RDCC Urges Congress to Reject Proposals that Undermine the Intent of Accelerated Approval for Rare Disease Patients

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:

• Urging the U.S. Department of Health and Human Services (HHS) not to move forward with the Accelerating Clinical Evidence Model as it will create a limiting barrier to continued investment for accelerated approval therapies 
• Rejecting the Medicaid and CHIP Payment and Access Commission’s (MACPAC) recommendation for state adoption of Medicare’s national coverage determination (NCD) as it discredits the FDA’s authority in determining a drug’s safety and effectiveness.

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.