When faced with a dire situation with no approved treatment options, patients and their families often find themselves navigating a complicated regulatory road looking for experimental treatments. There are two different ways to access investigational treatments outside of clinical trials: the FDA’s Long-reblished Expanded Access Program, also known as compassionate use, and the Newer Right to Try pathway created by federal law in 2018. Understanding the main differences, benefits, and limitations of these options is essential for patients facing difficult treatment decisions.
The FDA’s expanded access program has evolved significantly since its establishment in the 1980s during the HIV/AIDS crisis. This method requires a multi-step process: the treating physician must contact the pharmaceutical manufacturer to request a trial treatment; if the company agrees, the doctor submits an application to the FDA; and finally, an Institutional Review Board (IRB) must approve the treatment plan. Although this process may seem like an industry-intensive process, the FDA approves approximately 99% of the expansion access requests it receives, often within days or even hours for emergency situations.
The Right to Try Act, signed into law in May 2018, is the result of a movement that argues that sick patients should have access to experimental treatments without government oversight. Under this approach, eligible patients can work with their physicians to request investigational drugs directly from pharmaceutical companies, bypassing FDA inspections and greatly reducing documentation requirements. However, as the opportunity expands, pharmaceutical manufacturers are under no obligation to provide their research products.
The qualification process represents one major difference between these methods. The right to try specifically limits the access to patients with life-threatening diseases who have exhausted the recommended treatment options, cannot participate in clinical trials and receive a certificate from a qualified physician. The FDA’s Expanded Access Program applies these same general principles but retains flexibility to consider patients with serious (not immediately life-threatening) conditions when appropriate.
Access to treatment also varies between programs. Expanded access allows access to more research products, including those in early clinical trials. Trial authorization limits eligibility to drugs that have completed Phase 1 safety trials and remain in development. This restriction is intended to ensure that patients receive treatment with at least preliminary safety data, but it reduces the available options compared to the FDA program.
The risk-benefit ratio represents another important difference. Under Expanded Access, the FDA evaluates whether the potential benefits justify the patient’s specific risks, considering their unique circumstances and the severity of their conditions. This test provides additional protection but requires time and resources. The right to try eliminates independent testing, placing the cost-benefit assessment entirely in the hands of patients, physicians, and drug manufacturers.
Data collection requirements also vary widely. FDA programs include standardized program reporting and sometimes data collection procedures that contribute to a broader understanding of the treatment’s effects. Right to Try has minimal reporting requirements, requiring only a summary of the year from the manufacturer rather than a detailed safety report. Although this reduces the administrative burden, it may limit the opportunity to gather valuable information that may inform future treatment options.
Financial analysis is complicated under both approaches. No right to try or expand access requires insurance coverage for experimental treatments or additional costs such as administration and monitoring. Both allow manufacturers to reimburse patients for any costs associated with providing the drug, although trial rights are restricted from paying more than manufacturing costs. These financial barriers often pose significant barriers regardless of the path patients take.
Load protection differs subtly between programs. The right to try includes clear language protecting physicians and manufacturers from liability related to adverse outcomes, unless those outcomes are the result of negligence or malpractice. The Expanded Access Program offers similar protections but on a smaller scale, which may discourage manufacturers from participating.
Despite the legislative interest in testing rights, usage data show that there has not been as much access to experimental treatments as many advocates had hoped. A government accountability report found that in the first year after its launch, fewer than 10 patients received the drug through Right to Try. In contrast, the FDA makes more than 1,000 expanded access requests each year, the vast majority of which are approved. This difference suggests that manufacturers’ willingness to offer experimental treatments—not procedural barriers—is still the primary limiting factor for patient access.
Patient advocacy groups have different opinions on these approaches. Some organizations support the Right to Try, viewing it as an important assertion of patient autonomy against patriarchal control. Others express concern that going through FDA oversight could expose vulnerable patients to unnecessary risks without providing meaningful benefits. Most organizations now agree that both approaches have limitations and work to help patients navigate whichever option best suits their specific situation.
Medical experts continue to debate the merits of these two systems. Some argue that the right to appropriate testing leads to individual liberty, allowing patients facing terminal illness to calculate their own risk without government interference. Others argue that the FDA’s review process provides important protections for frustrated patients who may pursue treatments with poor benefit profiles. This conflict between freedom and protection reflects the public debate about the appropriate role of government in health care decisions.
For patients considering these options, several practical considerations can guide the decision. First, understanding that developer readiness represents the biggest hurdle under any approach can help set the right expectations. Second, patients with serious but not immediately life-threatening conditions may benefit from the Expanded Access Program. Third, those seeking treatments in the early stages of development will often need to pursue the FDA’s path, as Right to Trick only applies to drugs that have completed Phase 1 testing.
Working with health professionals who are familiar with both approaches provides valuable support. Some large academic medical institutions and cancer centers have established expanded access committees or pilots who can guide patients through the application process. Interdisciplinary teams often use resources to explain both options in specific disease situations.
As medicine continues to advance more aggressively, with more aggressive treatments for previously untreated conditions, the tension between strict monitoring and rapid patient access will only get worse. Both the ability to experiment and the opportunity to expand represent an imperfect but necessary mechanism for balancing these competing interests. Rather than viewing these methods as competing alternatives, patients benefit most when they understand the differences and choose the most appropriate method for their unique situation.
For those facing terminal illnesses with no standard treatment options, knowledge represents real power. Understanding the two approaches—their needs, limitations, and implications—helps patients make the right decisions during particularly difficult situations. Although neither program guarantees access to experimental treatments, both reflect public acceptance that patients with life-threatening conditions deserve a pathway to potentially beneficial assistance, even when the programs have not completed the traditional admissions process.
