1 – 2 March 2022 – 4:00PM – 7:30PM CET (both days) – Virtual event
Call for Expression of Interest
(deadline: 14 January 2022)
conect4children is pleased to announce that the second c4c International Multi-Stakeholder Meeting (MSM) will address paediatric Atopic Dermatitis (AD) and will take place VIRTUALLY on 1-2 MARCH 2022. The meeting is being organized with the participation of the European Medicines Agency (EMA) and the Food and Drug Administration (FDA).
Multi-Stakeholder Meetings have been conceived with the aim to facilitate dialogue and provide an opportunity for constructive interactions between relevant stakeholders (patients/patient representatives, clinicians, academics, pharmaceutical companies and regulators) on topics requiring open discussion on development of medicines in the best interests of children and adolescents. The goal of these meetings is to share information, in a pre-competitive setting, to define unmet medical needs, to define how best to address those needs and facilitate the development of innovative medicines towards eventually their introduction into the standard-of-care of children and adolescents.
Rationale
Approximately 10 to 20% of the paediatric population have AD. As a chronic inflammatory disease, AD is a systemic disease potentially associated with food allergy, asthma and co-morbidities in its moderate and severe forms. AD is life-long disease that impacts patients’ life daily. 40 % of paediatric and adolescent AD patients have moderate or severe AD.
AD treatment of the paediatric population consists mainly of topical treatments, with special considerations for those <2 years. When topical treatment does not suffice, systemic treatment options are limited to e.g. methotrexate and ciclosporin, two medicines not widely approved for paediatric use for this indication. During recent years, the development of novel systemic treatments such as interleukin inhibitors (anti-IL4Rα, anti-IL13 and anti-IL31) and JAK inhibitors have enlarged the therapeutic portfolio of systemic treatments in adults. For some of those, the indication has been extended to adolescents (≥12 years) or children (≥6 years). Paediatric development is ongoing for some of these compounds.
There are many other assets in development and the issue is how best to address unmet medical needs of paediatric patients with AD.
Topics to be address and discussed
Through updating the biology and scientific rationale for drug development in AD, defining the unmet medical needs of children and adolescents with AD, hearing the voices of patients and parents coping with the disease, and sharing public updates of ongoing paediatric and adult drug development, the meeting will discuss and address:
- How to best address the unmet needs of children and develop safe and effective therapies for all, that are also accessible and affordable?
- How to improve trials in children (best trial designs, use of surrogate endpoints, reduced number of patients to be enrolled, extrapolation from adult data, improve trial execution)?
- How to accelerate the developments in children and set up prioritization among assets in development when needed?
- Lack of biomarkers and the current research thereof in AD.
- How to define the strategic use of new medicines to better address paediatric patients’ needs and journeys?
- How to identify early in their disease patients who have or will develop moderate or severe AD and be candidates for innovative therapies?
- How to position novel systemic treatments in the life-long journey of young people living with AD. E.g. when to start and when to stop (or not stop)?
- How to weigh up the choice of treatment regarding comorbidities, e.g. anti-IL4Rα/IL13 in those with comorbid asthma.
The overarching objective of the meeting is to propose a strategy to improve the timely development of therapeutic innovations for children and adolescents with AD, properly addressing paediatric unmet needs through science, introducing innovative development pathways and increasing accessibility for all patients. The goal is to set up a cooperation of academia and industry, with support of advocacy and regulators within a new and facilitating regulatory environment.
The meeting will provide the opportunity to share and discuss all recent advances and progress in the field of paediatric AD, to review and discuss molecules with an ongoing paediatric development plan. New development pathways introducing alternative innovative trial designs and pharmacological evaluation, concrete use of extrapolation, use of real world data and earlier initiation of paediatric studies will be defined.
Outputs
The output of the meeting will be a common understanding of the needs, issues and available assets by ALL stakeholders and a proposed strategy based on a stronger cooperation between academia and industry, which will include patient advocates’ point of view and facilitated by interactions with regulatory agencies.
No regulatory decisions will be made during the meeting which will share publicly available information. However, the findings and conclusions of the meeting will pave the way for future development plans and concrete actions. Following the meeting conclusions, an article will be submitted to a peer reviewed journal in the specialty.
Who can participate?
International academic experts in paediatric atopic dermatitis, representatives from pharmaceutical companies developing assets in atopic dermatitis, representatives from regulators (EMA and FDA, as well as HTAs) and patients, parents and their advocates.
The meeting will be on invitation only following expression of interest. Interested participants can apply by filling the Expression of Interest form available HERE.
Invited participants will be contacted in due time.
Related posts:
EMA and the EC DG-Sante have published the Joint action plan to support the development of medicines for children in Europe
‘Prescribing Medicines for Children’: new international textbook published
New educational toolkit available for young patients and families
c4c submits a shared response to the FDA consultation