TEDDY Network’s activities are constantly addressed to the promotion of adequate health policies taking into account children diseases specificities and unmet needs of paediatric population. TEDDY is always proactive in addressing ethical, legal, social and regulatory issues of paediatric research, enhancing the awareness of Regulatory Authorities, policy decision makers and the main stakeholders on the still unmet needs and peculiarities of children. For this reason, over the last years TEDDY has participated in several European initiatives and Public Consultations, also with the aim to promote the engagement of children and young patients in decisions related to clinical research and healthcare.
Below, you can find a list of the initiatives.
TEDDY Comments to the EMA Discussion Paper: Secondary Use of Data for Medicines and Public Health Purposes
TEDDY Network provided its contribution to the EMA discussion paper “The General Data Protection Regulation: Secondary Use of Data for Medicines and Public Health Purposes”. The guidance released by the European Medicines Agency (EMA) addresses the impact of EU data protection legislation on the secondary use of health data in support of the development, evaluation and supervision of medicines.
TEDDY Network provided its suggestion, underlining to use multiple databases to foster scientific research and suggested making specific reference to minors in different sections of the document, such as “Establishing the legal basis for processing personal data”, “Rights of the data subject” and “Transparency”. It is possible to read the complete TEDDY’s consideration and comments to the Discussion Paper by clicking here.
Submission of comments on 'Guideline on safety and efficacy follow-up and risk management of Advanced Therapy Medicinal Products Draft' (EMEA/149995/2008 rev.1)
In February 2018, the European Medicines Agency released a revision of the “Guideline on safety and efficacy follow-up and risk management of advanced therapy medicinal products” and invited all the stakeholders to provide comments and suggestion. The guideline describes specific aspects of pharmacovigilance, risk management planning, safety and efficacy follow-up of authorised ATMPs, as well as some aspects of clinical follow-up of patients treated with such products.
TEDDY Network participate in the Consultation and suggested including in the guideline paediatric issues and specificities. And mention the EMA and ICH paediatric provisions (e.g. ICH Topic E11, the 2007 EMEA Guideline on pharmacovigilance for medicines used by the paediatric population EMEA/CHMP/PhVWP/235910/2005- rev.1). Given the increasing number of ATMPs tested and administered to children and the genetic nature of many paediatric diseases, TEDDY suggests addressing these issues should be addressed as well as requiring the feedback from PDCO.
You can read all the comments provided by TEDDY here.
The current version of the Guideline is available at this link.
Concept paper on the need for a paediatric addendum of the guideline on clinical investigation of medicinal products for the treatment and prophylaxis of venous thromboembolic disease
In 2017 the European Medicines Agency addressed the need for a paediatric addendum of the guideline on clinical investigation of medicinal products for the treatment and prophylaxis of venous thromboembolic disease (VTE). The current version of the guideline is available at this link.
TEDDY Network provided its comment to the Concept paper and suggested implementing the document with further data about the paediatrics population. For example, TEDDY proposed to add supporting data from existing registries/epidemiological data to justify that in neonates and young children VTE occurs more often (60%) in the upper venous system (vs. only 2% in adults). Likewise, TEDDY experts underlined the importance to include some data on the practical approach to VTE therapy, considering the factual problems experienced by paediatricians in administering low-molecular weight heparin in childhood and infancy.
All the TEDDY comments are available at this link.
Submission of comments on “ICH E11 (R1) guideline on clinical investigation of medicinal products in the pediatric population” (EMA/CHMP/ICH/2711/1999)
Paediatric drug development has evolved since the original ICH E11 Guideline (2000), requiring consideration of regulatory and scientific advances relevant to paediatric populations. This document provides an outline of critical issues in paediatric drug development and approaches to the safe, efficient, and ethical study of medicinal products in the paediatric population. The purpose of the addendum of the “ICH-E11 (R1) guideline on clinical investigation of medicinal products in the pediatric population” released by EMA in 2017 is to complement and provide clarification and current regulatory perspective on topics in paediatric drug development. Before its adoption, a Public Consultation was launched to receive any suggestions on the Addendum. TEDDY Network was pleased to provide its comments and further stress some important aspects to be considered in case of clinical investigation of medicines in the paediatric population, such as:
the inclusion of paediatric subpopulations in adult studies or adult subpopulation in paediatric studies is not the rule, but an exception and has to be properly justified (e.g. in the case of rare diseases)
the reference to the need to address patients’ compliance when planning the development of new paediatric formulations
addition of dissent definition in the glossary
TEDDY complete contribution is available at this link.
Survey for assessment of expertise and services for paediatric clinical research in Europe
In 2017, TEDDY Network carried out a survey to determine the capacity and competence of paediatric centres to perform research studies. A cross‐sectional, web‐based pilot survey was conducted from October 2016 to April 2017 with paediatric clinical research centres in 11 countries: Albania, Austria, Belgium, Denmark, Iceland, Ireland, Italy, Norway, Spain, Switzerland and the United Kingdom. 107 centres provided data on their experiences and expertise in paediatric clinical trials. Four groups of performance indicators were identified, referring to scientific experience, trial readiness, trial competence, regulatory issues, ethics and patients. Most centres were actively involved in paediatric clinical research: 53 centres (84.1%) had received funds for more than five paediatric studies in the last 5 years, and 42 (66.7%) had a specific clinical trial unit and dedicated study coordinators. The analysis of results showed that the European centres investigated with the survey had the capability and capacity to conduct paediatric trials, but there was still room for improvement, including enhanced collaboration.
The survey results were described in the article “Survey by TEDDY European Network of Excellence for Paediatric Clinical Research demonstrates potential for Europe‐wide trials” prepared by Lucia Ruggieri from the Gianni Benzi Foundation in collaboration with TEDDY representatives and published on the Acta Paediatrica Journal. To get access the entire paper, please contact Dr. Lucia Ruggieri (email@example.com).
With a proposed budget of 100 billion euro from 2021 to 2027, the Horizon Europe framework programme represents the largest collaborative multinational research and innovation investment in Europe and is open to participants worldwide. The European Parliament and the Council, the co-legislators have provisionally agreed on the Horizon Europe legislative package. Based on the agreement, a Strategic Plan will put forward the targeted impacts for the investment in research and innovation and the priorities for the first four years of implementation of Horizon Europe.
European commission invited all the stakeholders to co-design and contribute shaping the future research and innovation investment by responding to a questionnaire.
TEDDY provided its own contribution to the design of the next programme participating to the consultation and drawing attention on the paediatric issues that need stronger investments in order to deliver more benefits to the paediatric population.
Survey on expert panels (Medical Device Regulation, Article 106)
In 2018 European Commission disseminate a survey targeted at Member States, industry, notified bodies and other stakeholders with the aim to retrieve information and views relevant for the establishment and running of expert panels (Medical Device Regulation (MDR), Article 106). The survey draws on a scoping paper produced by JRC (Joint Research Centre) in consultation with DG GROW (DG for International Market, Industry, Entrepreneurship and SMEs) which provides more detailed information on some aspects of the survey (areas of competence relevant for defining a landscape of expert panels, selection criteria, roles, practical steps towards their establishment, detailed workflows, estimates of their workload etc.). This survey has been designed to collect information from relevant parties and to gauge their opinions, views and preferences, in view of the establishment of expert panels in agreement with the provisions of the MDR and IVDR (In vitro Diagnostics Medical Device Regulation). TEDDY Network suggested to include in the expert panel specialists in paediatrics with documented expertise in paediatric research methodology and evaluation procedures, clinical studies and healthcare processes. A special attention should be also draw to children affected by chronic diseases and disabilities, including genetic and rare diseases, with extra vulnerabilities and possibly increased need of devices.
Evaluation of the legislation on medicines for children and rare diseases (medicines for special populations)
In 2017 the European Commission proposed a joint evaluation covering the legislation on medicines for special purposes, in particular medicines for children and medicines to treat rare diseases. The evaluation assessed to which extent the EU legislation is efficient and effective and considered whether it is fit for purpose in the light of developments in the area of pharmaceuticals. It looked in particular in the impact of the incentives introduced for research, development and marketing, for these specific medicines. TEDDY Network participated in the initiatives and increase the awareness about different point the discussion, such as:
After 10 years from the entry into force of the Paediatric Regulation, the number of paediatric medicines in Europe has tripled, but they remain about one third out of the centrally authorised medicines (TEDDY European Paediatric Medicines Database, 2nd General Assembly).
With reference to the special funds devoted to paediatric medicines development the funds that the European Commission have been addressed to projects with demonstrated therapeutic interest for children have been delivered ‘only’ for a short period under the Seventh Framework Programme and the amount was very limited to cover the cost of registrative trials (about 6 million/project).
It is agreed that paediatric and rare diseases overlap, but it should be considered more likely that a considerable number of rare diseases qualify as paediatric ones.
TEDDY complete contribution is available at this link.
Here you can find more information about the evaluation initiative.
TEDDY contribution to Public consultation on Summary of Clinical Trial Results for Laypersons
In 2016, the European Commission launched a Public Consultation on “Summary of Clinical Trial Results for Laypersons” for the Implementation of Regulation (EU) No 536/2014
TEDDY-European Network of Excellence for Paediatric Research participated in the public consultation, proposing to include the paediatric aspect in the guideline.
To include the paediatric aspect in the consultation document by adding a paediatric paragraph and a paediatric section in the Annex-1
To add specific reference to the paediatric population in the proposed recommendations and templates for authors of laypersons summary
To develop laypersons summary in age appropriate, simple and understandable language to ensure easy reading by parents and by children
TEDDY proposal can be downloaded here.
After TEDDY request, a specific paragraph on children has been integrated. The entire document is available at this link.
TEDDY Inventory of procedures for obtaining Paediatric Clinical Trials approvals
TEDDY collects information on the documentation to be prepared and the procedures to be followed to obtain the authorisation to conduct a clinical trial in different EU and non-EU countries.
Our inventory provides instructions on the documents to be included in the clinical trial application, the submission modalities for both competent authorities and ethics committees, and other practical information such as contact details.
Moreover, the database provides information regarding country-specific provisions for informed consent and assent required for the participation of paediatric patients, such as the age in which the consent of the child is considered mandatory.
Other specific national arrangements for paediatric clinical trials are also provided.