11th Orphan Drugs & Rare Diseases Congress 2019

Though by definition not more than 5 out of 10,000 people are affected by a rare disease in the European Union, such diseases are not that rare in total: estimates assume that about 30 million people are affected by a rare disease in the EU. Since most experts and patients are widely distributed across the world, events which enable all parties concerned with rare disease to exchange their thoughts, expertise and ideas for the future are extremely important. For this reason, we are very pleased to be the main sponsor of this meaningful congress for the fourth time.

Prof. Matthias P. Schönermark, M.D., Ph.D., is proud to be the chairperson of the event, which allows him to guide the attendees through the program of the first day. Professional experts from different background are gathering at plenary discussions, keynote panels and round table discussions. The exemplary range of the experts speakers covers:

• Amit Rakhit, Chief Medical Officer and Head of Research & Development, Ovid Therapeutics
• Jim Shaffer, Chief Business Officer, Eiger BioPharmaceuticals
• Dan Donovan, Founder & CEO, rareLife solutions, creators of onevoice
• Derek Ansel, Director of Rare and Orphan Diseases, Synteract
• Hannah E. Kane, State Representative, Boston, Massachusetts
• Khrystal Davis, CEO, Founder, Zebra Leaf Publishing, Writer, Hunt for a Cure, Author
• Lisa Deck, Founder and Director of Sisters@Heart (non-profit organization)
• Dr. Neena Nizar, Founder and Executive Director, Jansen's Foundation and
• Representatives of the FDA

Prof. Schönermark will hold a plenary keynote, which is about "Risky business: success and failure in orphan drugs' business model". He is going to address these essential topics in his speech:

• The Orphan Drugs and Rare Diseases dilemma - population size vs. price premium
• Key success factors for a powerful and sustainable market access
• Learning from failure: avoidable roadblocks and pitfalls
• Collaborative approaches for a complex environment

Femida Gwadry‐Sridhar, founder and CEO of Pulse Infoframe will co-chair the event on the second day. "Orphan drugs in Europe: current developments in a heterogeneous market" will be debated with Prof. Schönermark in a roundtable discussion on that day. Shortly after the networking lunch, the panel discussion about "Pricing and ROI of Orphan Drugs: What is the right price strategy for orphan drugs to continue to treat patients and remain commercially viable for manufacturers?" will take place, examining these key issues:

• Devising funding strategies to develop drugs for the treatment of rare diseases remains a challenge as patient groups are often too small
• Bold new ways to secure investment
• Budget impact of RD therapies create a real concern for payers
• Responsible and evidence-based pricing, and innovative contracting
• Forward thinking approach for better market access to rare disease therapies"

Based on the impressive experiences from last year's congress in Boston, SKC consulting is looking forward to stimulating speeches with pharmaceutical entrepreneurs and to gaining in-depth insights from patient representatives demonstrating the importance of the work on therapies for rare diseases.

Sources: 

https://www.orphandrugsus.com/

Bundesgesundheitsministerium: Seltene Erkrankungen