Pharmaceuticals Policy and Law - Volume 12, issue 1-2
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The new international review,
Pharmaceuticals Policy and Law, appears with the aim of studying and evaluating the
legal status of medicinal products in the European Union, and its implications in other markets such as the USA and Japan, without neglecting the specific problems of developing countries.
Pharmaceuticals Policy and Law intends to participate in the process of world convergence of pharmaceutical legislation helped by a network of academic centers specializing in pharmaceutical law, without omitting a scientific, economic and social approach to medicinal products.
The specificity of medicinal products conditions their legal status. Legislation regulating other goods cannot be applied to them. To begin with, they are the result of scientific and technical innovation. Research policies determine their progress. The pharmaceutical industry is, by nature, multinational. But, next to these global trends, different traditions still remain at a national level. Within the EU, barriers to free trade in medicinal products still remain despite more than thirty years of harmonisation. The social dimension of medicinal products is complex and very significant in the preoccupations of our societies. Patenting is essential but not sufficient. The life-cycle of medicinal products is protected by professional responsibility, required in the general concept of health safety. It is important to remember their ethical dimension, including research and innovation in new fields such as genetic manipulation and biotechnology, which requires social consent to preserve human dignity and fundamental rights.
Abstract: In the past, drug discovery developed in an environment where resources in healthcare where believed to be adequate, and all patients affected by a given disease where considered similar, therefore in need of a similar treatment. However, these concepts are no longer valid, and the present reality shows that current circumstances are characterised by limited financial resources in the presence of always growing expectations. In addition, current research trend is progressing toward the identification of novel…molecular targets and a personalised medicine approach.
Keywords: Innovation, R&D expenditure, risk-sharing, Italian Medicines Agency
Abstract: Key figures of the sector demonstrate that the pharmaceutical companies play a pivotal role in health research, in terms of resources and investments. Despite these efforts, only 1 substance out of 5–10.000 successfully passes the necessary tests, and 2 drugs out of 10 can defray R&D costs . This inevitably leads to the need of new R&D models which may include more focused clinical studies with e-designed mechanisms (Fig. 1) and open source protection of intellectual…property. Innovation is also expected to boost the research environment and the pharmaceutical industries are responding by outsourcing and buyout biotech companies, with investing only 20% in in-house research . In the Italian environment an integrated approach for the formation of a public-private network with pharmaceutical and biotech companies, public R&D institutions and universities, with patient and non-profit organisation is believed to be the key for success by offering the possibility to link internal excellence to the international research network.
Abstract: The mission of the Telethon Foundation is to boost research towards a therapy for muscular dystrophies and other genetic disorders, giving priority to those diseases neglected by public or private funding. Telethon operates in the Italian environment and has adopted a transparency policy on how donations are distributed to finance research.
Abstract: A public debate is currently ongoing on the perception that the licensing regulations tend to give precedence to the interests of drug companies with programs, such as the "fast-track" program, aimed at shortening review times and allowing approval at much earlier stages of clinical development in order to allow early access to new drugs. The main concern is that lower standards may become acceptable, with serious consequences to public health. To this end, NICE developed a…series of national clinical guidelines to secure consistent, high quality, evidence based assessment of clinical trial practice.
Abstract: To date, prescription of drugs which are unlicensed and/or unlabelled in the paediatric population is a concerning reality, particularly in the neonatal units (90%) and in the paediatric units (49%). Therefore, there is the need to develop effective and safe pharmacological treatments for the paediatric population in general.
Keywords: Paediatric population, clinical trial, orphan drug
Abstract: MolMed developed a TK cell based therapy to enable safe and effective haematopoietic stem cell transplantation (HSCT) from partially compatible donors (haplo-HSCT), currently in clinical development for high-risk leukaemia patients (AML and ALL). TK acquired the Orphan Drug status in EU (2003) and in US (2005).
Keywords: Haematopoietic stem cell transplantation, TK cell based therapy, leukaemia
Abstract: More than half (55%) of the drugs that received a positive opinion on orphan designation are for medical conditions affecting children (Fig. 1). Orphan drug designations for haematological conditions represent a substantial portion of all OD designations granted in the period 2000–2009 in EU. According to the EU regulation 141/2000 , all cancers, with the exception of breast, lung, colorectal, prostate and bladder, are rare diseases, with the consequence that all haematological diseases are…orphan diseases, with 70% of orphan designated conditions affecting less that 1 in 10,000 people. In the period 2000–2007 orphan designed medicines to treat oncological (17%) and haematological (33%) conditions have received more marketing authorisations from EC than those of any other therapeutic area.
Abstract: In 1970 the World Health Organization stated that the function of the clinical pharmacologist was: 1) to improve patient care by promoting safer and more effective use of drugs, 2) to increase knowledge through research, 3) to pass on knowledge through teaching and 4) to provide services (e.g., analyses, drug information, and advice in the design of experiments). The role of the clinical pharmacologist in the fields of drug discovery, drug development, and regulatory sciences has…significantly changed over the last decades. There is no discussion about the pivotal role of clinical pharmacologists in ensuring rational use of drugs, in translating pharmacological concepts from the bench to the clinic, in clinical drug development, or in teaching the discipline to medical and pharmacy students. Specifically, the role of paediatric clinical pharmacology is evolving and changing dramatically. This paper will summarize the current role of the (paediatric) clinical pharmacologist.
Keywords: Paediatric protocol, drug research, neonates, infants, paediatric clinical pharmacologist, pharmacokinetics, therapeutic drug monitoring, population pharmacokinetics, pharmacogenetics