Evolution of the ODA
Since 1983, “a number of amendments ... made the original [ODA] stronger or refined it in various ways,” said Mr. Saltonstall. NORD continues to “very carefully watch any attempts to change it, alter it, move it, take pieces away from it, or – some people say – to make it ‘better.’ To us, it’s a very important law.”
Orphan Drug Credit (ODC). Ms. Ross elaborated: “In recent years, Congress has considered significant changes to the Orphan Drug Credit, which is one of several critical incentives established by the ODA.” When established in 1983, the ODC provided sponsors of a drug with a 50% tax credit for qualified clinical testing expenses associated with developing an orphan drug.
“Unfortunately, despite fierce opposition from NORD and our army of membership organizations and grassroots advocates,” Ms. Ross said, “the Tax Cuts and Jobs Act of 2017 reduced the ODC from 50% to 25%. Given the significant amount of time it takes to conduct research and development into rare diseases, we still don’t have a good sense as to the impact of the ODC being reduced to 25%.”
However, efforts to further limit the availability of the ODC during debate around the Build Back Better framework in 2021 were ultimately defeated. That defeat was, in part, a result of NORD’s rare disease community’s advocacy,16 when no changes to the ODC were included in the Inflation Reduction Act.
Patient Assistance Program. In 1987, NORD’s multifaceted patient assistance program (serving today under the umbrella name RareCare17) was founded to help patients obtain lifesaving or life-sustaining medication that they could not otherwise afford, Ms. Ross said. This was based on an unmet need for patients living and struggling with a rare disease who didn’t have access to the care and treatment they needed. “There was no other foundation equipped to do this at the time. NORD came forward to develop the first program of its kind.”
The program provides medication, financial assistance with insurance premiums and copays, diagnostic testing assistance, and travel assistance for clinical trials or consultation with disease specialists. According to Ms. Ross, “today, NORD’s patient assistance program provides, on average, $30 million to $40 million in patient assistance annually to eligible patients and families.”
Pharmaceutical companies do contribute to the patient assistance program. “However, they’re in the dark as to how the money is allocated,” said Mr. Saltonstall. “For example, a given company might donate money for patients with a particular disease but won’t know if patients who have that disease will necessarily receive funds to use toward that company’s drug. The patient might receive assistance to take a drug made by a different company – a competitor.”
There are approximately 14 foundations around the country that have worked within the constraints of the Department of Health & Human Services Office of Inspector General, which established rules about how foundations must operate. Under the broader umbrella of NORD, these foundations also help pay coinsurance costs or provide drugs at no cost to patients who meet financial criteria.