4th DEEP Scientific Open Meeting (Padua 2016)
CHALLENGES AND OPPORTUNITIES OF CONDUCTING CLINICAL TRIALS IN PAEDIATRICS
Clinical research: where are we now with the new CT Regulation by Prof. Hugo Devlieger, DEEP Ethics Board Member, Department of Development and Regeneration, University of Leuven, Leuven, Belgium
The new European regulation on clinical trials was adopted in May 2014 and was supposed to enter into force by May 2016. This regulation is intended to abolish Directive 2001/20/EC and correct some of its shortcomings e.g. the lack of harmonization, and hence its attractiveness especially for multinational trials. Central to this harmonization process is the registration of the CTA in a unique EU portal, set up and managed by EMA, and that will function as a communication center between the sponsor and the MS. The risk based approach led to the differentiation between CT and low-intervention trial requiring less stringent demands. Other important changes are the co-sponsorship and the short timelines. The sponsor has to register the trial in the EU portal together with the EU medicinal product number and propose a reporting MS that will assess the scientific part of the application while other MS interact or tacitly approve The informed consent process is still assessed by each MS but within the same short timeline. The regulation takes into account a great number but not all of the EC Paediatric Recommendation (2008). Due to delay in the development and agreement of functionality of the EU portal, the new regulation will not enter into force before October 2018. In conclusion, only the implementation of the new regulation, intended to harmonize and coordinate, will learn us if it holds its promises and lead to improve the quantity and quality of CT in children without bending the ethical principles.
Challenges and new opportunities of cooperation with the Mediterranean countries in the field of paediatric clinical research by Prof. Amal El-Beshlawy, Professor of Paediatric Hematology, Cairo University, President of the Egyptian Thalassemia Association, Egypt
Research is a complex process, generation of knowledge is only one part of the research process, for knowledge to be useful it should be shared with other researchers in different nations and communities. The EuroMediterranean network of research centers of thalassaemia and other haemoglobinopathies is a good example for enhancing the scientific potential for a high impact collaborative research in Egypt and other Mediterranean countries. Infrastructure for thalassaemia research network (ITHANET) and the Euro-Mediterranean Network for Genetic services (MedGeNet) were a good example for constructive and beneficial collaborative work in the domain of medical management and diagnostic skills for haemoglobinopathies. The DEEP Project (FP7) is a new aspect of Euro-Mediterranean collaborative study for the development of an old drug in a new liquid formulation specific for the pediatric population with haemoglobinopathies.
Although we have some challenges in the research studies in Egypt, we have got many advantages: a huge population with many diseases diversity; big pool of treatment naïve subjects; university hospitals with a strong infrastructure and facilities; GCP compliant regulators and Ethics Committees; no regulatory limitations on pediatric researches and highly qualified investigators with international accreditations.
The Ethical issues in EU and non EU countries by Viviana Giannuzzi, Researcher and regulatory expert at the Gianni Benzi Pharmacological Research Foundation, Italy
From the ethical point of view, paediatric research looks for the balance between protecting children while making sure they benefit from safe and efficient medicines. The complexity increases because paediatric studies are typically multi-centre and multi-national, and ethical requirements differ among countries and national legislative frameworks.
This has been highlighted both at international and European level, where Directive 2001/20/EC, guidelines and recommendations have been differently implemented in national laws. The upcoming European Regulation 536/2014 is expected to harmonise practices in Europe and possibly outside Europe.
In the framework of FP7 programme, many paediatric multi-national trials have been funded and faced the challenges related with the multi-ethnicity of trial populations and the need for complying with GCP and ethical standards guaranteeing children’s protection. Most of them have been already approved by the local ethics committees and are ongoing all around Europe and outside.
Today, many clinical and therapeutic information from paediatric patients are obtained from the secondary use through the retrospective collection of ‘big data’. This is requiring greater and greater effort to guarantee an ethical standard for children, protection for confidentiality and personal data, including genetic data, given that the ethical framework in this field is less regulated.
Global initiatives for paediatric research: the path to the European Paediatric Clinical Trials Research Infrastructure (EPCTRI) setup by Donato Bonifazi, Chief Executive Officer at Consorzio per Valutazioni Biologiche e Farmacologiche, DEEP Project Manager
A Research Infrastructure is a research tool identified in the European Research Agenda with reference to facilities, resources and related services used by the scientific community to conduct top-level research in their respective fields. Unlike the clinical research landscape in adults, there is no commercial or academic research infrastructure to efficiently perform paediatric clinical research. This is due to the well recognised difficulties in developing paediatric medicines as children cannot be addressed with adult instruments in medical settings. In March 2015, a large group of top level research networks located in several European countries, led by the ENPR-EMA Co-Chair Prof. Mark Turner, developed and submitted a proposal to build an infrastructure addressing this gap all across Europe with innovative scientific and methodological strategies.
This new research infrastructure, named EPCT-RI (European Paediatric Clinical Trial – Research Infrastructure) will include many therapeutic expertise over a broad spectrum of disease indications and conditions as well as innovative research methodologies.
Expected results: EPCTRI will enable researchers to manage paediatric clinical research and implement important research activities through the creation of innovative platforms and will bring many advantages: aggregation and growth of scientific structures in the field of innovation and life sciences; development of new medicines for children to enhance their health, safety and quality of life; promotion and development of new business and industrial processes.