Introduction of the Rare Disease Advancement, Research, and Education (Rare) Act

Floor Speech

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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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