On 19 March, the Committee on the Environment, Public Health and Food Safety (ENVI), as the committee responsible for the procedure, adopted its position on the new Directive and Regulation on medicinal products for human use.
MEPs agreed on a number of compromise amendments, which in some cases did not change the proposed text of the legislative texts. Full details of the compromised amendments are available in the original files for the Directive and the Regulation.
Among the paediatric provisions proposed by EPTRI in its Position Paper, it is noteworthy that the Regulation foresees an ad-hoc working group on Paediatric Medicinal Products (art. 150 as amended)
This aspect, which was not included in the text proposed by the Commission, was supported by several MEPs from different political parties.
In addition, the Regulation lays down:
- The provision to involve, on an advisory basis, parties concerned with the use of medicinal products for human use, in particular patient and consumer organisations, including paediatric representatives.
- A reform of the waiver system for Paediatric Investigation Plan (PIP) based on the medicinal product mechanism of action. It would not be possible to have a waiver if a medicinal product is directed at a molecular target or due to its mechanism of action, is responsible for a different disease or condition in the same therapeutic area in children than the one in adults.
- The possibility of having an initial paediatric investigation plan, in cases it is not scientifically sounded to have a complete paediatric development based on the available information. Timetables for providing further details for a full plan must also be provided.
- The need to provide scientific, technical or public health considerations to justify a deferral to an agreed Paediatric Investigation Plan has been emphasised.
Additional aspects of the Regulation not directly related to paediatric issues could potentially have a positive impact on the advancement of paediatric research and empowerment:
- Improving the readability, clarity and comprehensibility of summaries of European Public Assessment Reports (EPARs) and user-friendly information on medicines.
- Increased transparency on a number of processes, including scientific advice and the PRIME scheme in EPARs.
- Improved action and information on drug shortages, including the involvement of patients and families.
- Establishment and involvement of a patient-consumer working group.
- Reference to refinement and replacement strategies for animal testing, such as non-animal in vitro and silico approaches.
- Work with patient organisations and healthcare professionals to develop guidelines for determining therapeutic added value.
- Integration of patient-reported outcome measures (PROMs) and patient-reported experience measures (PREMs) into clinical data supporting marketing authorisation.
The Directive has highlighted the need to make publicly available the conclusions of the assessment of compliance with the paediatric investigation plan. It has introduced the need for indicators to measure access to medicines in the EU, including paediatric medicines. The amended text of the Directive recognises the need to make efforts to address the problems encountered with paediatric medicines, in particular the failure to complete paediatric clinical trials and obtain data required for marketing authorisation in a timely manner, which delays the authorisation of paediatric medicines.
As additional aspects:
- The general marketing authorisation provisions of the Directive mention that in the absence of a paediatric investigation plan or in cases where a comparative study has not been carried out, a justification and, where appropriate, evidence from long-term post-marketing studies should be provided.
- The need to address the risk-benefit balance and other paediatric specificities (storage, assembly, cleanliness and technique) for medicinal products in combination with medical devices.
- Specific mention of pharmacovigilance to monitor long-term post-authorisation safety and efficacy studies in children, including relevant data from off-label use of medicines.
Although the new legislation could be more rigorous in addressing paediatric specificities throughout the lifecycle of medicines development and evaluation, EPTRI strongly welcomes the establishment of a Paediatric Medicines Working Group.
If the mandate, composition and procedures of this group are appropriate and well established, the extensive expertise developed within the Paediatric Committee will not be wasted.
MEPs will debate and vote on the Parliament’s position during the plenary session on 10-11 April. The dossier will be taken up by the new Parliament after the European elections on 6-9 June.