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: The introduction of innovation into the drug development process is expected to bring to patients, including children, in the upcoming years a faster treatment, increased safety in term of side effects and tolerability, decreased interactions with other drugs, increased compliance, new treatments for untreated disorders, and increased quality of life and life duration. The implementation of the European Regulation EC/1901/2006 "Better Medicines for the Children of Europe" and EC/1902/06 foresaw the creation at the EMA of a…new Paediatric Committee and assigned it a number of tasks. The legal provision also forced Industry to develop their new drugs for children as soon as reasonably sound, as appropriate. An early paediatric investigation plan (PIP) is to be submitted to the PDCO as soon as the concept of the candidate medicinal product is sufficiently established in adults, at the end of phase I. The PIP has to be agreed by the PDCO, some room being left over for discussions, modifications and amendments whilst the candidate medicinal product will evolve along its life cycle.
Abstract: Rare diseases are frequently genetically determined and caused by an inborn metabolic error, thus occurring early in life and affecting normal growth, and sexual and CNS maturation. The development of a paediatric orphan medicine presents a real hurdle for pharmaceutical companies because of the nature of the patient population where little knowledge is available. In addition, clinical trials are more difficult to run, take longer and cost more. An overview of the current situation in the paediatric…orphan medicines field in Europe is provided below through the identification of unmet therapeutic needs still existing in the field (TEDDY NoE work), which represents a description of the orphan drugs so far authorised in Europe and of the paediatric drugs under development (Paediatric Investigation Plan – PIP).
Abstract: The development of medical devices for children is probably the next frontier in scientific and regulatory development. While some pediatric devices may have a potentially large market share, many do not and this makes them "orphans" in their own right. Recently the U.S. government has started to pay new attention to the area of pediatric device development. There are several challenges to paediatric device development and as, a consequence, it currently lags…5 to 10 years behind those for adults. Thus, many physicians and surgeons are forced to adapt adult devices in order to suit their paediatric patients. Challenges in paediatric device development reside in the fact that children differ from adults not only in terms of their size, but also because they are growing, so often a static device may not be adequate for a growing child. In addition, after a few months to years, an infant or child may outgrow a device and need a new one.
Abstract: The Paediatric Committee (PDCO) is a multidisciplinary setting composed by 5 CHMP members, 22 experts from the National Competent Authorities, 2 EEA, 3 patient/family's organisation representatives and 3 health professionals. The PDCO is accountable for providing opinion on PIPs, with deferrals/waivers and on compliance, and expert opinion on quality, safety and efficacy upon request by CHMP (marketing authorisations, scientific advice). In addition, PDCO is responsible for surveying paediatric needs, promoting…networking, and monitoring Eudra CT.
Keywords: Paediatric committee, paediatric investigation plan, paediatric working party
Abstract: In US three governance models related to funding type are available: grant: Investigator led with funding organization oversight; cooperative agreement: partnership between investigators and funding organisation; contract: investigators provide services to funding organization. Hybrid funding models are also plausible and functional.
Abstract: Pediatric rheumatic diseases (PRD) encompass an heterogeneous group of rare diseases, ranging from juvenile idiopathic arthritis (JIA), juvenile systemic lupus erythematosus (JSLE) to juvenile dermatomyositis (JDM) and others. The PRD constitute a small market which is of little interest for pharmaceutical industries. Difficulties in operating in this specific area relate to problems which are peculiar to children, such as specific methods for the assessment of the primary outcome, or paediatric formulations, with the…need of setting large networks in order to collect in a reasonable time frame an adequate number of patients.
Abstract: Childhood cancer affects 12,000 new patients/year in Europe with more than 60 different malignancies. To date, the overall cure rate at 5 years is around 80% in Europe  and U.S. , with a powerful increase as compared to the cure rates accessible 15 years ago. Although cancer in children is a rare condition, being only 1% of all cancers, it remains the first cause of death in children older than 1 year; still, 3,000 deaths are reported every…year. Accordingly, paediatric cancer still represents a therapeutic need and new efficient treatments must be developed, regardless the present cure rates allow to figure out that one adult aged from 16 to 45 years out of 900 individuals has been cured by cancer during childhood .
Abstract: The Ministry of Health, Labour and Welfare (MHLW) and related government agencies have been implementing strategies to facilitate clinical trials in the last few years , and the infrastructure for paediatric clinical trials is being strengthened. The revision of the Pharmaceutical Affairs Law (PAL) in 2002 has enabled physicians to conduct sponsor-investigator trials in Japan. There have been 9 protocols for 6 drugs funded by the MHLW in children completed or ongoing as of…August, 2009. The National Centre for Child Health and Development (NCCHD) has been involved in 7 of these protocols. Of these 7 protocols, NCCHD has been involved in project management for 5 protocols.
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
Abstract: Modern paediatric intensive care became organized in Europe in 1970s. Since that, time medical and technological advances resulted in a significant improvement of the treatment for infants, children and adolescents admitted to the Paediatric Intensive Care Units (PICUs) with a wide range of medical and surgical conditions. Now numerous conditions that were previously considered fatal have become treatable with an overall survival rate of 85–98%. However, the profile change of patients and the appearance of new…conditions (CLD, VAP, VILI) have caused new problems, together with the consideration that 90% of medicinal products used in PICUs are still unauthorised or administered off-label. PICU constitutes a unique setting for pharmacotherapy, as the patient is often characterised by respiratory or multiorgan failure, with an underlying pathology, and for which drug interaction, mode of administration and organ maturation are not properly taken into account.