Rare Disease Week 2026: Senate Hearing Recap

What a way to head into the final days of Rare Disease Week 2026! Despite a little drizzle and some gray clouds, hundreds of rare disease advocates participated in more than 300 Hill visits on Thursday to share their stories and advocate for rare disease policies.

Yesterday started with our community in the Senate spotlight. The EveryLife Foundation for Rare Diseases was honored to help represent the community by participating in the Senate Special Committee on Aging’s hearing on the effects of FDA delays on rare disease therapy development. Annie Kennedy, our Chief Mission Officer, joined other expert witnesses, Dr. Jeremy Schmahmann, Director of Massachusetts General Hospital Ataxia Center; Bradley Campbell, President & CEO of Amicus Therapeutics; and Dr. Cara O’Neill, Chief Science Officer and Co-Founder of the Cure Sanfilippo Foundation to give expert testimony.

We would like to extend our deepest gratitude to Chairman Scott, Ranking Member Gillibrand, Senator Johnson, Senator McCormick, Senator Alsobrooks, and Senator Kim for the important dialogue in this hearing.

Inside the Hearing

“We are here today because Congressional action is needed to ensure that this generation of patients will benefit from our existing rare disease treatment pipeline.” – Annie Kennedy

With a hearing room full of rare disease patients and families, the hearing included remarks from Senators, witness testimony and questions, and highlighted the importance of using all available tools that Congress has given the FDA to ensure predictable and consistent approaches to reviewing rare disease therapies.

Gratitude for Congress’ and the FDA’s Rare Disease Actions

“Congress has been clear on an overwhelmingly bipartisan basis. We have given the FDA flexibility to move faster for patients with serious and life-threatening conditions.” – Chairman Scott

• The expert witnesses continually noted the importance of Congressional actions that have given the FDA flexibilities that have historically aided the rare disease community.

• It is because of Congressional actions that tools like the accelerated approval pathway and advisory committees exist. These are integral to the process of rare disease therapy development.
  
• Witnesses reinforced the community’s gratitude to thousands of dedicated career professionals at the FDA who, over decades, have been partners in the fight to accelerate rare disease regulatory innovation.

A number of issues around regulatory delays in rare disease therapy development were explored, such as:

Not Utilizing Existing Tools

“Every regulatory flexibility must be leveraged to meet this uniquely urgent need.” – Dr. Cara O’Neill

• Since 2025, the FDA has issued at least 23 Continuing Resolution Letters (CRLs) that have delayed and halted the development of rare disease therapies. At the same time, the use of advisory committees declined by 65% compared to 2024, so external experts and patient perspectives were left out of the process.
• Recent decisions suggest the agency has backed away from using these powers, despite Congress granting the FDA broad flexibility to apply a tailored approach when evaluating rare disease therapies through tools like the accelerated approval pathway that uses surrogate biomarkers to speed therapies to patients, and the use of innovative clinical trial designs.
• With the use of advisory committees declining in the past year, expert witnesses illuminated the need for this to remain a consistent tool. As Annie mentioned, “Advisory committees don’t always vote yes; that’s not the point. The point is for external experts, including clinicians, including those with statistical expertise, manufacturing expertise that are not always internal to the agency, to be brought to bear on the regulatory decisions.”
• Mr. Campbell also noted that the Rare Disease Innovation Hub is “underfunded and under-powered to do what it needs to do.” At the urging of the rare disease community, this resource was established by Congress and meant to encourage cross-center collaboration in the FDA. With resources, the Hub has the potential to further ramp up its work and improve consistency, share expertise, and grow opportunities for rare disease stakeholders to engage in the process.

The Need for Consistency and Transparency

“If we continue to create this uncertainty… I am confident that those investor dollars would go somewhere else.” – Bradley Campbell

• This hearing brought to light what is at stake when regulatory approaches in rare disease decisions aren’t predictable and consistent. With recent trends in CRLs and decreased involvement of the rare disease community, many treatment denials are met with confusion and uncertainty.
• Mr. Campbell noted that biotech companies are not being given proper guidance on how to resolve the concerns of the FDA. While CRLs are one tool in the toolbox, we’re failing to see consistent and appropriate use of alternative measures, such as different meetings enabled by PDUFA legislation.

Ensuring Ethical Practices in Rare Disease Clinical Trials

“This is a policy of death by technicality. These tiny glitches end up not producing drugs and the patients pay the consequences.” – Dr. Jeremy Schmahmann

• A recurring topic of the hearing was the concerns around ethical practices in clinical trials for rare diseases, especially progressive diseases, where every day without treatment can mean irreversible loss of function. We cannot approach these clinical trials with the same methods used for other illnesses. While placebo-controlled trial requirements are still standard, these methods often put rare patients at risk.
• When the FDA does not take real expert clinician and patient experiences into account, a drug that might have improved symptoms in ways that weren’t expected when the trial was designed, might not make it to patients.
• Dr. O’Neill, whose family is also affected by Sanfilippo, powerfully commented, “We are concerned about the ethics of animal testing, more than we are concerned about the ethics of allowing brain injuries of children in clinical trials.”

Increasing Manufacturing Transparency and Speed

“I want to raise another issue, which is just how long it takes to build any manufacturing facilities here in the United States. The level of slowness… I want that level of urgency on the manufacturing side as well.” – Senator Kim

• Mr. Campbell highlighted that “the United States has been the beacon of therapeutic innovation for decades,” but is now at risk of falling behind international competitors.

• While international manufacturing facilities can get up and running within a year or two, the typical process in the U.S. takes three to five years to build a manufacturing facility, and another two to finalize inspections. These delays result in many pharmaceutical sponsors moving their business abroad.
• To increase the urgency, consistency, and speed of manufacturing these life-saving drugs, witnesses suggested the United States can streamline the permitting process, prioritize home-grown facilities, and support manufacturing domestically.

Our message, our stories, and our urgency were heard. Conversations with the Senate Special Committee on Aging will continue in the coming days. We will keep you updated on any future Committee interactions. For now, though, thank you for continuing to show up, speak out, and use your voice on behalf of the rare disease community. You are being heard!

Watch the full hearing and read witness testimonies.