As a biochemist and public health expert, I have witnessed officials turning a blind eye to the needs, concerns, and voices of families caring for the nearly 30 million Americans — half of them children — with debilitating and terminal rare conditions.These patients and families have a simple, reasonable demand of the FDA — access to new treatments that offer the hope of longer and more productive lives. But rather than clearing the path, the FDA keeps blocking the gate with cumbersome bureaucracy.
GOP ATTORNEYS GENERAL DEMAND ABORTION PILL WATER SAFETY TESTS FROM EPA
Many critical decisions affecting whether treatments reach patients in a timely manner are influenced by FDA Advisory Committees, or “AdComms.” These panels are comprised of experts from outside FDA who evaluate the safety and efficacy of new therapies, particularly those involving emerging technologies.
The AdComms carry enormous influence. Their conclusions shape regulatory decisions and can determine whether patients gain access to potentially life-saving treatments.
For families battling rare and often fatal diseases, these decisions are a matter of life and death. Patients are dying while committees debate. That is why the entire AdComm structure requires urgent reform.
