A public consultation on a 3-year work plan draft was launched in November 2023 by the Methodology Working Party (MWP) to ask for feedback and gather comments from all key stakeholders. The MWP was established by the Committee for Medicinal Products for Human Use (CHMP) at EMA to gather expertise in key methodology areas, including pharmacokinetics, modelling and simulation, real world evidence (RWE)-based data and pharmacogenomics. Teddy commented on the need to revise and update existing guidelines on pharmacogenomics, including relevant information for the paediatric population, as well as to provide indications on the use of RWE applied to small populations. In addition, Teddy emphasised the importance of providing continued input to the EC for revision of the pharmaceutical legislation, specifically including the Orphan and Paediatric Regulation.

In November 2023, Teddy helped disseminate the survey on neonatal acute kidney injury, supporting its partner, the European Network of Paediatric Research at the European Medicines Agency (EnprEMA). The survey arised by a collaborative effort between the Medical University of South Carolina and the Neonatal Kidney Collaborative (NKC), an international and multi-disciplinary group of Pediatric Nephrologists and Neonatologists performing research on the incidence, causes, and outcomes of acute kidney injury (AKI) in the neonatal population. The study survey was addressed to Pediatric Nephrologists’ and Neonatologists’ to collect information on the management of paediatric AKI in routine clinical care.

Late 2021, The European Medicines Agency carried out a survey to gather information on the experience with the Orphan Maintenance Assessment Reports (OMARs) / Orphan Designation Withdrawal Assessment Reports resulting from the review by the Committee for Orphan Medicinal Products (COMP) of the orphan designation criteria at the time of marketing authorisation application. The OMAR was introduced to bridge the gap of publicly available information between the marketing authorisation and the review of the orphan criteria and to increase transparency of the assessment. The survey results suggested that more details should be provided about the prevalence and existing treatments described by OMARs. It was agreed to further discuss how to update OMARs in any upcoming meetings or during the plenary.

In February 2021, the WHO performed a comprehensive gap analysis of the most needed medicines for children in view of its review of the Essential Medicines List for children (EMLc). To support the WHO review of the Essential Medicines List for children, the Global Accelerator for Paediatric formulations (GAP-f), a WHO-led network focused on achieving Universal Health Coverage by providing better medicines for children in need, developed, in collaboration with the PENTA Foundation, a survey addressed to experts and healthcare specialists. The survey is aimed to gather the opinion of experts and health care specialists on which paediatric formulations are missing. The survey indicated that 290 medicines were reported as missing (completely or the child-appropriate formulation). The top three most mentioned were ciprofloxacin together with phenobarbital and omeprazole. The complete outcome of the survey output is contained in the article published in 2023, available here.

In December 2020, the European Paediatric Formulation Initiative (EuPFI) worked to improve the preparation of better and safer medicines for children. One area they are interested in is the “gadgets” or devices (e.g. measuring spoons, dosing cups, inhalers) that are used to help patients take their medicines. The EuPFI devices workstream designed a short questionnaire to understand what devices children use and how easy they are to handle, to help to find ways of making the devices more user-friendly. The survey was addressed to boys and girls between 10 to 18 years of age and their parents, available in different languages.

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 in a dedicated Workshop. Here’s the link to the discussion paper.

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 suggestions. 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. The current version of the Guideline is available at this link.

In 2018, the European Commission disseminated a survey targeted to the Member States, industry, notified bodies and other stakeholders with the aim to retrieve information and views relevant for the establishment of an expert panel (Medical Device Regulation (MDR), Article 106). 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. Special attention should also be drawn to children affected by chronic diseases and disabilities, including genetic and rare diseases, as well as those with extra vulnerabilities who require an urgent and increasing need of devices. You can find TEDDY answer at this link.

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. All the TEDDY comments are available at this link.

TEDDY provided its perspectives and further stressed important aspects to be included in the clinical investigation of paediatric medicines, such as:

• The inclusion of paediatric subpopulations in adult studies or adult subpopulation in paediatric studies
• The reference to address patients’ compliance when planning the development of new paediatric formulations
• Addition of dissent definition in the glossary

In 2017 the European Commission proposed a joint evaluation covering the legislation on medicines for children and medicines to treat rare diseases. TEDDY Network participated in several steps of the consultation focusing on the following topics:

• 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 registration 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.

Furthemore, TEDDY has participated in the preparation of a Position Paper on the final proposal of the EC revising the European Pharmaceutical Strategy and stating on the repealing of the Paediatric Population. The PP was promoted by EPTRI and signed by 24 key experts and paediatric research organisations is available at this link.