More than 100 stakeholders from academia, industry, regulators and patients/patient associations attended our first Multi-Stakeholder Meeting (MSM) on April 14 and 15, 2021.
MSMs are designed to facilitate discussion and interaction among relevant stakeholders on the development of medicines in the best interest of children and adolescents. Regulatory decisions are not made during the meeting and only publicly available information is shared. These are valuable meetings, which in recent years have been shown to have impact in paediatric oncology by ACCELERATE.
Our first c4c MSM was held online and focused on Paediatric inflammatory Bowel Disease (IBD). There are multiple drugs in development for IBD, however several clinical trials, as part of agreed pediatric development plans, are attempting to enroll patients with limited success. Only two new medicines have been approved for children (infliximab, adalimumab), and major delays (more than 7 years) are found between medicines approvals for adults and approvals for children. As a consequence, children do not have timely access to innovative drugs in development.
The meeting was organized with participation of the FDA, EMA and PDCO.
The MSM aimed to identify the current hurdles and develop a strategy to best address unmet needs of paediatric patients with IBD through science, the introduction of innovative development pathways and increased accessibility for all patients.
By increasing multistakeholder cooperation in paediatric IBD we can improve the development of innovative medicines for children and adolescents.
Day 1: Setting the scene
Each stakeholder provided their perspective and existing networks and registries in the field of paediatric IBD were presented. It became apparent there are several important initiatives which have been developed and could support innovative and more efficient development pathways.
Day 2: Panel discussions
Two panel discussions were held with multi-stakeholder experts, patients and advocates, allowing a deep dive into the questions: 1. How to best extrapolate? and 2. How best to use data from sources other than clinical trials? This paved the way for additional dialogue for the development of a potential strategy to take forward, allowing for improved amongst multi-stakeholders working in the field of paediatric IBD ultimately leading to improved development of innovative medicines.
The MSM highlighted that:
- This is a well networked professional community, especially wih PIBDNet
- Major initiatives have been developed over the recent years that could contribute to a new strategic development path. This could include the Inception safety and efficacy cohort during the PIBDsetQuality european project and the CREATEtm safety registry being developed by C-PATH
- Patients, parents and associations are ready to engage much more.
Agreed Future Objectives:
- Reduce the time between medicines approvals in adults and children
- Provide children and adolescents with timely access to innovative medicines in order to eventually reduce off-label use : start paediatric studies early before 1st marketing authorization in adullts
- Make the best use of extrapolation, and real world data including registries, and networks
- Address prioritization of which compounds developed in adults should be evaluated in chilren to best address patients’ unmet needs (changes in current regulations may be needed to facilitate).
- Consider novel study designs to achieve the most appropriate paediatric development plan and do not duplicate the adult development plan in children
- Lower the age inclusion criteria for adult clinical trials to allow participation of adolescents in more studies – this is fully supported by both the FDA and EMA in oncology.
What next for paediatric IBD?
- The group coordinators will publish the meeting content and the proposed strategy in a peer-reviewed journal
- An International multi-stakeholder core group will be set up to drive forward the paediatric IBD agenda to include:
- Finalization of an agreed new development path;
- Definition of a workplan to deliver on the future objectives;
- Set up of a international multistakeholder consensus meeting on use of extrapolation of IBD data;
- Set up focus multistakeholder meetings on class of compounds (eg anti- IL123).
This activity will be driven by the PIBD community.