Fondazione Gianni Benzi is pleased to share the participation of our Head of Research and Innovation Department, Dr Viviana Giannuzzi, at the World Orphan Drug Congress 2024, held in Barcelona from October 22-25.
Viviana gave a lecture on the work done by Fondazione Benzi in collaboration with the coordinator, the Ethics Advisor team and other partners of the EJP-RD project that has paved the way to the new ERDERA project, the EU-wide initiative dedicated to rare disease research and patient care. In the presentation “Ethics and Regulatory Guidance for Not-for-Profit Research for Rare Diseases: The Experience of a European Funded Programme”, she described the current and unique ethical and regulatory challenges faced by researchers and highlighted the crucial role of a comprehensive ethics and regulatory support and guidance to help them in addressing such issues. She also emphasized the importance of ethical standards and regulatory guidance to ensure responsible research practices.
In addition, Viviana participated in two impactful panel discussions focused on critical topics in the rare disease field. One was dedicated to the potential and challenges of Artificial Intelligence in rare disease research. She provided the state-of-the art of the existing regulatory framework within the EU aimed at maintaining accuracy, reliability, and ethical integrity in AI applications, particularly in high-risk systems. In rare disease contexts, where sample sizes are often limited, AI shows promise in improving diagnosis and treatment. Viviana emphasized additional safeguards to protect patient privacy, including strict GDPR compliance through data minimization, anonymization, and encryption, and mechanisms to prevent bias in high-risk AI applications.
The other panel discussion was on treatment access across Europe for rare disease patients. She shared insights on the impact of a diverse availability and accessibility of orphan medicines across European countries, although these medicines are authorized centrally. This disparity still represents a current problem and calls for solutions, like harmonized pricing and improved availability measures to make orphan medicines more accessible across all EU member states.
Viviana’s contribution and participation underscore Fondazione Gianni Benzi’s commitment to advancing ethical, regulatory, and technological progress in rare disease research.
We look forward to a continuous collaboration and dialogue within the rare disease community to achieve more equitable healthcare solutions across Europe.