The 8th World Orphan Drug Congress is taking place 13 - 15 November, Barcelona and is not an event to be missed this year. Now recognised as the largest and most established European orphan drug event, we are once again proud to deliver another fantastic speaker line up and content filled agenda that represents the whole orphan drug scene. The 8th annual event to address the strategic and commercial aspects of bringing new treatments to patients who suffer from rare diseases. You’ll notice that the Patient Advocacy Track is no longer here, it’s because we’ve integrated the patient voice in every part of the whole agenda.

Through strategic keynote plenaries, themed tracks and dedicated networking sessions, you have the chance to really tailor the event to the needs of your day-to-day role.

Attend the Pre-Congress Workshop on the 13th November to learn from interactive presentations and make new contacts during the networking breaks. 


Plenary discussion on best practices to generate pre and post-marketing data – the value of shared governance of registries from an international registry , regulator and pharma perspective – don’t miss out!

Keynote sessions from the ORPH-VAL working group, using best practice European principles in assessing the value of orphan medicines . Join us with UK, France and Germany HTA representatives in correcting the access discrepancy between countries

Interview executives from the industry . Find out what Genzyme, GSK, Roche and Prometic ultimately hope to achieve in the orphan drug space


Pre-congress workshops on the 13th November will give you a full days’ worth of content where you get the chance to learn topics from health economics, payer and evidence generation to unique challenges in regulation, pricing, reimbursement & market access for cell and gene therapies for rare diseases – pick this option on your registration to join


Progress your treatment with relevant clinical endpoints , navigate through the approval process and design innovative trial designs by hearing from Alexion, Takeda, Bluebird Bio, Premier Research, PRA Health Sciences, INC Research and more


The value of ODs . Join our hard hitting access & pricing talks with HTA perspectives from UK, Spain, Germany , Poland and Austria as well as industry experts from Sobi, BMS  and Dolon   

World Orphan Drug Congress Europe Agenda Overview

Two days of Gene Therapy sessions – the sector has grown tremendously in the last few years. How would you price a cure and manufacture a gene therapy sustainably? Hear from NHS England, GSK, Pfizer, Biomarin, Chiesi, Genethon, Orchard Therapeutics, ReGenXBio, Lysogene, Abeona Therapeutics and plenty more


New investors in the Pitch & Partner track will help biotechs and academics understand how to gain captial and progress their treatments


Combining Science & Strategy is what our congress does best, which is why we have invited high-level speakers to give us insights into their business model , patient engagement methods and the impact of precision medicine from Findacure, AbbVie, Action Duchenne, Merck, RARE Science, Horizon Pharma, Armetheon, Caribou Biosciences, Cellectis Therapeutics, GW Pharmaceuticals & many more