Scott Schliebner, Vice President, Scientific Affairs, Rare Diseases, Federal Programs at PRA Health Sciences gave us some of his thoughts on how the sector will develop in the coming year ahead of Rare Disease Day.
What do you think will be the biggest hurdle to the development of treatments for rare diseases in the next year?
Multi-stakeholder collaboration is critical for rare disease drug development, and the industry is recognizing the need to include many voices in the process. Achieving consensus – amongst industry, regulators, payers, and patients – while moving drug development forward rapidly, will be our next challenge as a community.
What do you predict will be the biggest thing to shake up the sector this year?
With the mostly favorable financial markets, I believe we will continue to see more acquisitions and consolidation involving rare disease-focused biotech start-ups. Look for large and medium-sized pharma to expand into the rare disease space by acquiring technology and assets being developed in the start-up space.
How do you feel the relationship between industry/academics and patient advocacy groups will develop?
I look to leaders in the space to set examples for others to follow. Long-term, transparent, honest, and mutually beneficial partnerships between industry and advocacy groups help us all achieve our goal of bringing treatments to rare disease patients faster.
The Challenge was a fantastic, engaging, and creative way to build awareness, become involved, and help make a difference for those suffering from ALS. We had great participation in our organization, from our executives down to project teams on the ground.
What are your plans for Rare Disease Day 2015?
I will be meeting with drug developers to create a clinical development plan for a new treatment for ataxia.
The World Orphan Drug Congress will be taking place in Geneva on 12-13 November; we hope you will join us. Visit the website for more information.