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

Authors: Brasseur, Daniel

Article Type: Research Article

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. Show more

Keywords: Innovation, PIP, paediatric, PDCO

DOI: 10.3233/PPL-2010-0273

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 81-83, 2010

Authors: Catapano, Mariana | Folgori, Laura

Article Type: Research Article

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). Show more

Keywords: Paediatric, orphan, rare disease, TEDDY

DOI: 10.3233/PPL-2010-0274

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 85-88, 2010

Authors: Ulrich, Linda C.

Article Type: Research Article

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. Show more

Keywords: Device, paediatric, FDAAA, HDE

DOI: 10.3233/PPL-2010-0275

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 89-91, 2010

Authors: Rossi, Paolo | Scarselli, Alessia

Article Type: Research Article

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. Show more

Keywords: Paediatric committee, paediatric investigation plan, paediatric working party

DOI: 10.3233/PPL-2010-0276

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 93-95, 2010

Authors: Hirschfeld, Steven | Shriver,

Article Type: Research Article

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.

Keywords: Funding, US, cancer, NICHD

DOI: 10.3233/PPL-2010-0277

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 97-99, 2010

Authors: Jacqz-Aigrain, Evelyne

Article Type: Research Article

DOI: 10.3233/PPL-2010-0278

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 101-103, 2010

Authors: Ruperto, Nicolino

Article Type: Research Article

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. Show more

Keywords: Paediatric, rheumatic disease, PRINTO, JIA, JDM, JSLE

DOI: 10.3233/PPL-2010-0279

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 105-107, 2010

Authors: Paolucci, Paolo | Cioni, Vera | Caramaschi, Elisa | Cano, Carmen

Article Type: Research Article

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 [1] and U.S. [2], 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 [3]. Show more

Keywords: Cancer, children, clinical trials, chemotherapy

DOI: 10.3233/PPL-2010-0280

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 109-113, 2010

Authors: Giaquinto, Carlo

Article Type: Research Article

Keywords: PENTA, paediatric, AIDS, clinical trial

DOI: 10.3233/PPL-2010-0281

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 115-116, 2010

Authors: Nakamura, Hidefumi

Article Type: Research Article

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 [1], 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. Show more

Keywords: Pharmaceutical Affairs Law, Japan, NCCHD, incentives, paediatrics

DOI: 10.3233/PPL-2010-0282

Citation: Pharmaceuticals, Policy and Law, vol. 12, no. 1-2, pp. 117-121, 2010