The Telethon Foundation's Translational Project Management and Regulatory Affairs Office helps researchers at Telethon Research Institutes to navigate the regulatory landscape and fulfil their obligations in the development of advanced therapy drugs, supporting them from the early stages of product production and preclinical research, through development and up to registration and market access.
What we do?
The Office of Translational Project Management and Regulatory Affairs of the Telethon Foundation consists of a team of experts in drug development management who support projects regardless of how they are funded (internally funded, publicly funded, externally funded through alliances with pharmaceutical companies or start-ups).
The office also offers its researchers regulatory support from the earliest stages of obtaining orphan drug designation in order to maximise access to the incentives reserved for this special category of drugs and throughout the entire development pathway up to patient access. Specifically, the team offers regulatory expertise for the development of orphan, paediatric and advanced therapy drugs and helps clinicians to give access to their innovative drugs to patients with rare diseases who come from outside Italy with a hope of a cure.
Specifically, the team can help researchers to build development plans, but also to obtain Orphan Drug Designation (ODD), to study the possibility of applying for the Innovation Task Force (ITF) or subsequently for the PRIME programme, prepare the application and follow the procedure to obtain scientific advice of development protocols from the European Medicines Agency (EMA Scientific Advice) and national competent authorities, fulfil the obligation to submit the paediatric investigation plan (PIP) and intercept other dialogue opportunities offered by the EMA to academic developers. In addition, the Telethon Foundation's Translational Projects Management and Regulatory Affairs Office helps the clinical team to assess the possibility of taking part in national programmes for expanded access and early access to medicines.
