When facing a life-threatening condition with no approved treatment options, patients and their families often find themselves navigating complex regulatory pathways in search of experimental therapies. Two distinct routes exist for accessing investigational treatments outside of clinical trials: the FDA’s long-established Expanded Access Program, also known as compassionate use, and the newer Right to Try pathway created by federal legislation in 2018. Understanding the key differences, advantages, and limitations of these options is crucial for patients confronting difficult treatment decisions.
The FDA’s Expanded Access Program has evolved significantly since its formal establishment in the 1980s during the HIV/AIDS crisis. This pathway requires a multi-step process: the treating physician must contact the drug manufacturer to request the investigational treatment; if the company agrees, the physician then submits an application to the FDA; and finally, an Institutional Review Board (IRB) must approve the treatment plan. While this process may seem bureaucratically cumbersome, the FDA approves approximately 99% of expanded access requests it receives, often within days or even hours for emergency situations.
The Right to Try Act, signed into law in May 2018, emerged from a movement arguing that terminally ill patients should have direct access to experimental treatments without government oversight. Under this pathway, eligible patients can work with their physicians to request investigational drugs directly from pharmaceutical companies, bypassing FDA review and significantly reducing paperwork requirements. However, similar to expanded access, drug manufacturers remain under no obligation to provide their investigational products.
Eligibility criteria represent one significant difference between these pathways. Right to Try specifically limits access to patients with life-threatening diseases who have exhausted approved treatment options, cannot participate in clinical trials, and have received certification from a qualified physician. The FDA’s Expanded Access Program applies these same general principles but maintains flexibility to consider patients with serious (not necessarily immediately life-threatening) conditions when appropriate.
Treatment accessibility also differs between the programs. Expanded Access allows access to a broader range of investigational products, including those in early-stage clinical testing. Right to Try limits eligibility to drugs that have completed Phase 1 safety trials and remain in active development. This restriction aims to ensure patients receive treatments with at least preliminary safety data, but it also narrows the available options compared to the FDA’s program.
The risk-benefit calculation represents another important distinction. Under Expanded Access, the FDA evaluates whether the potential benefits justify the potential risks for the specific patient, considering their unique circumstances and condition severity. This review provides an additional layer of protection but requires time and resources. Right to Try eliminates this independent review, placing the risk-benefit assessment entirely in the hands of patients, physicians, and drug manufacturers.
Data collection requirements also diverge significantly. The FDA’s program includes structured adverse event reporting and sometimes data collection protocols that contribute to the broader understanding of the treatment’s effects. Right to Try has minimal reporting requirements, requiring only annual summaries from manufacturers rather than detailed safety reports. While this reduces administrative burdens, it potentially limits opportunities to gather valuable information that could inform future treatment approaches.
Financial considerations remain complex under both pathways. Neither Right to Try nor Expanded Access requires insurance coverage for experimental treatments or associated costs like administration and monitoring. Both allow manufacturers to charge patients for direct costs associated with providing the drug, though Right to Try explicitly prohibits charging more than production costs. These financial barriers often create significant obstacles regardless of which pathway patients pursue.
Liability protections differ subtly between the programs. Right to Try includes explicit language protecting physicians and manufacturers from liability related to adverse outcomes, unless those outcomes result from reckless conduct or intentional wrongdoing. The Expanded Access Program offers similar but less comprehensive protections, potentially making manufacturers more hesitant to participate.
Despite the legislative fanfare surrounding Right to Try, utilization data suggests it hasn’t dramatically increased access to experimental treatments as many proponents anticipated. A Government Accountability Office report found that in the first year after passage, fewer than 10 patients accessed drugs through Right to Try. In contrast, the FDA processes over 1,000 expanded access requests annually, the vast majority of which receive approval. This disparity suggests that manufacturer willingness to provide experimental treatments—not regulatory hurdles—remains the primary limiting factor for patient access.
Patient advocacy groups hold mixed opinions on these pathways. Some organizations strongly supported Right to Try, viewing it as an important assertion of patient autonomy against regulatory paternalism. Others express concern that circumventing FDA oversight might expose vulnerable patients to unnecessary risks without providing meaningful benefits. Most groups now acknowledge that both pathways have limitations and work to help patients navigate whichever option best suits their specific circumstances.
Medical ethicists continue debating the implications of these dual systems. Some argue that Right to Try appropriately prioritizes individual autonomy, allowing patients facing terminal illnesses to make their own risk calculations without government interference. Others counter that the FDA’s review process provides essential protection for desperate patients who might otherwise pursue treatments with unfavorable risk-benefit profiles. This tension between autonomy and protection reflects broader societal debates about the proper role of government in healthcare decisions.
For patients evaluating these options, several practical considerations can guide decision-making. First, understanding that manufacturer willingness represents the most significant barrier under either pathway can help set realistic expectations. Second, patients with serious but non-immediately life-threatening conditions may find the Expanded Access Program more accommodating. Third, those seeking treatments in very early development stages will generally need to pursue the FDA pathway, as Right to Try only applies to drugs that have completed Phase 1 trials.
Working with experienced healthcare providers familiar with both pathways provides invaluable assistance. Some major academic medical centers and comprehensive cancer centers have established expanded access committees or navigators who can guide patients through application processes. Patient advocacy organizations specific to particular diseases often maintain resources explaining both options in disease-specific contexts.
As medicine continues advancing rapidly, with breakthrough treatments emerging for previously untreatable conditions, the tension between rigorous evaluation and urgent patient access will likely intensify. Both Right to Try and Expanded Access represent imperfect but important mechanisms for balancing these competing priorities. Rather than viewing these pathways as competing alternatives, patients benefit most when they understand the nuanced differences and choose the approach most appropriate for their unique circumstances.
For those facing terminal illnesses without standard treatment options, knowledge truly represents power. Understanding both pathways—their requirements, limitations, and practical implications—enables patients to make informed decisions during extraordinarily difficult circumstances. While neither program guarantees access to experimental treatments, both reflect society’s recognition that patients with life-threatening conditions deserve pathways to potentially beneficial interventions, even when those interventions haven’t completed the traditional approval process.
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