When the FDA is Hazardous to Your Health

October 1, 2020
By Naomi Lopez

Too many terminally ill patients are fighting not just their illnesses, but they’re also combatting slow and outdated bureaucracy that prevents them from trying treatments that could save their lives. Here’s the story of one of those Americans, a cancer patient who is in the fight of his life. (For his privacy, we will call him “Bob,” but that’s not his real name.)

What started as cancerous tumors on Bob’s spine have now spread to his brain. Sadly, one last opportunity at trying an investigational treatment was blocked by the U.S. Food and Drug Administration (FDA) even though the patient, the patient’s neuro-oncologist who is at a world-renowned hospital, and the manufacturer that was providing the treatment for free believed it offered Bob one last opportunity to meaningfully extend his life.

“A team of thoughtful physicians and scientists prepared a treatment plan for [Bob] that has a theoretical chance of working and included safety labs to help ensure the treatment was administered safely. Despite all of this effort, the FDA has gone beyond its regulatory and legal obligation to reach [a] position where it is judging the likelihood of success and criticizing a company for collecting data to help not only [Bob] but other patients that may receive this product in the future,” Bob’s wife recently told the Goldwater Institute.

The FDA rejected the application to treat Bob with the investigational treatment at a low dose under its so-called compassionate use program, a limited exception that allows people to access medications outside a clinical trial. But going even further, the FDA even blocked the use of blood work to monitor Bob during treatment, which is important for ensuring his safety. What makes this case even more disturbing is that, according to Bob’s wife, the FDA also rejected a compromise that would have started with a lower dose and then gradually increased over time.

The fact that the FDA would override the recommendations of the manufacturer and the patient’s doctor but would recommend a treatment protocol that had no realistic chance of helping the patient—and might have been more dangerous as it would have suppressed him immune system with no belief that there would be a therapeutic benefit—is reckless and cruel. “The result is the FDA is ‘protecting’ [Bob] by requiring the company give a dose of [treatment] that the professionals believe will not work, eliminate safety testing because the company might learn something from that exercise, or simply refer him to hospice to die,” his wife said.

But patients who are at the end of FDA-approved options to treat their illnesses are not seeking government “protection.” The Goldwater Institute has filed a Freedom of Information Act request for details about the FDA’s drug approval process in Bob’s case. Unfortunately, that information will come too late for Bob, who is currently preparing to enter hospice care. But fortunately, Congress has the authority – and the responsibility—to also request these records, hold oversight hearings, and investigate if and how the FDA is practicing medicine so that this never happens again.

We already know that less than 1 percent of terminally ill patients are able to take advantage of the FDA’s compassionate use program. While the FDA has improved the efficiency of its compassionate use program over the past couple of years, a system this bureaucratic and powerful—one that would allow the FDA to override the medical expertise of physicians even though it has no legal authority to regulate the practice of medicine—is nothing short of disturbing. Americans also deserve to know if and how often the FDA actively overrides the medical recommendations of these terminal patients’ doctors when they are running out of time and options.

This sad reality is why the Goldwater Institute worked with patients, doctors, and policymakers across the country to create the Right to Try Act, which allows patients—under their doctor’s care—to decide for themselves whether to try medicines that could prolong or even save their lives. Forty-one states passed this groundbreaking reform with overwhelming bipartisan support, and in 2018, it was signed into federal law. Since the passage of the federal Right to Try law, patients who are terminally ill or facing a life-threatening illness have another option. Patients are now being treated, and lives are being extended and saved.

Even though the company that produces the investigational treatment that could have helped Bob was willing to provide the treatment free of charge, they are now, rightfully, concerned that moving forward to treat under Right to Try could put them in the crosshairs of the FDA. That leaves Bob out of options and hope. In America today, no one should be forced to beg the federal government for permission to try to save their own lives—and for too many terminal patients, the FDA is posing yet a further hazard to their health.

Naomi Lopez is the Director of Healthcare Policy at the Goldwater Institute.

To protect his privacy, we have changed the patient’s name. Media interested in learning more about this case should reach out the Goldwater Institute and we will share the detailed medical account and timeline of the patient’s experience which we verified.