Access to care for rare diseases still varies by state: NORD report card
Ongoing gaps seen in medical nutrition and insurance coverage
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Access to care for people with rare diseases in the U.S. still varies significantly by state, with ongoing gaps in medical nutrition and insurance coverage that can directly affect whether patients receive timely and affordable treatment, according to the latest State Report Card from the National Organization for Rare Disorders (NORD).
Only four states — Colorado, Delaware, Maine, and New Jersey — earned top grades overall. Thirteen states failed on medical nutrition coverage, and 22 states lack protections against short-term health insurance plans that offer limited coverage and can exclude essential benefits and treatments.
“It remains unacceptable that access to [lifesaving] care still depends on where a family lives,” Pamela K. Gavin, NORD’s CEO, said in an organization press release. “No one is immune to acquiring a rare disease. It affects millions of families in every state. Policymakers must strengthen protections so patients can access treatment without facing devastating financial hardship.”
US earned a B grade overall
The State Report Card grades each state’s policies for rare diseases, such as aromatic L-amino acid decarboxylase (AADC) deficiency. NORD hopes it can serve as a tool for patients, advocates, and policymakers to identify gaps and push for changes that better meet the community’s needs.
Overall, the country earned a B grade. However, the national average masks wide differences among states, showing that access to care still depends heavily on where a patient lives. Only Colorado, Delaware, Maine, and New Jersey earned A grades, while most states fell in the middle range, with 25 earning B grades and 22 earning C grades.
NORD reviews nine key policy areas, including Medicaid eligibility, medical nutrition, newborn screening, insurance safeguards, advisory councils, step therapy (fail first), telehealth, and the out-of-pocket costs of prescription medications. Better access to telehealth and more affordable prescriptions helped raise the national average in 2025.
“We applaud the progress over the past year,” Gavin said.
In 2025, four states — Indiana, Iowa, Maryland, and Nevada — banned copay accumulators, which prevent patient assistance from counting toward insurance deductibles. Reforms targeting step therapy, which requires trying one or more cheaper treatments first, improved grades in Alaska (F to C) and New Jersey (F to A).
“Step therapy has been a big challenge for my family. Just this year, three of my immediate family members were taken off medications that were working very well and were required to try other, less expensive medications,” said Tammy from Arkansas, which earned an A grade in step therapy reforms. “Step therapy should not be used for patients who are already stable on a medication that is effective for them.”
43 states earning passing grades for telehealth policies
Added in 2022 to the list of policy areas featured in the State Report Card, telehealth refers to the use of telecommunication technologies to facilitate long-distance clinical care. Its use continues to expand, with 43 states earning passing grades for telehealth policies. Expanded use of telehealth can help patients connect with rare disease specialists who are often located far from their homes.
One of the most concerning areas is medical nutrition, which refers to formulas and foods prescribed to treat diseases with specific nutritional needs. Thirteen states received failing grades, and none earned an A. Insurance safeguards also remain uneven across the U.S., as 22 states failed to protect patients from so-called “junk” insurance plans. These plans can exclude essential benefits and treatments.
“I have often found that insurance does not cover the cheaper and less invasive treatment option. My physicians wanted me on three to four nutritional drinks a day, consumed orally, but insurance would not cover this cost,” said Jennifer, a patient from Kentucky who requires medical nutrition as part of her treatment.
Another area of concern is the lack of Rare Disease Advisory Councils in 19 states. These advisory councils give patients and caregivers a formal role in shaping healthcare policy. Since 2020, NORD has helped create 25 of the 33 councils nationwide. In 2025, Vermont, Oklahoma, Hawaii, and Pennsylvania introduced legislation to establish new advisory councils.
“Every Vermonter deserves to be seen, heard, and supported, especially those living with rare diseases who too often fall through the cracks of our healthcare system,” state Rep. Mary-Katherine Stone said. “The Rare Disease Advisory Council is how we start making that commitment real.”
