Nextep was with Medvance at the World Orphan Drug Congress
This year, the program has been extended to gene therapies, cell therapies, and rare cancers.
Medvance organized at the World Orphan Drug conference a roundtable regarding early access for orphan drugs in Europe, underlying thru compassionate use or ATUs in France the commonalities of different systems based on the same vision of offering innovative drugs to patients as quickly as possible without putting too much cost pressure on the healthcare budget. It is also a good opportunity for the industry to collect real life data when clearly clinical studies are usually not conclusive enough given the limited volume of patients.
About the World Orphan Drug Congress
The World Orphan Drug Congress USA brings together world leaders in orphan drug development and rare disease research, alongside regulators, payers, and patients. Entirely dedicated to rare diseases, this congress explores the new therapies available and the means to facilitate the access of patients with rare diseases to these treatments.