Skip to main content
Home » Rare Diseases » Accelerated Approval Delivers Hope for Rare Disease Patients
Rare Diseases

Accelerated Approval Delivers Hope for Rare Disease Patients

aap-accelerated approval pathway-fda-raap-rare disease-rare-treatment
aap-accelerated approval pathway-fda-raap-rare disease-rare-treatment

The Accelerated Approval Pathway (AAP) has delivered innovative therapies to rare disease patients for three decades. 

However, the recent and controversial approval by the Food and Drug Administration (FDA) of an Alzheimer’s therapy could lead Congress to reevaluate the program. Any reforms must preserve and strengthen this important tool for innovation, not just throw out the baby with the bathwater. 


In 1992, the FDA, Congress, and the public joined forces to give the FDA flexibility in the drug approval process to expedite scientific discoveries into treatments

This AAP is designed to carefully speed access to new drugs, while preserving the FDA’s gold standards for safety and efficacy — the same approval standard as all approvals. Accelerated approval simply permits the FDA to accept a different type of data for approval. In exchange, the drug company must conduct post-approval studies to confirm the expected benefit.

In the United States, a rare disease is one that affects fewer than 200,000 people. According to the National Institutes of Health, there are thousands of rare diseases that together affect as many as 30 million Americans. 

These are often serious or life-threatening diseases — 80 percent are genetic in origin, 50 percent impact children, and 30 percent of those children will not live to see their fifth birthday. Yet, there are only approximately 700 drugs currently approved for rare diseases.

Overcoming challenges

Developing drugs for rare diseases can be challenging due to specific characteristics such as small heterogeneous patient populations, long timeframes for disease progression, a poor understanding of disease natural history, and a lack of prior clinical studies. Therefore, the AAP pathway is an essential tool used to bring new treatments to patients living with a rare disease.  

By relying on surrogate and intermediate clinical endpoints, sponsors can bring therapies to market under shorter timeframes, with less cost and smaller participant groups than traditional clinical trials, and patients can have earlier access to these medicines.

We at the Rare Access Action Project (RAAP), agree with Dr. Richard Pazdur of FDA’s Oncology Center of Excellence when he says that instead of attacking the program, we need to address “what’s going right” with it. There have been 165 accelerated approvals over the past 10 years, with only about 10 therapies removed from the market. 

As Congress considers FDA’s user fee programs, which are up for reauthorization this year, it is likely the AAP will also be a key topic of debate. Let’s not allow the recent controversies obscure the overall dramatic successes of the program.

Without accelerated approval, many with rare diseases would be left with few, if any, treatments because traditional clinical trials would be nearly impossible to conduct. 

As the 30-year anniversary of AAP approaches, RAAP urges that any potential changes be designed to strengthen the areas of success and reinforce tools that offer patients access to medicines that can treat serious and life-threatening rare diseases and conditions.

Next article