For patients facing life-threatening illnesses who have exhausted all approved treatment options, expanded access programs offer a glimmer of hope. These programs, also known as compassionate use, provide a pathway for patients to receive experimental treatments that haven’t yet completed the FDA approval process.
Sarah Chen never expected to find herself researching experimental treatments. At 42, the mother of two was diagnosed with an aggressive form of breast cancer that proved resistant to standard therapies. “After trying three different treatment protocols without success, my oncologist told me we were running out of options,” Chen recalls. “That’s when she mentioned expanded access as a possibility.”
Expanded access programs have existed for decades, gaining prominence during the HIV/AIDS crisis of the 1980s when desperate patients demanded access to experimental medications. Today, these programs serve as a crucial bridge between clinical trials and FDA approval, allowing seriously ill patients to access promising treatments still under investigation.
Dr. Marcus Rodriguez, an oncologist at Memorial Sloan Kettering Cancer Center, explains the process: “When we have patients who’ve exhausted standard treatments, we look at experimental therapies that might help. If there’s a promising drug in development that matches their condition, we can request access from the pharmaceutical company through the FDA’s expanded access program.”
However, navigating the system can be challenging. Pharmaceutical companies aren’t required to provide their experimental treatments through expanded access programs. Even when they do, the process involves complex paperwork, coordination between multiple parties, and no guarantee of success. However, Early Access Care offers customized Expanded Access solutions for some of those that may not qualify.
The FDA reports approving 99% of expanded access requests it receives, but this statistic doesn’t tell the whole story. Many requests never reach the FDA because pharmaceutical companies decline to provide the treatment. Companies often cite limited drug supply, concerns about interfering with ongoing clinical trials, or liability risks.
“The hardest part for patients is understanding that just because a treatment exists in development, doesn’t mean they’ll be able to access it,” says Elena Martinez, a patient advocate who helps seriously ill patients navigate expanded access requests. “Companies have legitimate concerns about their limited supplies and the potential impact on their clinical trials, which are necessary to get these treatments approved for all patients.”
Recent technological advances have made it easier for patients to learn about experimental treatments. Online platforms now aggregate information about clinical trials and expanded access programs, while social media enables patients to connect with others facing similar challenges. Patient advocacy groups have also become more sophisticated in helping people navigate the system.
The Right to Try Act, passed in 2018, created an alternative pathway for patients to access experimental treatments without FDA involvement. However, this hasn’t eliminated the need for traditional expanded access programs. “Right to Try hasn’t been widely used because it doesn’t address the main obstacle – getting companies to provide the treatment,” explains Dr. Rodriguez. “Most companies still prefer working through the FDA’s established expanded access process.”
For some patients, expanded access programs have proved life-changing. James Wilson, diagnosed with a rare form of lymphoma, received an experimental immunotherapy through expanded access after standard treatments failed. “That treatment gave me my life back,” Wilson says. “I’m now three years cancer-free, and the drug that helped me was approved by the FDA last year.”
However, not all stories have happy endings. The experimental nature of these treatments means there’s no guarantee of success, and some patients experience serious side effects or no benefit at all. “It’s crucial that patients understand these are investigational treatments,” emphasizes Dr. Rodriguez. “We have limited data on their effectiveness and safety.”
The future of expanded access programs may be shaped by lessons learned during the COVID-19 pandemic, which highlighted both the potential and challenges of providing experimental treatments during a health crisis. The pandemic led to increased public awareness of expanded access programs and sparked discussions about improving the system.
Patient advocacy groups are pushing for reforms to make the process more transparent and equitable. “We need better systems to ensure all eligible patients have fair access to these programs, regardless of their location or resources,” says Martinez. “Currently, patients at major medical centers often have better access than those in rural areas or underserved communities.”
For Sarah Chen, expanded access provided an opportunity she might not have had otherwise. Through an expanded access program, she received an experimental targeted therapy that has kept her cancer stable for the past eighteen months. “I know this treatment might not work forever,” she says, “but it’s given me precious time with my family that I wouldn’t have had otherwise.”
As medical research continues to advance, expanded access programs remain a critical lifeline for seriously ill patients who have exhausted standard treatments. While the system isn’t perfect, it offers hope to those who might otherwise have none, while contributing to the broader development of new treatments that may eventually help many more patients.c
