Axne Joins the Fight to Cure Rare Diseases

October 25, 2019
Press Release
Axne helps lead bipartisan legislation supported by over 140 patient advocacy groups to better treat and cure rare diseases

WASHINGTON – Today, U.S. Rep. Cindy Axne (IA-03) signed onto legislation that would improve treatment, research, and diagnosis of rare diseases. The Rare Diseases Advancement, Surveillance Research, and Education (RARE) Act expands the ability of the National Institutes of Health (NIH) and the Centers for Disease Control and Prevention (CDC) to improve their research, diagnosis, treatment, education, and awareness programs for rare conditions and diseases.

“I’ve met with Iowans who have these rare diseases, such as ALS, Cerebral Palsy, Ectodermal Dysplasia, Hereditary Spastic Paraplegia, Huntington’s Disease, Leukemia, and more. It is heartbreaking to meet children, mothers, fathers, grandparents, and friends who are sick and hurting, and we still don’t know why,”  said Rep. Axne. “We need to do more to accelerate innovation, develop new treatments, and better understand how and why rare diseases happen. We know that more research brings us closer to a cure.”

The RARE Act will expand the National Institutes of Health Rare Disease Clinical Research Network, which consists of 21 research centers of excellence focused on rare disease. This network supports critical research and clinical trials for over 190 rare diseases and increases the availability of rare disease information.

“The ALS Association Iowa Chapter applauds Representative Axne for cosponsoring the Rare Disease Advancement, Research, and Education (RARE) Act. This bill would provide much-needed investment in rare disease research, increase federal efforts in tracking and quantifying rare diseases, and implement rare disease outreach and awareness campaigns for physicians. We urge Congress to pass the RARE Act to accelerate therapeutic development for rare diseases like ALS” said Josh Nuss, Executive Director for the Iowa Chapter.

This legislation would require the Centers for Disease Control to create a National Rare Disease or Condition Surveillance System. The system would be modeled on similar systems for other conditions and used to track rare disease data. This centralized database helps researchers understand commonalities between diseases and unusual symptoms.

It would also require the Agency for Healthcare Research and Quality to expand and intensify its work to ensure that health professionals are aware of rare disease diagnoses and treatments.

Research is vital to ensuring we continue to be a world leader in the treatment and education of diseases, particularly those which are understudied. As our faculty and students engage in research, we emphasize the objective of creating positive changes in the health of entire communities. That same fundamental principle applies to the RARE Act,” stated Mercy College of Health Sciences President, Douglas J. Fiore, PhD.

“Understanding, treating, and preventing diseases is a necessary part of good health care. I came to Washington to fight the high cost of prescription drugs, increase access to quality healthcare, and work to ensure our most vulnerable have the care and treatment they need. The RARE Act is one more piece of helping Iowans and improving our care,” Axne said.

Congresswoman Axne is a member of the Congressional Health Care Task Force and the Rare Disease Caucus. She has introduced legislation that would protect Iowans from surprise bills, and is committed to expanding access to quality, affordable health care, lowering the cost of prescription drugs, and ensuring those with preexisting conditions are covered by their insurance.