Dr. Violeta Stoyanova-Beninska, MD, PhD, MPH has combined in her professional career clinical work, research in genetics, and regulation of medicines, particularly of therapies for rare diseases. Violeta has joined the European Medicines Agency in 2024 as senior scientific specialist at the Human Division. Her focus is on rare neurodevelopmental and neurodegenerative disorders, but also on medicines for rare diseases in general. In 2024 Violeta finished the 6 years mandate as Chair of the Committee for Orphan Medicinal Products (COMP) at EMA. She has also been member of the COMP, the Central Nervous System working party and Scientific Advice working party. At the Medicines Evaluation Board Violeta has fulfilled various roles, among which Chair of the National Scientific and Regulatory Advice. Violeta is involved in several international initiatives such as ICMRA and IRDiRC. She is the current Chair of the Regulatory Science Committee of IRDiRC. Violeta is guest faculty at several universities, Editorial board member of scientific journals, Advisory board for research projects, academic supervisor of PhD and master students at Utrecht UMC and member of PhD committee at Amsterdam UMC. In 2025 Violeta has received the EURORDIS Black Pearl Award for Leadership in rare diseases.
- What’s in the pipeline and what rare diseases have been targeted?
- How have global launch strategies been impacted by MFN?
- What are the emerging platforms (gene, cell, RNA etc) and novel trial designs for small patient populations?
- How will regulatory pathways accelerate approval, orphan designation and it’s measure overall risk/benefit
- What impacts are new technologies such as AI, Digital Health and medical devices having on the rare disease ecosystem and early diagnosis?
- Have we developed sustainable reimbursement models to fully embrace new these therapies?