Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance

2020 ◽  
Vol 27 (3) ◽  
pp. 213-231
Author(s):  
Annagrazia Altavilla ◽  
Viviana Giannuzzi ◽  
Mariangela Lupo ◽  
Donato Bonifazi ◽  
Adriana Ceci
Keyword(s):  
Translational Research ◽  
Research Infrastructure ◽  
Paediatric Research ◽  
Adequate Model ◽  
Regulatory Issues ◽  
Societal Issues ◽  
Translational Approach ◽  
Legal Frameworks ◽  
Ethical Oversight ◽  
Paediatric Medicines

Abstract The lack of paediatric medicines, including innovative and advanced ones, is a long-lasting and well-known problem at European and international levels. Despite the existing legal frameworks and incentives, children remain deprived of many kinds of therapy because of challenges faced in appropriately study and tailoring medicinal and other products for them. In this context, the necessity to foster paediatric research addressing unsolved and uncovered issues within a ‘translational approach’ has appeared. This article, after having clarified the concept of translational research in the perspective of the establishment of a European paediatric research infrastructure (RI), will identify and point out ethical, legal and regulatory issues particularly relevant in a children’s rights perspective. It concludes asking for the setting up of an adequate model of governance within a future RI, including adequate and independent ethical oversight and a pluridisciplinary common service dealing with ethical, legal and societal issues relevant for children.

Translational research will fail without surgical leadership: SCOTRRCC a successful surgeon-led Nationwide translational research infrastructure in renal cancer

The Surgeon ◽  
2015 ◽  
Vol 13 (4) ◽  
pp. 181-186 ◽  
Author(s):  
Grant D. Stewart ◽  
Antony C.P. Riddick ◽  
Frances Rae ◽  
Craig Marshall ◽  
Linda MacLeod ◽  
...  
Keyword(s):  
Translational Research ◽  
Renal Cancer ◽  
Research Infrastructure

Paediatric Medicines: Regulatory and Scientific Issues

Drug Research ◽  
2017 ◽  
Vol 67 (07) ◽  
pp. 377-384 ◽  
Author(s):  
Chrysa Daousani ◽  
Vangelis Karalis
Keyword(s):  
Research Network ◽  
Screening Methods ◽  
Paediatric Research ◽  
Treatment Needs ◽  
Paediatric Drug ◽  
Drug Products ◽  
Accurate Dose ◽  
Paediatric Drug Development ◽  
Set Up ◽  
Paediatric Medicines

AbstractIn the past, dosage regimens authorized for adults were extrapolated to children relying mainly on empirical dosage adjustments. However, children are not small adults, but a distinct and heterogeneous group in terms of physiology, disease occurrence, pharmacokinetics, pharmacodynamics and also psychological, cognitive, and behavioral aspects. Even though it would be helpful to know the physiological changes and the special drug treatment needs in children, this task could not be performed due to ethical reasons. Important issues to consider for the development of paediatric drug products refer to the administration of the accurate dose, the use of the appropriate excipients, and acceptability. The latter is crucial and taste-screening methods (like electronic tongues) have been developed. A new era in paediatric medicines started with the entry into force of paediatric regulations. In the early ‘80s, the FDA started the set-up of a regulatory framework by authorizing issues like the Paediatric Rule, the Best Pharmaceuticals for Children Act, the Paediatric Research Equity Act, and the Food and Drug Administration Safety and Innovation Act. Similar efforts have been made in the EU, mainly through the entry into force of the Paediatric Regulation and the establishment of the Paediatric Committee, the Paediatric Investigation Plan, the Paediatric Use Marketing Authorization, and the European Paediatric Research Network. Other efforts to bridge the gap, between knowledge in adults and the children’s special requirements, include the extrapolation concept of safety/efficacy aspects, the application of modeling/simulation approaches in paediatric drug development, and the development of a paediatric Biopharmaceutics Classification Scheme.

scholarly journals EPTRI – European Paediatric Translational Research Infrastructure. Bridging the gaps of the paediatric excellence medicine

2019 ◽  
Vol 4 (1) ◽  
Author(s):  
Adriana Ceci ◽  
Catherine Tuleu ◽  
Donato Bonifazi ◽  
Hana Kubova ◽  
Irmgard Toni ◽  
...  
Keyword(s):  
Translational Research ◽  
Research Infrastructure ◽  
European Paediatric

scholarly journals Cancer Core Europe: A translational research infrastructure for a European mission on cancer

2019 ◽  
Vol 13 (3) ◽  
pp. 521-527 ◽  
Author(s):  
Alexander M. M. Eggermont ◽  
Giovanni Apolone ◽  
Michael Baumann ◽  
Carlos Caldas ◽  
Julio E. Celis ◽  
...  
Keyword(s):  
Translational Research ◽  
Research Infrastructure

scholarly journals Biobanking and biomolecular resource research infrastructure: The Italian node

2020 ◽  
Author(s):  
Barbara Parodi
Keyword(s):  
Quality Management ◽  
Biomedical Research ◽  
Research Infrastructure ◽  
European Research ◽  
Societal Issues ◽  
Online Tools ◽  
New Treatments ◽  
Resource Research ◽  
Management Services

BBMRI-ERIC is a European research infrastructure for biobanking, bringing together all the main players from the biobanking field – researchers, biobankers, industry, and patients – to boost biomedical research. To that end, it offers quality management services, support with ethical, legal and societal issues, and a number of online tools and software solutions. Ultimately, the goal of BBMRI-ERIC and its National Nodes is to make new treatments possible.

scholarly journals Children’s Preferences for Oral Dosage Forms and Their Involvement in Formulation Research via EPTRI (European Paediatric Translational Research Infrastructure)

Pharmaceutics ◽  
2021 ◽  
Vol 13 (5) ◽  
pp. 730
Author(s):  
Elisa Alessandrini ◽  
Francis Brako ◽  
Mariagiovanna Scarpa ◽  
Mariangela Lupo ◽  
Donato Bonifazi ◽  
...  
Keyword(s):  
Translational Research ◽  
Dosage Forms ◽  
European Countries ◽  
Research Infrastructure ◽  
Oral Dosage ◽  
Orodispersible Films ◽  
Advisory Groups ◽  
European Paediatric ◽  
Form Development ◽  
Oral Dosage Forms

The paucity of evidence-based data on formulation characteristics preferred by the children is known to limit the design of tailored paediatric dosage forms. The European Paediatric Translational Research Infrastructure (EPTRI) commissioned a study to evaluate children’s dosage forms perceived preferences in some European countries and explore the feasibility of using the young persons advisory groups (YPAGs) to involve children in formulation research. An online, age-adapted survey was developed and translated into six languages. The survey link was disseminated across seven European countries: Albania, Italy, the Netherlands, and Dutch-speaking part of Belgium, Romania, Spain, and the United Kingdom. Respondents’ (n = 1172) perceived preferences for oral dosage forms primarily differed based on age, health status, and experience. Conventional dosage forms, i.e., liquid (35%), tablets (19%), and capsules (14%), were the most selected. Liquid was widely selected by children less than 12 years and by those healthy and taking medicines rarely. Monolithic solid forms were mostly chosen by adolescents and by children with a chronic disease taking medicines frequently. There was a clear lack of familiarity with more novel dosage forms (e.g., orodispersible films and granules). Noteworthy, granules were not appreciated, particularly by adolescents (52.8%). To rationalise the creation of paediatric formulations, it is important to involve children as active stakeholders and to apply tools assessing children’s perspectives on medicines to inform acceptable dosage form development from the start.

Developing translational research infrastructure and capabilities associated with cancer clinical trials

2013 ◽  
Vol 15 ◽  
Author(s):  
Jacqueline A Hall ◽  
Robert Brown
Keyword(s):  
Clinical Trials ◽  
Clinical Research ◽  
Translational Research ◽  
Cancer Biology ◽  
Clinical Decision Making ◽  
Treatment Strategies ◽  
Scientific Progress ◽  
Clinical Decision ◽  
Research Infrastructure ◽  
High Quality

The integration of molecular information in clinical decision making is becoming a reality. These changes are shaping the way clinical research is conducted, and as reality sets in, the challenges in conducting, managing and organising multi-disciplinary research become apparent. Clinical trials provide a platform to conduct translational research (TR) within the context of high quality clinical data accrual. Integrating TR objectives in trials allows the execution of pivotal studies that provide clinical evidence for biomarker-driven treatment strategies, targeting early drug development trials to a homogeneous and well defined patient population, supports the development of companion diagnostics and provides an opportunity for deepening our understanding of cancer biology and mechanisms of drug action. To achieve these goals within a clinical trial, developing translational research infrastructure and capabilities (TRIC) plays a critical catalytic role for translating preclinical data into successful clinical research and development. TRIC represents a technical platform, dedicated resources and access to expertise promoting high quality standards, logistical and operational support and unified streamlined procedures under an appropriate governance framework. TRIC promotes integration of multiple disciplines including biobanking, laboratory analysis, molecular data, informatics, statistical analysis and dissemination of results which are all required for successful TR projects and scientific progress. Such a supporting infrastructure is absolutely essential in order to promote high quality robust research, avoid duplication and coordinate resources. Lack of such infrastructure, we would argue, is one reason for the limited effect of TR in clinical practice beyond clinical trials.

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