Finding Financial Assistance for Rare Diseases in the U.S.

Amy Niles, Chief Mission Officer for the PAN Foundation, shares options for those looking for financial assistance for those living with a rare disease in the United States. 

There are more than 10,000 distinct types of rare and genetic diseases, impacting one in ten people, according to previous Global Genes research.  As part of the Global Genes community, you are intimately aware of the challenges that come with living with a rare disease—and the costs.

It takes an average of six or more years for rare disease patients to get an accurate diagnosis, and even then, treatments aren’t available for many conditions. That means that millions of people each year are working with their healthcare teams to best treat their symptoms and facing high out-of-pocket costs to do so.

One study conducted by the National Institutes of Health (NIH) found that healthcare costs for people with rare diseases were three to five times greater than costs for people without a rare disease.

As the Chief Mission Officer at the PAN Foundation, I’m pleased to share an overview of our financial assistance resources, including disease-specific funding for more than 25 rare diseases. Over the last 20 years, we have provided more than $4 billion in financial assistance to underinsured people with life-threatening, chronic, and rare diseases across every U.S. state and territory.

It takes a village to manage a rare diagnosis and PAN would love to be a part of your support team.

About PAN’s financial assistance

The PAN Foundation provides financial assistance to help pay for medication copay costs, health insurance premiums, and transportation for a 12-month period.

We have more than 70 disease-specific financial assistance funds, with over 25 rare diseases—from graft vs. host disease to myasthenia gravis and many more. On our website, you can search by treatment or disease, check your eligibility, and apply for a disease-specific grant in just a few minutes. Patients can apply directly or their loved ones, healthcare provider, or pharmacy can apply on their behalf.

Grant amounts and eligibility requirements vary by fund, but generally speaking we help people who:

• live in the United States or U.S. territories (citizenship not required),
• have insurance coverage,
• are prescribed a covered medication for the given disease fund,
• and have an income of less than 500 percent of the federal poverty level.

In 2024, that means a one-person household earning less than $75,300 per year may be eligible.

If a fund is closed, patients—or their loved ones or healthcare providers—can sign up for PAN’s wait list. When more funding is available, everyone on the wait list who meets the eligibility criteria will get an alert that they can apply before the fund opens to the general public. Those patients who apply during the wait list period are then provided grants in the order they joined the wait list (not the order they submitted their application).

Richard’s story

Richard Haller, from Minnesota, lives with amyloidosis. He has a fixed income, and the cost of his treatment was exorbitant, even with health insurance.

He said he spent hours and hours looking for financial assistance before he found PAN, and his grant helped him afford his treatment. His medication helps slow the progress of his amyloidosis, which has allowed him to focus on spending time with his children and grandchildren, and fishing with his brother.

“I am on seven other meds. Some are high cost, and sometimes you have to skip meals or skip doses of other meds to make ends meet. Thanks to the PAN Foundation, I can stay on my medication and enjoy the extended time I have had,” he said. “It keeps me going, and gives me a reason to keep fighting, because I feel like some IS trying to help.”

PAN offers financial assistance for rare diseases, including:
Acromegaly
Acute myeloid leukemia
Amyloidosis
Biliary tract cancer
Cushing’s disease or syndrome
Duchenne muscular dystrophy
Fabry disease*
Gaucher disease
Graft versus host disease
Hemolytic uremic syndrome*
Hemophilia*
Inherited retinal disease*
Liver cancer
Long-chain fatty acid oxidation
Lysosomal acid lipase deficiency*
Myasthenia gravis
Neurofibromatosis
Neuromyelitis optica spectrum disorder
Neurotrophic keratitis
Paroxysmal nocturnal hemoglobinuria
Pemphigus Vulgaris
Pompe disease*
Rett syndrome
Sickle cell disease
Short bowel syndrome*
Small cell lung cancer
Spinal muscular atrophy
Uveitis
Von Willebrand disease

*PAN offers copay assistance and health insurance premium assistance for these diagnoses

Which medications are covered

With copay grants, patients choose which pharmacies and providers they use, as well as which covered medications in their treatment plan they want to use.

The PAN Foundation creates a standardized list of medications for each disease fund, known as a formulary, that is compliant with all applicable federal laws and regulations. In general, we cover all prescription medications, including generic or bioequivalent drugs, that are FDA-approved, listed in official compendia, or are published in evidence-based or clinical guidelines.

If the FDA has approved only a single drug for a given disease fund, or only drugs marketed by a single manufacturer or its affiliates have been approved, PAN will provide support for other medical needs of patients with the disease. In these cases, the PAN Foundation’s formulary will cover all drugs used to treat the symptoms of the disease, including treatment of side effects.

How to apply

You can apply for a PAN grant if you are:

• Currently in treatment or taking medication for your diagnosis
• Got treatment in the last 90 days
• Scheduled to begin treatment in the next 120 days

If you have previously applied for a PAN grant or joined one of our disease fund wait lists, you can apply online through the online PAN portal or by phone. If not, please apply by phone.

To apply by phone, call us at 1-866-316-7263 Monday through Friday, 9 a.m. to 5:30 p.m. ET. We have translation services available in 100 languages to support callers, as well as Spanish language resources on our website.

Healthcare providers and pharmacies can apply online through the PAN portal or by phone on behalf of their patients.

More ways to find financial assistance

The PAN Foundation developed and maintains FundFinder, a free online tool that lets you track more than 200 financial assistance programs across nine charitable organizations, including PAN. Anyone can sign up for real-time email or text alerts when funding is available. To date, we’ve sent more than 2 million notifications to alert people about available assistance.

FundFinder also lists nearly 150 advocacy and patient support organizations, connecting patients with additional resources like support groups, clinical trial information, educational resources, and more.

Learn more about the PAN Foundation or apply for financial assistance

About the author
Amy Niles is the Chief Mission Officer for the Patient Access Network (PAN) Foundation, an independent, national 501(c)(3) organization dedicated to helping underinsured people with life-threatening, chronic, and rare diseases with the out-of-pocket costs for their prescribed medications. She leads the organization’s education initiatives, program development, clinical affairs, and advocacy strategy, while overseeing public policy and advocacy initiatives, external relations, and marketing and communications.