The Rare Disease Congressional Caucus has named Congresswoman Doris Matsui (CA-6) as co-chair, replacing retiring co-chair G. K. Butterfield (NC-1). Matsui will join fellow caucus co-chairs Gus Bilirakis (FL-12) and Senators Roger Wicker (MS) and Amy Klobuchar (MN) in the 118th Congress. With 186 members, the Rare Disease Congressional Caucus is a bipartisan, bicameral caucus, providing a platform to educate and engage Members of Congress on policy issues impacting the rare disease community. The announcement was made during a Rare Disease Congressional Caucus briefing on the Orphan Drug Act of 1983. Approaching its 40th anniversary in January, strengthening and protecting the law’s incentives is anticipated to be a major rare disease community focus in 2023.
“We are so grateful to Congressman Butterfield for his dedication and service to the rare disease community and welcome incoming co-chair Congresswoman Matsui. She is personally committed to elevating the patient voice and closing expansive gaps in access to treatments,” said Julia Jenkins, EveryLife Foundation of Rare Disease Executive Director. “We are excited to partner with Congresswoman Matsui to help improve policy affecting rare patients.”
A strong proponent of incorporating the patient voice in drug development and improving access to treatment for rare and ultra-rare patients, Matsui introduced two vital rare disease bills during the 117th Congress. The first bill, the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act would provide patients and patient advocates the ability to play a larger role in the Food and Drug Administration’s (FDA) benefit-risk framework for drug approval. The second bill, the Access to Rare Indications Act of 2021 would support patients with rare and ultra-rare diseases in getting evidence-based, medically necessary care covered by their health insurance. Matsui was honored by the EveryLife Foundation in 2014 with a RareVoice Abbey Award.
“Access to the best medical care is crucial for patients living with rare and ultra-rare diseases, and that is why I have long made it a priority to work on policies that will advance the science of rare disease therapies and expand access to care,” said Congresswoman Matsui. “For the millions of Americans with rare diseases, we can and must do better. That’s why I am proud to co-chair the Rare Disease Congressional Caucus and continue to advocate for our community of rare disease patients and their families. I thank Rep. Butterfield for his years of advocacy and service, and I am ready to get to work on these important issues in the new Congress.”
In addition, Matsui has worked to improve access to high-quality, affordable, accessible health care and was instrumental in crafting the Affordable Care Act (ACA), as a member of the Energy and Commerce Committee. She also serves on the bipartisan Telehealth Working Group, helping to open reimbursement for telehealth within the Medicare program.
