From cell and gene therapy, genetic testing, and market access, to real world evidence, this one meeting covers the whole orphan drugs value chain where science, government and manufacturers all come together to create groundbreaking progress.
Conference key topics
Access & pricing
Understand the dynamics governing global rare disease markets & what interventions can be made for better access.
BioPharma showcases
Highlighting upcoming and existing orphan drug developers and what’s new on the horizon. Showcasing the latest pipeline, potential partnering opportunities and highlighting the growing interest in multiple disease areas.
Cell & Gene Therapy
Find out how the latest gene therapies are transforming patients living with rare diseases, and what the future holds for the next wave of innovative medicines.
Clinical development
Understand how to work with small patient group sizes using meaningful endpoints with patients and regulators, moving your orphan drug candidates through development phases to licensure.
Investment & funding
Innovative funding strategies and what do investors look for before investing in rare disease. Learn about what investment models there are to overcome Orphan drug development bottlenecks.
Manufacturing
The future of manufacturing within Cell & Gene therapy and best practices to overcoming manufacturing and commercialisation obstacles.
Patient Centricity
Truly represent the patient voice and advocate for them by hearing from our partners and finding out firsthand, what has been achieved and what can be improved further.
Precision Medicine
From new gene editing technologies to new born screening, find out how precision medicine is changing the way we look and define rare disease diagnosis and treatment.
Real World Evidence
Find out how to use real world evidence should be evaluated for potential applications to increase speed of product approval, reimbursement and clinical practice adoption.
Science Strategy
Bridge the gap between science, policy and corporate strategy in order to secure commitment from all stakeholders
