How FDA Incentives and Patient Advocacy Accelerate Rare Disease Drug Development in Biopharma (Part 2)

The field of rare-disease drug development is rapidly evolving on the back of regulatory incentives and a growing recognition of unmet patient needs. Both biotech companies and large bio/pharmaceutical firms are increasingly focusing on rare diseases, thanks to programs such as orphan drug designations, which offer benefits such as market exclusivity, reduced regulatory fees, and tax credits. These incentives help offset the traditionally high costs of developing treatments for small patient populations, says Dan Williams, CEO of SynaptixBio, in an interview with the Pharmaceutical Technology® Group.

“If you can improve a quality of life, [patients] can tell you how that will really affect them. And that kind of input is extremely valuable for moving the drug process [and] development process forward,” Williams says.

Williams notes that a significant development in the rare disease space is FDA’s priority review voucher system, particularly the Rare Pediatric Disease designation. This program awards vouchers to companies that develop drugs for rare pediatric conditions, allowing them to expedite the review process for future drugs or sell the voucher to other companies. These vouchers have fetched prices as high as $150 million, providing substantial financial returns that can be reinvested in further research, Williams explains.

Collaboration with patient advocacy groups has also become essential, he stresses, as their firsthand insights help shape drug development to better meet patient needs. This partnership not only accelerates progress but ensures that new therapies have a meaningful impact on patients’ lives.

Click above to view the interview.

Click here to watch Part 1.

About the speaker

Dan Williams, PhD, CEO, SynaptixBio

Dan Williams, PhD, studied biochemistry and physiology at the University of Dundee, after which he worked his way up to senior scientist—managing a cell research group and preclinical development. Following this, he moved to drug development, focusing on the organization and management of both manufacturing and clinical trials. Williams then moved to Adaptimmune, developing cell therapies. He set up the development groups, project managing preclinical research and clinical trials, before becoming vice-president of Research Operations. From there, he moved to Meatable as chief product officer. Williams co-founded SynaptixBio in 2021.