Mr. President, Senate Leaders, and Members of Congress:
We write to you not as political activists or industry insiders, but as patients and families living with terminal rare diseases that are hurting us and the people we love.
We represent families battling Duchenne muscular dystrophy, Sanfilippo syndrome and Huntington’s disease. Our diseases differ, but we share the same devastating reality: as loved ones decline, the Food and Drug Administration (FDA) is not delivering on its promise to bring forward life-saving therapies in the timeframe needed to combat these diseases.
Prior to now-former FDA Commissioner Dr. Martin Makary leaving the agency on May 12, the U.S. Senate Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies had scheduled a statutory FDA budget hearing for May 13.
Although the hearing was postponed due to Dr. Makary’s departure, FDA leadership is still accountable to the Administration, Congress, and the public as to whether or not the FDA’s $7.23 billion 2027 budget request will see funds allocated in a manner that provides the rare disease community with hope.
The FDA Commissioner holds one of the most important public health positions in the world, particularly when it comes to approving therapies for patients with rare and often fatal diseases. Nearly 30 million Americans live with a rare disease, and approximately half of those are children.
Decisions made by departing FDA Commissioner Makary slowed the pace of scientific review, access to treatment, and the future of medical innovation in the United States. The Commissioner is entrusted not only with protecting public health, but with ensuring that patients facing terminal illness are not left behind by unnecessary regulatory delay. It is critical that the next individual appointed to this role have a deep understanding of the urgent needs of all patients and a desire to meet those needs with innovation and transparency.
For many rare disease families, those responsibilities have not been adequately fulfilled.
Angelina has already buried two brothers, Angelo and Antonio, who died from Duchenne muscular dystrophy. Today, she watches her 14-year-old son, Ryu Antonio Olivera, slowly lose the ability to move, breathe, and care for himself while suffering from the same disease. Every night, he depends on breathing machines to survive.
Rachel watched Huntington’s disease destroy her grandmother and devastate her father physically and mentally before he ultimately took his own life. Last year, Rachel tested positive for the Huntington’s disease gene. Without treatment, she now faces the possibility of the same fate endured by prior generations of her family.
Abby is reliving history as her daughter Lottie battles Sanfilippo syndrome, the same condition that took the life of her cousin, Samantha, nearly 20 years ago at the age of eleven. Lottie, who also has Down syndrome, will not qualify for most clinical trials, so an FDA approved treatment may be her only chance.
We are united by one undeniable truth: rare disease families do not have time for bureaucracy.
Every month without treatment means more muscle loss, neurological decline, irreversible damage, and too often, more funerals. The FDA may view delay as “procedural caution,” but rare disease families experience it as suffering and loss while therapies remain tied up in months or years of unexplained review.
Mr. President, many in the rare disease community deeply appreciate your leadership in signing the Right to Try Act into law. You recognized that Americans who are terminally ill or have exhausted traditional treatments deserve the ability to make informed decisions about their own care when no meaningful alternatives exist.
The greatest risk we face as a community is the risk of not having access to treatments.
That same urgency contained in the Right To Try Act is desperately needed today for rare disease patients whose therapies remain trapped in prolonged regulatory review. We are asking the FDA to fully utilize accelerated approval pathways and provide rare disease families a right to try potentially life-extending treatments.
The FDA has recently left many families feeling abandoned by the very agency entrusted with protecting them. Promising therapies for Duchenne muscular dystrophy, Huntington’s disease, Sanfilippo syndrome, Hunter syndrome, and many other rare diseases continue to face shifting goalposts, delays, and regulatory paralysis while patients deteriorate in real time.
Traditional regulatory timelines and risk assessments were never designed for diseases where children may live only into their teenage years or early adulthood.
Mr. President, your leadership has often been defined by decisiveness and a willingness to cut through bureaucracy when Americans are suffering. Rare disease families need that leadership now as you consider a new FDA Commissioner.
We respectfully ask, Mr. President, that you and our Congressional leaders demand accountability and urgency from FDA leadership. Behind every delayed approval, blocked therapy, and additional year of bureaucracy is a patient, child, or parent running out of time.
As Dr. Makary departs the FDA and Kyle Diamantas serves as interim Commissioner, there is a new opportunity to ensure that the next generation of FDA leadership, including directors of the Center for Biologics Evaluation and Research - recently vacated after the firing of Dr. Vinay Prassad - and the Center for Drug Evaluation and Research, which is also under an acting director.
Rare disease families are asking for the opportunity to access treatments that could help save or extend the lives of the people they love, and we hope new leadership is guided by a rare disease-centered standard:
Accelerate cures. Listen to families. Deliver urgency for rare diseases. Put rare disease patients first — with speed, transparency, and action.
With best regards,
Angelina Olivera, Mother and Duchenne Muscular Dystrophy Advocate
Rachel Reising, Huntington’s Disease Advocate positive for HD
Abby Milburn, Mother and Sanfilippo Syndrome and Down Syndrome Advocate
