In recent decades, we have made incredible progress in the fight against rare diseases. But we still have a long way to go: Today, 30 million people in our country are living with one of the 7,000 known rare diseases.
Behcet’s disease, also called Behcet’s syndrome, is a rare disease affecting approximately 20,000 patients like Dustin in our country. It causes blood vessel inflammation throughout the body, and can affect the mouth, eyes, skin, joints, vascular system, digestive system, and brain. Complications of the disease can be severe, and even life-threatening. Treatments are available to manage symptoms, but a cure has yet to be discovered. Developing medicines for patients with rare disease like Behcet’s is one of the greatest health challenges of our time. The causes of these diseases are often complex, making it difficult to design and implement research strategies. Additionally, because the population of patients with rare diseases is relatively small, recruiting for clinical studies can be difficult. Still, researchers and scientists continue their quest to find new treatments for patients with rare diseases.
In 1992, Congress passed the Prescription Drug User Fee Act (PDUFA), allowing the Food and Drug Administration (FDA) to collect fees from drug manufacturers to supplement Congressional funding in order to ensure the timely review of new medicines. With these additional resources, the FDA continues to enhance the drug review process, ultimately enabling innovative, safe, and effective drugs to reach patients more quickly. For nearly 25 years, PDUFA has helped increase the number of treatments available to patients like Dustin who are living with rare diseases, while researchers and scientists continue to search for cures. With more than 560 medicines in the pipeline for rare diseases, it will be critical that FDA is equipped to keep up with the rapid pace of treatment innovation. Every five years, PDUFA must be reauthorized by Congress. PDUFA will expire in September 2017. For the millions of Americans like Dustin who are living with rare diseases, the reauthorization of PDUFA next year may support the availability of new and innovative treatments. In addition, PDUFA reauthorization could mean the difference between life and death for millions of other patients with life-threatening diseases such as HIV/AIDS and cancer.