USA – Experts address clinical challenges for ultra-rare diseases at BIO

A panel of experts at BIO Digital on Wednesday discussed some of the challenges and regulatory considerations for sponsors developing treatments for ultra-rare diseases where clinical trials could involve a very small number of patients.

When asked where companies should begin their development for extremely rare diseases, Brad Glasscock, global vice president and head of global regulatory affairs at BioMarin said, “First and foremost I think is understanding whether anybody has been there before. Is there past precedent available, either directly in the disease of interest or a related disease,” and emphasized the importance of natural history data and understanding the patient perspective.

Glasscock said that FDA’s recent guidance on patient-focused drug development has helped integrate the patient perspective into BioMarin’s product development strategy.

“We’re seeing that play a bigger part in our regulatory strategies and bringing it earlier into the process so that we ensure we’re developing drugs and interventions that are most responsive to the needs and desires of the patient community,” he said…