How to Get Help with Medical Bills for Rare Disease

Hundreds of new treatments for rare conditions are in various stages of development all over the world. What if the most promising new treatment for your condition is not available in the United States?

Rare patients can run into trouble accessing treatments for any of a number of reasons. Perhaps the medication your doctor would like to try is not yet FDA-approved for your condition. There might be a clinical trial you want to take part in, but you do not meet the eligibility requirements for that trial. Or maybe your health insurer is reluctant to pay for a newly available medication. Whatever the reason, there are programs that may offer hope.

Expanding patient access to treatment around the world through Managed Access

“Managed access” is a broad term for programs that aim to expand access to medications that are not available to patients through the usual channels. These programs offer ways through which health authorities can grant physicians and patients access to investigational drugs—medications that are being studied, but are not yet approved by the FDA. These programs go by various names, including compassionate use, expanded access, named-patient, and early access programs. Patient assistance programs are also available to those who need help affording treatment.

One organization working to make a difference in this area is RareiTi, founded by two women with a personal stake in the rare disease world. RareiTi is a unique company dedicated to advancing the development of rare disease treatments and expanding patient access to them. RareiTi works with pharmaceutical companies, clinical research organizations, regulatory agencies, healthcare providers, patient advocacy groups, and rare disease communities worldwide to develop and coordinate customized programs that meet specific patient needs.

RareiTi co-founders Wendy White and Natalie Douglas were already successful healthcare industry entrepreneurs when they recognized an important unmet need. Patient access to new and investigational treatments was not keeping pace with fast-moving advances in rare disease research and development. Both women have strong personal ties to the rare community; White’s daughter has a connective tissue disorder, and Douglas lost a sister to a rare cancer. With their perspectives, expertise, and contacts in the field, they knew they could make a unique contribution. “With RareiTi, we plan to be an ally in rare disease to change the care paradigm to better support families, patients, clinicians, and the pharmaceutical industry,” says White. “We believe we are in a great position to put together a solid global strategy for corporations. We can also help patients where they are with what they need by collecting patient experience data and insights at the point of care through a co-created app that will support faster approvals, faster global uptake, and better patient outcomes.”

Getting help paying for medications through a manufacturer’s patient assistance programs

In the United States, patients may face another kind of access challenge if they cannot afford a co-pay for an expensive therapy. This is where patient assistance programs can help. Though such programs may not be widely publicized, virtually all pharmaceutical companies offer financial aid programs for patients who have trouble affording medications. Eligibility requirements vary from program to program, but all require patients to demonstrate financial need.

The Pharmaceutical Research and Manufacturers of America (PhARMA), an industry association, offers various resources to help patients access medications they need, including financial assistance programs. Their Medicine Assistance Tool (MAT) is an online database that matches patients with resources and cost-sharing programs that can help lower out-of-pocket medication costs, whether or not patients are insured.

Listed below are some of the many resources currently offered by patient advocacy groups, pharmaceutical companies, and industry associations. Dozens more are just an online search away.