Premier Research is sponsoring the Orphan & Rare Disease session track at this year’s BIO International Convention, June 3-6 in Philadelphia. Our own Angi Robinson, vice president for clinical development services in rare diseases and pediatrics, will participate in the panel discussion Rare Finds: Innovative Clinical Trial Design for Rare and Orphan Diseases on June 6 at 10:30 a.m.
“Whether it’s recruiting from small populations, deriving conclusions from limited data, or helping sponsors attain orphan drug status, we’re well-attuned to the challenges that are unique to development of rare disease drugs,” Robinson said.
Even as regulators have incentivized development of orphan drugs, major obstacles remain. One of the biggest is designing effective clinical trials for newly defined diseases and those with extremely small patient populations and few or no established endpoints. Robinson will join two biotech colleagues to discuss strategies for identifying patients, gathering data, and identifying new biomarkers to serve as clinical endpoints.
The session will address knowledge gaps, trial design innovations, engaging patients and their advocates, and the significant work that remains to treat rare diseases. Additional Premier experts will also be present at BIO and available to discuss all aspects of rare disease clinical development.
“Developing drugs to treat rare disease is a race against time, and BIO 2019 is an ideal opportunity to meet with thought leaders who are at the forefront of driving clinical research toward achievement of treatment breakthroughs,” Robinson said.
Premier Research’s rare disease experience spans all aspects of clinical trials and drug development, encompassing more than 140 rare diseases across 50-plus indications in the past five years. Our deep connections with advocacy groups help us locate hard-to-find patients, and our long experience has taught us it’s important to treat them like people, not subjects.
Drawing on our expertise, sponsors are better positioned to get their products into clinical development — and ultimately to the patients who need them. If you are attending BIO please come by booth 4049 and meet our experts.