The RDCC sent a letter to Congress opposing recent policy proposals that undermine the intent of the accelerated approval pathway, and would limit patient access to life-saving treatments for rare disease patients. Any future policy reforms to accelerated approval should protect patient access, build upon Congress’ recent steps to boost FDA oversight of accelerated approval products, leverage and enhance its existing authorities, and protect the integrity of this critical pathway.
The RDCC calls on Congress to reject these proposals by:
For 30 years, the accelerated approval pathway has served as a critical lifeline for rare disease patients, leading to significant advances in the treatment of serious, life-threatening diseases. Congress and the FDA have regularly reaffirmed that medical products approved through the accelerated approval pathway meet the nation’s stringent standards for safety and effectiveness. The policy proposals currently being brought forward would upend past progress and prevent future discovery.
Click here to download and read the letter sent to Members of key Congressional committees.