Although patient need is extensive, no single clearinghouse for information on financial assistance for rare disease patients currently exists. Patients must make do with information gleaned from online searches, providers, pharmacies and pharmaceutical companies.
Patient Need for Financial Assistance
Patients’ need for financial assistance with rare disease drug costs stems from three primary sources. First, the cost of rare disease treatment is higher than for more common conditions. Second, rare disease patients are more likely to belong to cohorts with limited access to financial resources, such as impoverished communities. Third, even patients who have insurance face barriers to receiving insurance coverage for their rare disease medications.
High Costs and Vulnerable Cohorts
Rare disease treatment imposes “globally ubiquitous high costs,” write Claudia C.Y. Chung and fellow researchers in The Lancet.
Rare disease patients may already be members of groups that need higher than usual rates of financial assistance. The researchers’ study of rare disease treatment costs in Hong Kong found that poverty rates among rare disease patients were “significantly higher than global estimates.” Pediatric patients also tended to report higher costs than adult patients; being children, these patients are typically dependent upon the adults in their lives to provide the financial support to meet their basic needs.
Rare diseases also impose higher costs than more common conditions. In a 2021 study, Ainslie Tisdale, et al. attempted to quantify the per-patient direct medical costs of rare diseases versus more common conditions. By matching rare disease patients with control patients, they estimated that rare disease patients’ direct medical costs are three to five times higher than similarly-aged patients without rare diseases. Rare disease patients also faced longer journeys to diagnosis than other patients, which raised costs and often complicated their medical conditions, requiring more intensive and costly treatments.
The most common sources of direct medical costs for rare diseases are inpatient hospitalization and prescription drug costs, write Grace Yang and fellow researchers in an article in the Orphanet Journal of Rare Diseases. Missed work and lost productivity at work add indirect costs that affect society more generally.
Many rare disease patients have fewer resources than similarly-situated patients with more common conditions — yet rare disease patients must face higher costs. Even where private insurance coverage exists, applying it to rare disease treatment costs can prove daunting.
Insurance Coverage Challenges and Carve-Outs
For rare disease patients, having private health insurance coverage does not guarantee coverage of their treatment needs. It only opens the door to procedural and paperwork demands. The risk of denial is often high.
In a series of interviews with the parents of children with metachromatic leukodystrophy (MLD) and spinal muscular atrophy (SMA), researchers Tai L.S. Pasquini, Sarah L. Goff and Jennifer M. Whitehill found that parents with private health insurance plans that covered their children faced three major hurdles:
- Preauthorizations and other insurance demands made it difficult for parents to access necessary health services for their children.
- Parents found themselves dealing with health insurance representatives frequently or repeatedly.
- Their children often did not qualify for secondary insurance coverage under their state’s rules, even if their primary insurance would not cover part or all of the cost of treatment.
Pasquini, Goff and Whitehill recommended several steps that could help parents, such as time limits on claims processing, more transparent insurer processes, and continuing authorizations for ongoing needs such as monthly infusions.
Even rare disease patients who have private insurance coverage generally may not have private insurance coverage for their specific condition or prescribed treatments. Insurance carve-outs exempt certain medications from insurance coverage. The patient may have an insurance plan — but not one that covers the treatment they actually need.
Carve-out plans typically come with some form of support intended to help patients connect to financial assistance programs or secondary insurance, writes Mark Slomiany, director of advisory at The Marwood Group. Accessing this assistance can prove challenging: It can also lengthen delays in treatment, further complicating patients’ health status and driving up treatment costs.
Available Financial Assistance Resources
Rare diseases are “rare” in the sense that any one rare disease affects a relatively low number of patients. Still, as a whole, rare diseases affect approximately 25 to 30 million people in the United States and over 300 million people worldwide, write Tisdale, et al.
Public Programs and Resources
Public insurance programs like Medicaid and the Children’s Health Insurance Program (CHIP) provide primary coverage for many impoverished Americans, as well as secondary coverage in some instances. Many pharmaceutical assistance programs work to enroll patients in these programs if they qualify.
Other state and federal offices in the U.S. provide information connecting patients to financial assistance resources but do not provide such resources directly. These include resource pages hosted by the National Center for Advancing Translational Sciences (NCATS) and NCATS’s Genetic and Rare Diseases Information Center. On a page titled “Financial Aid for Medical Treatment,” however, the NIH specifies that the organization “is not authorized to provide routine medical assistance or treatment funds.” The NIH can share information, but it does not provide a direct source of financial assistance to rare disease patients.
Pharmaceutical Company and Charitable Patient Assistance Programs
“Industry-sponsored patient support programs have become an essential ‘lubricant’ for coordinating care of patients with complex medical needs,” writes Nicholas Basta, editor emeritus at Pharmaceutical Commerce. Yet patients often do not access these programs — if they even know about them. Basta notes that only about 18 percent of patients are aware that pharmaceutical companies offer financial assistance programs.
Lack of communication drives this lack of patient awareness. Information on industry-sponsored patient assistance programs can be difficult to find online. Healthcare providers and pharmacists may not have access to information, which prevents them from providing it to their patients.
Several charities have also made it their mission to provide financial assistance to rare disease patients. The National Organization for Rare Disorders (NORD) has a list of such patient assistance programs.
Like rare disease patient organizations, charitable organizations can fill necessary gaps for certain patients. But because charities are often limited in their scope and funding, the assistance they provide is piecemeal at best and nonexistent for some types of rare diseases or treatment needs.
Rare Disease Patient Organizations
Rare disease patient organizations (RDPOs) play a key role in connecting patients to financial assistance. One strength of RDPOs is their ability to collect and share information on financial assistance that targets a specific condition or type of treatment. Patients who rely on RDPO resources may spend less time searching for assistance programs that include their needs and more time applying to that focus on their disease or treatment specifically.
Yet RDPOs face several challenges when it comes to assisting patients, write Christina Q. Nguyen and fellow researchers in a 2022 article. Among these are financial limitations, including limits on the financial assistance RDPOs can provide to patients who need help paying for medications. While RDPOs play an important role in the financial assistance landscape, they cannot carry the entire burden.
How to Connect Patients to Available Resources
Pressure on financial assistance programs is unlikely to abate in the near future. With limited power to control spending on rare disease treatments, payers are likely to continue relying on methods like carve-outs, despite the fact that these methods remain unpopular among providers, pharmacists, pharma manufacturers and patients.
One way to improve patient access to needed medications is to make it easier to connect patients to relevant sources of financial assistance.
Using the right digital tools can benefit rare disease research and treatment in a number of ways. Using improved electronic health records (EHR) can facilitate research, write Sheldon Garrison and fellow researchers. Similarly, using a single platform that manages a patient’s journey from receiving a written prescription through obtaining financial support and filling that prescription can streamline the patient journey.
“There needs to be greater public awareness of the large and growing medical footprint of rare diseases in society,” says Anne Pariser, director of the NCATS Office of Rare Diseases Research. Greater public awareness can help to drive reforms that address costs and improve access to treatment for patients with rare diseases. Digital tools that streamline the assistance process can also help patients avoid unnecessary delays in treatment.
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