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Despite Healthcare Advances, Challenges with Treating and Curing Rare Diseases Persist as IRA and FDA Policies Create Legislative and Regulatory Hurdles

The WHIC's discussion about rare diseases and the promise and the hurdles was both powerful and disheartening.

Washington D.C. -- 

The Washington Health Innovation Council met recently at the headquarters of the Advanced Medical Technology Association (AdvaMed), to learn and discuss advancing rare disease treatment and federal policy headwinds. Discussion included IRA price controls, the “federal march-in” proposal, and FDA regulatory barriers.

WHIC attendees appreciated Pat Fogarty, Deputy GC, AdvaMed welcoming the group and spending time highlighting a number of initiatives. He complemented the WHIC and reinforced AdvaMed’s commitment as a partner to the WHIC. He highlighted the upcoming 2024 MedTech Conference and the focus on AI-powered diagnostics and therapeutic devices. Helping veterans get mentored and given counseling and resources to help them transition into civilian life and specifically into the medtech community. He also thanked the group for highlighted the industry’s unique program (below).

The entire discussion about rare diseases and the promise and the hurdles was both powerful and disheartening. We heard from a rare disease parent, advocate and former senior HHS official, Judy Stecker (Sr. VP Hill & Knowlton, US Healthcare Media and Public Affairs) on the impact of federal policies on rare diseases R&D and regulatory approvals. She discussed the realities of her family’s situation and a deep knowledge of CLN3 and treatments available - and not yet available, noting that we are “still on the cusp of gene and biologic therapies.”

  • Judy complemented the leadership of CBER Director Dr Peter Marks and called him a coach.

  • Judy also pointedly said that “no one gets fired for not approving a drug” and there needs to be “some rational flexibility; yes, the mission is to protect public health, but protect the public’s health by ensuring access to technologies that exist today.”

There was fantastic back and forth with attendees including Eric Schlorff from SeaStar Medical who discussed his journey with the FDA in receiving a Humanitarian Device Exemption. Judy responded: “If we don’t figure out how to work in these realms… then we are going to lose innovation and lose lives.” 

Here is her recent Wall Street Journal article bravely threading the needle on calling for change and a new perspective while respecting the missions and work of the FDA in “The FDA Could Help Save My Son From a Rare Disease - WSJ

Judy’s journey, work at HHS, and interactions with the FDA and NIH has turned her into a leading Beyond Battens Disease Foundation advocate.

A representative of the Prevent Blindness discussed the ASPECT program - a resource teaching families how to advocate and empower and communicate their lived experience to ensure they receive what’s necessary to accommodate them - new treatment, clinical trials, to making sidewalks in their hometown more accessible.

Help them find safe spaces to talk about their needs; and supporting a child navigating vision loss.

AstraZeneca - The group heard perspectives from Sarah Arbes, Head of Federal Affairs and Policy at AstraZeneca about the concerns of the innovator community of an unintended consequence of the new Medicare drug pricing law that may lead manufacturers to consider first launching new products that treat rare diseases outside of the US. Sarah also discussed her company’s commitment to R&D to help create and commercialize an increasing number of medicines.

James Pinkerton, a former White House official in two administrations, highlighted how the problems he was hearing are symptomatic of a bureaucratic regime that incentivizes inaction vs problem solving. He discussed points he makes in his soon-to-be-released book about how states have an opportunity with AI-driven healthcare to move beyond Washington and find a home in states to be developed and approved/regulated.

The Washington Health Innovation Council thanks our guest speakers and attendees for a thoughtful dialogue and ongoing commitment to advocate for patients and innovations in healthcare.

For questions or more information about the topics presented here, or joining the Washington Health Innovation Council, contact our team today.


Speaker Resources





AdvaMed Alliance of Healthcare Sharing Ministries

American Academy of Ophthalmology

Beyond Batten Foundation  Boston Scientific 

DigitalDx Ventures 


Hill & Knowlton / Wheelers Warriors


Leavitt Partners, LLC


Nahigian Strategies

National Pharmaceutical Council

Prevent Blindness

Strategies IPS

Sullivan & Cromwell LLP

The Herald Group US Chamber of Commerce


JK Strategies 


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