Main Conference

22-25 October,  2024

Strategy, Advocacy & Partnering For The Global Orphan Drug Industry


The World Orphan Drug Congress is the largest, most established European meeting dedicated to orphan drugs.

From cell & gene therapy, genetic testing, 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.


Cell & Gene Therapy

Find out how the latest gene therapies are transforming patients with rare diseases, and how we move on the pay and make these treatments accessible for all.


Clinical Devleopment

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.


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


Technology Showcases

Hear from investors and the most innovative biotechs finding new solutions to advance orphan drugs development.