical development. Non-profit associations such as the Brazilian Society of Pharmaceutical Medicine (Sociedade Brasileira de Medicina Farmacêutica-SBMF), the Brazilian Association of CROs (Associação Brasileira de Organizações Representativas de Pesquisa Clínica-ABRACRO), the Brazilian Clinical Research Alliance (Aliança Pesquisa Clínica Brasil), amongst others, helped to give the impulse to trigger such changes. It is time to invest heavily in developing educational programs to address the growing need for clinical development scientists and physicians.
Akiko Kishi, Ichiro Uchida, Daisuke Koide, Kyoko Imamura
Journal of Medicines Development Sciences, Volume 3; doi:10.18063/jmds.v3i1.166

Abstract:Development of new medicines has become increasingly difficult with less possibility of success in seeds-finding and ever rising operational costs. Failure to comply with ethical standards for human research protection also erodes social trust in clinical development. In order to develop competence of professionals in medicines development such as clinical investigators and drug development scientists, a variety of educational courses and training programs have been developed and executed worldwide. As Japan is no exception and shares the same concerns, significant governmental and non-governmental efforts have been made to invest in the development of academic educational courses and adherence to international standards. This article introduces examples of the adoption of technologies to realize a user-friendly and sustainable learning management as well as an adaptation of syllabuses and core curricula to meet international standards in the era of global medicines development.
Deborah Chee, Minsoo Park, Sylvie Kim, Ji-Hoon Sohn
Journal of Medicines Development Sciences, Volume 1; doi:10.18063/jmds.2015.02.003

Abstract:Korea has continuously sought to improve its regulatory environment for clinical trials and has invested heavily in clinical trial infrastructure and technology since the early 2000’s. A strategic investment through the Korea National Enterprise for Clinical Trials (KoNECT) program began in 2007 and grew to encompass a network of regional clinical trial centers to promote clinical trial capabilities and human resource development. In early 2014, KoNECT became a permanent organization focused on the advancement of the country's clinical trial industry. This was followed by the establishment of the Korea Clinical Trials Global Initiative (KCGI) and the KoNECT Collaboration Center for global clinical trials (KCC). KCGI and KCC are now at the forefront of KoNECT’s efforts to promote higher operational efficiency in the country’s clinical trials. These new initiatives in clinical research are undertaking multichannel approaches to pursue a cohesive international collaboration model between government, industry and academia for the development of new treatments and improved patient care.
Sabrina Lucia Ena, Julia Ino, Aurelie Neirinck, Sandra Magali Pietri, Anna Tury, Enrico Bastianelli
Journal of Medicines Development Sciences, Volume 1; doi:10.18063/jmds.2015.02.004

Abstract:Over the last decade, there has been an increasing interest among researchers for human mesenchymal stromal cells (MSC). Their regenerative properties, multilineage differentiation capacity and immunomodulatory properties make them promising candidates for treatment in various conditions. Emerging biotechnology companies specialized in cellular and regenerative therapies have been focusing their interest on MSC-based therapies, and their use in clinical trials has steadily increased. Notably, MSC are currently tested in clinical trials addressing unmet medical needs in the field of bone fracture repair and more specifically in non-union and delayed union fractures where the bone repair process is impaired. Although MSC can be isolated from various tissues, the most commonly studied sources are bone marrow (BM) and adipose tissue (Ad). In this article, we reviewed the literature directly comparing BM- and Ad-MSC for their in vitro characteristics and in vivo osteogenic potential to determine which source of MSC would be more appropriate for bone fracture repair. As considerable variations in experimental settings between studies were found, our review was based on studies meeting specific sets of criteria, notably regarding donors’ age and gender. This review of side-by-side comparisons suggests that while BM-and Ad-MSC share common general characteristics, BM-MSC have a higher intrinsic osteogenic capacity in vitro and bone repair potential in vivo.
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