[Congressional Record (Bound Edition), Volume 160 (2014), Part 3]
[Extensions of Remarks]
[Page 3737]
[From the U.S. Government Publishing Office, www.gpo.gov]




          RECOGNIZING FEBRUARY 28TH, 2014 AS RARE DISEASE DAY

                                  _____
                                 

                            HON. LOIS CAPPS

                             of california

                    in the house of representatives

                         Tuesday, March 4, 2014

  Mrs. CAPPS. 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 6,000 
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 strides in the diagnosis and treatment 
of many rare diseases, far too many remain 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.

                          ____________________