Patients' Access to Treatments Act of 2013

Mr. Speaker, Friday, February 28, 2014 was Rare Disease Day. And that is why I rise to let Americans know the importance of research, recognition and advocacy that is critical for individuals facing a rare disease diagnosis--and their families. The over 6000 rare disease can be characterized by a large range of disorders and symptoms, some of which are relatively common. In the United States, a disease is considered rare when it affects fewer than 200,000 persons, and 50% of rare diseases affect children.

While we have made important stride in the diagnosis and treatment of many rare diseases, far too many remind without any treatment or cure. Additionally, these individuals are too-often misdiagnosed, or given the wrong treatments due to a lack of scientific knowledge and information.

Despite these troubling facts, there is some good news. Scientific progress continues to make it possible to cure previously-untreatable conditions. For example, the Ear Foundation of Santa Barbara, California has recently done ground-breaking research into Patulous Eustachian Tube (pET) dysfunction. This rare disease occurs as a result of the Eustachian tube in the ear continuously staying open. Because symptoms include a feeling of ear fullness or oceanic roaring sounds, many physicians are unaware of the disorder and as such are unable to properly diagnose. Thanks to similar research organizations, patient groups, private foundations, and federal research leaders like the National Institute of Health (NIH), patients with rare conditions are finding increased relief.

Additionally, Congress has been making moves to encourage better coordination among researchers to develop cures and treatments for rare diseases. For example, the National Pediatric Research Network Act, a bipartisan law I co-authored with Congresswoman McMorris-Rodgers from Washington, was an important step to facilitate the creation of pediatric research consortia through NIH focused on improving pediatric disease research, with a special emphasis on rare diseases like spinal muscular atrophy (SMA). With better coordination, researchers can quickly share best practices while fostering partnerships to make research and clinical trials more accessible to those afflicted.

The next step that Congress should take to support individuals with rare diseases is to pass H.R. 460, the Patient's Access to Treatment Act (PATA), into law. This bipartisan bill that I coauthored with Congressman McKinley from West Virginia would stop insurance companies from moving vital medications--often treatment for rare disorders--into costly ``specialty tiers,'' ending a significant financial barrier for insured individuals to access the care they need. With over eighty cosponsors thus far, this bill has the opportunity to affect positive change in the lives of thousands of patients and their families. I encourage my colleagues to join me as a cosponsor of this legislation to support all Americans currently living with rare diseases and those who will be diagnosed in the future.