PAP 2025: The Impact of Out-of-Pocket Costs on Patients with Rare Diseases

Charitable donations have long been a vital lifeline for patients struggling to afford life-saving medications, particularly those with chronic and rare diseases. Over the past few decades, various assistance programs—including nonprofit safety nets, third-party organizations, and manufacturer-sponsored free drug initiatives—have emerged to bridge the gap between medical advancements and patient affordability. However, despite these efforts, high out-of-pocket costs still prevent many individuals from accessing essential treatments. In an interview with Pharmaceutical Commerce, Jonathan James, CEO of Hope Charities, shares insights on reducing barriers to care and improving patient access.

PC: Can you provide insights into the real-world out-of-pocket costs patients face, particularly in high-cost therapeutic areas?

James: ’ll share a personal story. I was born with hemophilia, and managing hemophilia is costly, especially when considering life expectancy. As long as I can maintain access to my medications, my life expectancy is relatively in line with the general population. The big problem is that if I lose access to this medication at any point, it becomes a massive hurdle.

Many people with bleeding disorders today spend around $1 million annually. That cost has increased over time, but not exponentially—rather, it has risen incrementally year over year. There’s a strong focus on rare disease groups like hemophilia because they require high-cost medications. One of the biggest challenges we face is that keeping supplemental insurance is often more difficult than even developing the medication needed to treat the condition itself.

Thanks to medical advancements, people with hemophilia are now living longer and healthier lives. In the past, many were unable to work and were completely dependent on disability benefits, often confined to wheelchairs. Today, we have a thriving community of individuals who can live fully productive lives, attend college, and enter highly skilled professions. However, if they lose access to their medication, everything changes.

The challenge is that these individuals are now at risk of becoming vulnerable again—potentially returning to wheelchairs and being unable to work. This raises an important question: Is it better for someone to be a productive, taxpaying citizen who supports their family and contributes to the economy, or should they be forced into a situation where they are entirely dependent on social services?

We believe productivity is the better path, and out of that belief comes our drive, passion, and determination to do everything we can. This includes helping cover out-of-pocket expenses to ensure continued access to care.

Full Interview Summary: Charitable donations have played a crucial role in helping patients access life-saving medications, particularly those with chronic and rare diseases who face significant financial barriers. Over the past 25-30 years, various assistance programs—including nonprofit safety nets, third-party organizations, and manufacturer-sponsored free drug programs—have emerged to support patients struggling with the rising costs of healthcare. However, despite these efforts, many individuals still face challenges in affording their treatments, with high out-of-pocket costs often preventing them from receiving necessary care.

For conditions like hemophilia, where annual treatment costs can exceed $1 million, losing access to medication can mean a drastic decline in quality of life, forcing patients to rely on disability or face severe health complications. While charitable programs provide critical relief, they also struggle with sustainability due to funding shortages and limited public awareness. Many organizations must balance the need to educate patients about available assistance while ensuring they have enough resources to support those in need.

Healthcare policies such as the Inflation Reduction Act and Medicaid redeterminations have also influenced access to care, sometimes alleviating costs for certain populations while creating new challenges for others. While high-cost medications often receive the most scrutiny, hospitalization and inpatient care account for a much larger portion of healthcare spending. Expanding financial assistance for prescription medications could help reduce hospital admissions and lower overall healthcare costs.

Looking forward, there is a need for more sustainable models of charitable giving and financial aid to ensure that patients can consistently access the treatments they need. Policy reforms, expanded subsidies, and a greater focus on preventative care may help create a more equitable healthcare system while reducing the financial strain on both patients and assistance programs.