Chairman Rick Scott and Ranking Member Kirsten Gillibrand of the U.S. Senate Special Committee on Aging have sent a letter to Food and Drug Administration (FDA) Commissioner Dr. Martin Makary. The letter informs the commissioner that the committee will hold a hearing to review how the FDA authorizes treatments for rare diseases.
The focus of the hearing will be on the development process for drugs aimed at rare diseases, examining regulatory challenges and considering ways in which the FDA can encourage innovation while ensuring safety and scientific rigor.
In their letter, Scott and Gillibrand highlighted that more than 30 million Americans are living with rare diseases, most of whom do not have access to approved therapies. They requested information from Commissioner Makary regarding what steps the FDA is taking to address these issues as part of ongoing efforts to support patients and their families.
"More than 30 million Americans are affected by rare diseases, the vast majority of which have no approved therapies," wrote Chairman Scott and Ranking Member Gillibrand. "We ask you to outline the FDA’s efforts to find solutions as we work together to turn hope into reality for these patients and their families."
The upcoming hearing aims to bring attention to these challenges within drug development for rare diseases and seeks input from stakeholders on potential improvements in regulatory processes.
