In an age of accelerating medical innovation, the U.S. Food and Drug Administration (FDA) is facing renewed scrutiny for policies that actively hinder patient access to proven treatments. A growing chorus of patients, doctors, and policy advocates argue that the agency’s outdated bureaucratic processes are not just slow but also act as a direct cause of preventable harm and suffering, particularly for those with serious conditions.
A Health Care Crisis for Patients with Digestive Tract Paralysis
For patients with digestive tract paralysis, a condition affecting nearly 5 million Americans, the struggle for treatment is a decades-long battle. As a coalition of doctors and patients wrote in a recent letter, these conditions, including gastroparesis, are one of the most serious unaddressed conditions of the digestive tract. For these individuals, the peristaltic failure of the gut leads to severe pain, malnutrition, and, for many, the need for feeding tubes.
The core of their frustration lies with the FDA preventing Americans from accessing the medicine domperidone—used to treat gastroparesis—even though it is already available in other industrialized countries. The agency cites concerns about a potential cardiovascular side effect (Long QT Syndrome), yet as patient advocate Michael Smith pointed out, this risk is no greater than that of many drugs the FDA already allows. No medicine is without risk: acetaminophen (Tylenol) causes more than 500 deaths per year. The FDA’s restrictions have created a paradox: to avoid a potential risk, patients are forced into riskier choices, such as invasive surgeries to remove parts of their digestive tracts, dependence on tube feeding, and the desperate use of dangerous substances for pain relief.
The patient community has been engaged with the FDA for nearly two decades, advocating for a Patient-Centered Drug Development Initiative. However, as the coalition’s letter noted, the agency’s actions, including the failure to approve key drugs and ongoing supply shortages, demonstrate a clear failure to partner with those they are duty-bound to protect.
A System of Unnecessary Barriers
This issue is not isolated to a single condition. A comprehensive review by the Cato Institute identified 69 drugs approved in Canada that the FDA prohibits Americans from accessing, a list that includes treatments for Parkinson’s, cancer, and rare diseases. On average, Canadians have had access to these medications for over 25 years longer than Americans—no nation should be an entire generation ahead of the United States in medical access.
The FDA’s rigid approval process remains the root cause of this disparity, leading to two major problems:
- Drug Lag: Seriously ill patients suffer and sometimes die waiting for the FDA to approve a drug that is already proven safe and effective elsewhere. For example, the FDA withheld approval of insulin icodec, a revolutionary once-weekly insulin injection available in Europe, Canada, and Asia, arguing that the benefits didn’t outweigh the risks, a decision that a “bureaucratically-appointed panel” made for patients.
- Drug Loss: Pharmaceutical companies decide not to pursue approval for promising treatments in the U.S. because the cost and time required by the FDA are prohibitive. A prime example is benserazide for Parkinson’s disease, a drug on the World Health Organization’s list of essential medicines, which is widely available in Canada and Europe but not the U.S.
The FDA has even gone so far as to approve some drugs for animals but not humans. For instance, domperidone and hyoscine butylbromide were both approved by the FDA for horses before humans, an absurd and frustrating reality for patients suffering from their illnesses.
Rethinking Risk: When Something is Better Than Nothing
For millions of patients with debilitating or terminal illnesses, the FDA’s current approach to safety profoundly misaligns with their experiences. The argument that a drug might have risks and therefore should be withheld completely ignores the devastating reality of the patient’s current condition. Faced with unbearable pain, malnutrition, or even death, patients should have the right to pursue treatments that offer hope—even if those treatments carry risks.
Patients should not have to persuade manufacturers to assume the financial risk of navigating the American regulatory system just to access treatments available in other advanced countries. The onus for a safe and accessible drug supply belongs to the government, not patients fighting for their lives.
A Call for Immediate Reform
The FDA’s failures also extend to compounding pharmacies, which play a crucial role in creating customized medications for patients. The agency’s overbroad interpretation of its authority has forced compounding pharmacies to undergo costly and impractical approval processes for decades-old “grandfathered” medications, often making them unavailable to the patients who rely on them.
One remedy lies in embracing international drug reciprocity, a policy in which the FDA would automatically approve drugs that regulators have cleared in countries with comparable regulatory standards, like Canada, the European Union, or Australia, unless the FDA demonstrates compelling grounds to deny them. This change would be a significant step toward enhancing patient autonomy and would reduce the need for Americans to travel abroad for these services. Patients and their doctors—not federal bureaucrats—should decide how to weigh medical risks and benefits.
Jen Springman is the Coalitions Manager at the Goldwater Institute.
