[Congressional Record Volume 164, Number 35 (Tuesday, February 27, 2018)]
[Extensions of Remarks]
[Page E232]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]




 INTRODUCTION OF THE RARE DISEASE ADVANCEMENT, RESEARCH, AND EDUCATION 
                               (RARE) ACT

                                 ______
                                 

                           HON. ANDRE CARSON

                               of indiana

                    in the house of representatives

                       Tuesday, February 27, 2018

  Mr. CARSON of Indiana. Mr. Speaker, today I rise to introduce the 
Rare disease Advancement, Research, and Education (RARE) Act. This 
important, bipartisan legislation would address many of the issues 
facing rare disease patients and families. Today, Rare Disease Day, 
many of these individuals are sharing their stories and educating 
communities of researchers, health professionals, governments, 
families, and friends about how rare diseases affect them. I am proud 
to introduce the RARE Act not only in recognition of these efforts, but 
moreover to address the many unique challenges facing the rare disease 
patient community.
  Almost 7,000 rare diseases affect nearly 30,000,000 Americans, and 
many lack an effective treatment. There remains much Congress can do to 
improve rare disease awareness, education, research, surveillance, 
diagnosis, and treatment, and to address common challenges faced by 
rare disease patients.
  Over the course of my time in Congress, I have met with countless 
rare disease patients and their families. Each story is unique--and in 
many cases heartbreaking--but it is essential that Congress address 
some of the root causes behind these stories.
  Rare diseases cross the medical spectrum, but individuals with rare 
diseases face some common challenges. In large part due to their 
limited patient population size, these individuals may have difficulty 
obtaining an accurate diagnosis, finding physicians or treatment 
centers with expertise in their disease, and ultimately finding 
appropriate treatment options and cures. While over 450 drugs have been 
approved for the treatment of rare diseases, millions of Americans 
suffering from a rare disease have no approved treatment options.
  For decades, Congress has worked to support advancements in rare 
disease, with legislation ranging from the Orphan Drug Act to the Food 
and Drug Administration Safety and Innovation Act to most recently, the 
21st Century Cures Act. In addition, the Food and Drug Administration 
and the National Institutes of Health have made important strides, 
prioritizing research, development, and approval of treatment targeting 
rare diseases. Nonetheless, it remains clear that there remains much 
policymakers can do to further these critical efforts.
  The RARE Act would represent an important step in this direction, 
addressing some of the commonalities between rare diseases and aiming 
to improve rare disease treatment, research, and diagnostics.
  First, the RARE Act would expand our understanding of rare diseases 
by enhancing an existing and successful program: the Rare Diseases 
Clinical Research Network. The Rare Diseases Clinical Research Network 
at the National Institutes of Health (NIH) is made up of 21 research 
`centers of excellence' studying rare diseases in an interdisciplinary 
way, working with patients and others on clinical studies and other 
research.
  Second, the RARE Act would fill critical gaps in our healthcare 
system, improving coordination, surveillance, and awareness of rare 
diseases. For example, the RARE Act would require the Centers for 
Disease Control (CDC) to create a National Rare Disease or Condition 
Surveillance System. Modelled off of similar systems for other 
conditions, this formalized infrastructure would track rare disease 
data, helping researchers to understand commonalities between diseases. 
The RARE Act would also require the Agency for Healthcare Research and 
Quality (AHRQ) to expand and intensify its work to ensure that health 
professionals are aware of rare disease diagnoses and treatments. And 
it would mandate an updated report on rare disease efforts from the 
National Academies of Sciences, Engineering, and Medicine, to ensure 
that Congress has the best tools possible to address these issues.
  Passage of the RARE Act would represent a major stride in combating 
these unique diseases and conditions, and enhance the federal 
government, non-profit, and private sectors capacities to improve 
patients' lives. I urge my colleagues to join me in supporting the RARE 
Act.

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