Pharmaceuticals Policy and Law - Volume 12, issue 1-2

Authors: Rasi, Guido

Article Type: Research Article

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 However, these concepts are no longer valid, and the present reality shows that current circumstances are characterised by limited financial resources in the presence of always growing expectations.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. Show more

Keywords: Innovation, R&D expenditure, risk-sharing, Italian Medicines Agency

DOI: 10.3233/PPL-2010-0283

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 125-127, 2010

Authors: Dompé, Sergio

Article Type: Research Article

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 [1]. 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 [2]. 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. Show more

Keywords: R&D, advanced therapies, biotech products

DOI: 10.3233/PPL-2010-0284

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 129-131, 2010

Authors: Paganin, Carla

Article Type: Research Article

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.

Keywords: Telethon, genetic disorders, ADA-SCID, gene therapy

DOI: 10.3233/PPL-2010-0285

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 133-135, 2010

Authors: Tomino, Carlo

Article Type: Research Article

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. Show more

Keywords: AIFA, register, risk-sharing

DOI: 10.3233/PPL-2010-0286

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 137-138, 2010

Authors: Camozzi, Carlos R.

Article Type: Research Article

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

DOI: 10.3233/PPL-2010-0287

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 139-141, 2010

Authors: Dieci, Marco

Article Type: Research Article

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

DOI: 10.3233/PPL-2010-0288

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 143-145, 2010

Authors: Siani-Rose, Michael

Article Type: Research Article

Abstract: Anginera™ is a bioresorbable scaffold-based, three dimensional human dermal fibroblast culture developed for the treatment of diffuse small vessel disease in human hearts.

Keywords: Anginera™, fibroblast, small vessel disease

DOI: 10.3233/PPL-2010-0289

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 147-149, 2010

Authors: Maggio, A. | Acuto, S. | Moi, P. | Galanello, R. | Comoli, P. | Locatelli, F. | Motta, V. | Pecoraro, A. | Di Marzo, R. | Ferro, L. | Sadelain, M.

Article Type: Research Article

DOI: 10.3233/PPL-2010-0290

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 151-155, 2010

Authors: Zanon, Paola

Article Type: Research Article

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 [1], 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. Show more

Keywords: Haematology, cancer, rare disease, orphan

DOI: 10.3233/PPL-2010-0292

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 157-159, 2010

Authors: van den Anker, John N.

Article Type: Research Article

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. Show more

Keywords: Paediatric protocol, drug research, neonates, infants, paediatric clinical pharmacologist, pharmacokinetics, therapeutic drug monitoring, population pharmacokinetics, pharmacogenetics

DOI: 10.3233/PPL-2010-0293

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 161-165, 2010