September 9, 2019 - Issue: Vol. 165, No. 143 — Daily Edition116th Congress (2019 - 2020) - 1st Session
INTRODUCTION OF THE RARE DISEASE ADVANCEMENT, RESEARCH, AND EDUCATION (RARE) ACT, H.R. 4228; Congressional Record Vol. 165, No. 143
(Extensions of Remarks - September 09, 2019)
Text available as:
Formatting necessary for an accurate reading of this text may be shown by tags (e.g., <DELETED> or <BOLD>) or may be missing from this TXT display. For complete and accurate display of this text, see the PDF.
[Extensions of Remarks] [Pages E1107-E1108] From the Congressional Record Online through the Government Publishing Office [www.gpo.gov] INTRODUCTION OF THE RARE DISEASE ADVANCEMENT, RESEARCH, AND EDUCATION (RARE) ACT, H.R. 4228 ______ HON. ANDRE CARSON of indiana in the house of representatives Monday, September 9, 2019 Mr. CARSON of Indiana. Madam Speaker, I am pleased to reintroduce the Rare disease Advancement, Research, and Education (RARE) Act. This important, bipartisan legislation will address many of the issues facing rare disease patients and families. I am pleased that this legislation has been endorsed by 139 patient groups and cosponsored by a number of my colleagues from both sides of the aisle. But most importantly, this legislation will make a meaningful difference in the lives of those struggling with rare diseases by using increased research to help provide more accurate diagnoses and increased treatment options. During my time in Congress, I have been honored to represent and meet with many brave Hoosier families that are struggling with rare diseases. I have been moved by their courage. Their strength in the midst of trying conditions is not only inspiring, but also instructive. They have educated me and my colleagues about the necessity of increased research and rare disease surveillance in order to provide more treatment options and better diagnoses. One family in Indiana, the Meggenhofens, exemplify the challenges of accurately diagnosing and treating rare diseases. Jocelyn Meggenhofen was born with Leukodystrophy, an extremely rare brain disease that causes delays in cognitive development and poor motor skills. After years of seizures, misdiagnoses of a brain tumor, and denials from residential facilities, Jocelyn was finally able to receive the correct treatment. Her struggles were not over: the Meggenhofen's health insurance would not cover room and board for an inpatient stay at a facility in New Jersey and her school in Hancock County would not approve Jocelyn's education at the facility. However, despite being told by doctors that she wouldn't live past her fifth birthday, Jocelyn, now 15, has received treatment at Riley Children's Health hospital in Indianapolis and continues to fight her disease. In another example, Derrian Baker in Merrillville, Indiana suffered from Prader Willi Syndrome, a very rare genetic disorder. During his short life, Derrian and his family struggled to receive the necessary treatment after being denied an inpatient stay for treatment at the Children's Institute in Pittsburgh. Derrian passed away at the age of 26, underscoring the severity and high morbidity of many rare diseases if they cannot be treated. Unfortunately, the plights of people like Jocelyn and Derrian are not uncommon: Nearly one in ten Americans live with one or more of the roughly 7,000 known rare diseases. These largely inherited diseases-- defined as affecting 200,000 or fewer people--often lack substantive research investments and treatment options. In particular, African- Americans are especially vulnerable to certain rare diseases, including Sickle cell disease and beta-thalassemia. Specifically, the blood disorder Sickle cell disease affects 73 out of every 1,000 African American babies versus only three out of every 1,000 Caucasian babies. While rare diseases cross the medical spectrum, individuals with rare diseases face some common challenges. Largely 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. Moreover, it can be difficult to find patients for treatment studies, underscored by the example of Maria Isabel Bueso, who came to the U.S. from Guatemala in order to participate in a clinical trial. Thanks to her participation, a treatment for her rare disease, Mucopolysaccharidosis VI, was approved. Maria now faces the possibility of deportation because the current administration eliminated a program that allows immigrants like Maria to stay in the country while receiving lifesaving medical treatment. Frighteningly, roughly 90 percent of rare diseases still lack a treatment [[Page E1108]] approved by the U.S. Food and Drug Administration (FDA). While over 450 drugs have been approved for the treatment of rare diseases, millions of Americans who are suffering from a rare disease have no approved treatment options. Past Congressional action has helped support research at NIH and CDC, supported in part by the bipartisan appropriations letter I lead each year--signed by over 220 House members--in support of increased NIH funding. However, much more work needs to be done to help these agencies improve rare disease awareness, education, research, surveillance, diagnosis, and treatment. This is why the RARE Act is so important. It will expand the ability of the National Institutes of Health (NIH) and Centers for Disease Control and Prevention (CDC) to study rare diseases by improving treatment, research, and diagnostics of rare diseases through new and existing programs. I am proud to introduce the RARE Act to help address the many unique challenges facing the rare disease patient community, including patients like Jocelyn and Derrian. The RARE Act would provide an important step forward by addressing some of the common challenges faced by rare disease patients and improving rare disease treatment, research, and diagnostics. The RARE Act would expand an existing and successful program at NIH: the Rare Diseases Clinical Research Network (RDCRN). The RDCRN's 21 research ``centers of excellence'' support the research and clinical trials of over 190 rare diseases and increase the availability of rare disease information to doctors and patients. Expanding these centers, which are similar to the center that helped find an accurate diagnosis for Jocelyn, would help many more struggling patients to receive more accurate early diagnoses and treatments. The RARE Act would also fill critical gaps in our healthcare system by 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. This formalized infrastructure would track rare disease data and help researchers to understand commonalities between diseases and possible treatments, ultimately helping patients like Derrian to find better treatments. 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, leading to fewer misdiagnoses like Jocelyn experienced. The RARE Act would also 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. Madam Speaker, I hope my colleagues will join me in supporting this bill to help combat rare diseases. The stories of Jocelyn and Derrian remind us that we need further research and disease surveillance to improve rare disease patients' lives in Indiana and across the nation. I urge the House to support this bill. ____________________