Calling all patient advocacy groups - Get your free pass today!

Patient advocacy groups at the World Orphan Drug Congress 2015 Europe

Contact us for your free ticket >>

Patient advocacy groups are eligible for complimentary places at the event. Make sure you contact with your full name, job title and contact details to confirm your attendance.



Now more than ever it is recognised that patients and patient advocates need to be at the centre of orphan drug development, given their unique understanding of the conditions which treatments are being made for. 

Rare Disease Advocacy World at the World Orphan Drug Congress will mean that as well as having great opportunities to interact with sponsors, payers, government and more, there will be specific sessions delivered for patient group needs.

We had a great turn out this year with multiple advocacy groups attending and speaking at the congress. We are keen to develop this in 2016 with an even larger number of patient groups in attendence.  


► Previous topics discussed by patient advocates for patient advocates include:

  • Raising and providing funding 

  • Assessing how best to contribute to the scientific understanding of the disease and how to develop therapies, including choosing appropriate clinical endpoints. 

  • Designing and running clinical trials

  • Joining committees that decide regulatory and market access outcomes


We heard from a a range of different advocacy groups this year including: 

"Developing policies and practices for your patient group" - Christine Lavery , Chief Executive Officer, MPS Society

"The EHDN network clinical trial taskforce- The role of networks in clinical trials" - Dr Juliana Bronzova , Science Director, European Huntington's Disease Network (EHDN)

"Patient groups as equal partners in orphan drug development- The PTC Experience" - Manuela Maronati , VP of Marketing and Patient Advocacy, EMEA, PTC Therapeutics

"Industry Perspectives on patient organization-industry collaborations" - Hannah Saul , Associate Director Patient Advocacy, EMEA, Celgene

"Demystifying the process: Supporting your patients to get them involved in orphan drug development" - Alastair Kent , Director, Genetic Alliance UK

"Organising and running clinical trials as a patient group: What are the possibilities?" - Oliver Timmis , Head of Projects, AKU Society 



  • How to build a network of patient registries- Samantha Parker , Chief Patient Affairs/Health Policies Officer, Lysogene
  • How can a patient group more effectively approach sponsors- Dr Carlos R. Camozzi , Cheif Medicial Officer, ORPHAZYME
  • Challenges and a guide to building a network of partners within and outside of Europe to accelerate more collaborative cllinical trials- Prof Christoph Schuhmacher , Director of Clinical Operations, ECRIN-ERIC
  • Creating collaborations among different rare disease groups that share similar mechanisms of actions, targets and/ or therapies - Dr Bruce E. Bloom , President and Chief Science Officer, Cures Within Reach
  • Opportunities for greater patient engagement in the development process- Dr Diana Ribeiro,  Director of Research, Action Duchenne


►Patient alliance zone:

We're proud to be bringing back our Patient Alliance Zone (PAZ) on the exhibition floor this year. Here, patient groups will be able to have posters displaying their work and will be part of the specific PAZ session during the networking lunch on day one of the congress. For more information about having a spot in the patient zone, please contact .    


Contact for your free pass and join in making patients the core of orphan drug development.