On July 19, the kick-off meeting of the OrphaDev4Kids (OD4K) project was held in Bari, Italy.
The OD4K is a 3-year project - coordinated by Consorzio per Valutazioni Biologiche e Farmacologiche (Italy) and involving 6 European Institutions - aiming to create an ecosystem that facilitates the bringing together of skills, knowledge, research ideas and financial resources, maximizing effectiveness and efficiency in developing innovative solutions for children with rare diseases.
The role of Fondazione Gianni Benzi and its contribution to the success of the initiative was explained by Silvia Torretta and Franco Bartoloni during the event. Specifically, the Foundation will support the development of three medical devices for children with orphan diseases, not only by taking care of ethical and regulatory aspects, but also by providing tools to streamline the process. In collaboration with Young Patient Advisory Groups (YPAGs), the Foundation will also prepare age-appropriate ethical and regulatory documents for paediatric participants.
During the project life the stakeholders will have access to the following services, to be provided with support also from Fondazione Benzi:
1. Advice, mentoring and assistance to manage the development of prototypes and final paediatric and orphan MD products including MD design, quality, non-clinical and clinical studies, performance evaluation, post-marketing evaluation (safety monitoring and HTA).
2. Regulatory, ethic and privacy (GDPR applications) advice for experimental studies, marketing, and commercialization, including CE certification and Marketing Authorisation (MA).
3. Tools to match relevant players with orphan device ideas with potential manufacturers, to identify existing financing resources and to implement their direct knowledge on MD certification and MA procedures and disposition in Europe.
4. Services required with reference to three orphan and paediatric case studies having different needs and challenges in accordance with their developmental stage.