Congress Just Asked: Will America Still Lead in Cures? Here’s What They Need to Hear.
By Rare and Relentless
“Every family here today is here because science once felt impossible—and then delivered.”
—Sen. Patty Murray, Senate Appropriations Hearing on Biomedical Research, April 2025
This week on Capitol Hill, the Senate Appropriations Committee convened a pivotal hearing: "Biomedical Research: Keeping America’s Edge in Innovation." The title alone should make anyone in the rare disease community sit up. Because for families fighting for survival—like those facing classic galactosemia—innovation isn’t a luxury. It’s oxygen.
The question Senators asked was simple: Can the U.S. maintain its global leadership in cures, breakthroughs, and biotech ingenuity?
The answer, right now, teeters on the edge of collapse.
A Fractured Foundation: NIH, FDA, CMS in Crisis
Witness after witness at the hearing confirmed what families already know: America’s biomedical engine is sputtering. NIH funding remains chronically below what’s needed to sustain basic research. The FDA, paralyzed by internal restructuring, has delayed or denied promising rare disease treatments. And CMS, tasked with covering life-saving interventions, often treats medically necessary formula and therapies as “optional.”
This isn’t just bureaucracy—it’s betrayal.
For the 3,500 Americans with classic galactosemia, these cracks in the system amount to a lifetime cost of $7.23 million per patient in preventable burden. That’s not a projection. That’s real hospitalizations, therapy bills, denied claims, and lost income.
“We Have the Science—We’re Just Not Using It”
At the hearing, Senators praised the return on investment from biomedical research. Senator Tammy Baldwin cited cystic fibrosis advances that let patients become grandparents. But for many rare disease communities, that miracle moment is still out of reach—not because the science isn’t ready, but because the system isn’t.
Classic galactosemia has no FDA-approved treatment. Yet one drug, govorestat, showed promise—cutting toxic galactitol by 40%, improving tremors and speech. It was rejected by the FDA over data lost in a third-party error involving 11 children out of 50.
That is a textbook Type II error—where promising therapies get buried under statistical purism. And it costs lives.
$5 Million Could Save $425 Million. Why Wait?
At the hearing, NIH leaders warned that underfunding early research slows down everything from cancer breakthroughs to genetic therapies. That’s exactly why advocates are calling for a modest $5 million NIH investment in galactosemia. The return? Up to $425 million in savings through reduced disability, fewer medical crises, and more independent futures.
California’s Project Baby Bear already proved this. A $2.5M investment in genomic sequencing saved $12M and helped 178 infants. Galactosemia deserves the same urgency.
Medicaid Denials and the False Economy of Inaction
Meanwhile, CMS continues to deny coverage for medical formula unless a child is tube-fed—an archaic policy that endangers patients and drives up costs. Medicaid caps speech therapy visits for kids who can’t talk, and families are forced to fight insurance appeals just to afford basic care.
At the hearing, lawmakers rightly emphasized the economic value of biomedical advances. But let’s be clear: there is nothing economically sound about denying treatment until a child ends up in a PICU bed.
A Test Case for 30 Million Americans
Senators asked how we protect America’s lead in biomedical innovation. The answer isn’t just about labs and patents. It’s about whether we treat the rare disease community as worthy of the same effort given to cancer, diabetes, or COVID-19.
Galactosemia may be ultra-rare—but it is a test case for how we treat the other 30 million Americans with rare diseases.
Approving therapies like govorestat. Funding diagnostics. Covering medically necessary formula. These aren’t radical ideas. They’re cost-effective, life-saving, and scientifically supported steps Congress can act on now.
The Ask: Congress, Make This the Year You Back Rare Families
Here’s what Congress must do—this year:
Pass H.R. 6892 (Medical Nutrition Equity Act) to cover formula and end the tube-feeding loophole.
Revisit the FDA’s rejection of govorestat and apply existing flexibility for rare diseases.
Invest $5 million in galactosemia research to unlock hundreds of millions in long-term savings.
Add galactosemia to the SSA Blue Book, so families aren’t forced to fight for years to prove disability.
Sponsor the PROTECT Rare Act to guarantee off-label drug coverage when it’s guided by peer-reviewed evidence.
“We Don’t Have Time for the System to Catch Up”
One witness at the hearing spoke of how biomedical progress “gives time back to families.” For rare disease parents, time is everything. Time to hear your child speak. To avoid another ICU stay. To find hope in science before it’s too late.
Classic galactosemia isn’t waiting. And neither are we.
Let this hearing be more than talk. Let it be the beginning of action.
#GalactosemiaAction
