The course focused on the current status and advancements in clinical research, particularly in addressing unmet medical needs. Innovative methodologies, regulatory challenges, and the involvement of adolescents in clinical trials were discussed. The state of the art of funding strategies and investments was reviewed, taking into account public incentives and public–private funds at the EU, national, and regional levels. Access-related issues, such as joint procurement, national policies, HTA, and patient involvement in decision-making and access procedures, were also covered.

The event featured keynote speakers from the regulatory field, academia, and other stakeholder groups, and was free of charge.

The recordings of all sessions are available on our YouTube channel. The presentation slides are available below:

Introductory session – How a new Pharmaceutical Legislation could speed up the marketing of medicines for unmet needs in EU
Fedele (Duccio) Bonifazi, Fondazione Gianni Benzi onlus – Welcome
John Joseph Borg, Malta Medicines Authority – EU Pharmaceutical Law update – What on the table
Stefano Marino, DLA Piper – How a new Pharmaceutical Legislation could speed up the marketing of medicines for unmet needs in EU
Enrico Costa, Agenzia Italiana del Farmaco – How pharma regulation can promote innovation

Session 1 – Advancements in clinical research
Laura Pioppo, European Medicines Agency – The current state of the art of clinical research: the ACT-EU initiative
Donato Bonifazi, TEDDY Network – ACT-EU initiative for paediatric research
Viviana Giannuzzi, Fondazione Gianni Benzi onlus – Regulatory acceptability of innovative research methodologies for rare diseases
Giorgio Reggiardo, Clinical Validation from Biopharmaceutical Findings (CVBF) – Application of innovative methodologies and technologies: gaps and synergies between US and EU
Valentina Jalby, INVENTS Paediatric expert group – Showcasing the experience of involving adolescents in European funded projects and reviewing clinical study tools

Session 2 – Funding and investments for R&D
Christina Kyriakopoulou, European Commission – Opportunities for R&D on rare diseases and successful collaborations in EU
Solange Corriol-Rohou, Consultant – Public-private funding
Stefano Portolano, Azafaros– Where will industry invest?
Lucia Ruggieri, Fondazione Gianni Benzi onlus – And if the promoter is a not-for profit entity? The lesson from the Paediatric Regulation

Session 3 – Access to medicines and medical devices in Europe
Vincenzo Salvatore, Università degli Studi dell’Insubria – EU Joint Procurement: what we learnt from health threats and the proposal for Critical Medicines Act
Anna Ponzianelli, Moderna – Updates from the EU HTA Regulation
Michela Gabaldo, Evotec – The EU market of innovative medicines for unmet medical needs: where are we going?
François Houyez, EURORDIS – The patients’ point of view and their involvement in decision-making and access procedures