FDA Holds the Power of Life and Death: A Tale of Two Patients

Two patients are in troubling health situations — one patient you know, and one you don’t. Their care experiences paint a wildly different picture of how the U.S. Food and Drug Administration (FDA) treats patients in need of immediate attention — and what should be done to ensure more access to innovative treatments when available.

 

One patient’s story is undoubtedly familiar to all Americans: After his COVID-19 diagnosis, President Donald Trump became the most high-profile beneficiary of the FDA’s Expanded Access program, often referred to as compassionate use, which allows the FDA to grant a seriously ill patient access to a non-FDA approved treatment. To treat the president, the FDA signed off on the compassionate use request for a coronavirus antibody cocktail, a treatment currently in clinical trials.

 

The other patient’s story is one you haven’t heard before. A cancer patient is in the fight of his life after cancerous tumors on his spine spread to his brain. (Let’s call him “Bob.”*) Bob and his doctors submitted a request for an investigational treatment to treat his cancer under the FDA’s compassionate use program. Even though both Bob’s neuro-oncologist and the treatment’s manufacturer believed the drug could offer Bob one last opportunity to extend and improve his life, the FDA rejected the request. The FDA was only willing to approve a very low treatment dosage that Bob’s medical team did not believe would help him. Considering that Bob’s immune system would be weakened by the treatment, a dosage that wouldn’t help him fight his disease but would make him more susceptible to other illnesses would have been even more reckless.

 

While the FDA acted swiftly to facilitate President Trump’s care, Bob was not so lucky. Instead, the FDA forced Bob to “negotiate” to save his own life and ultimately abandoned him — relegating him to hospice care despite an available treatment. As Bob’s story shows, the compassionate use program doesn’t always live up to its name.

 

Bob is not alone. Fewer than 1 percent of terminally ill patients are able to take advantage of the FDA’s compassionate use program, because of an overly complex application that can take a physician dozens of hours to complete. And although the FDA has improved the efficiency of its compassionate use program over the past couple of years, Bob’s case shows that the FDA still has the power to override the medical expertise of physicians even though it has no legal authority to regulate the practice of medicine.

 

Fortunately, there is a way to put patients and their doctors at the center of life-and-death healthcare decisions without forcing them to navigate the FDA’s compassionate use process. Under Right to Try, a law signed by the president two years ago, patients facing terminal or life-threatening illness who have exhausted their other treatment options can seek access to an investigational treatment, as long as the patient’s doctor and the treatment’s manufacturer authorize it — without requiring the government’s approval first. In short, Right to Try helps make investigational treatments more accessible, while still ensuing medical oversight and patient safety.

 

Even though the company that produces the investigational treatment that could have helped Bob was willing to provide the treatment at no cost, it is now understandably concerned that moving forward to treat under Right to Try, which does not require FDA approval, could put them in the agency’s crosshairs.

 

The heartbreaking reality is that the FDA is more than willing to act in a matter of hours for a prominent individual but can leave patients like Bob waiting and “negotiating” for their lives for 30 days — the maximum legal time limit the agency has to respond to an application — and may ultimately reject the treatment recommendations of the patient’s medical team. That is nothing short of appalling, and it serves as a harsh reminder that the FDA can use its veto stamp to block a potentially lifesaving treatment for a patient whose life is hanging in the balance but whose story isn’t the stuff of media headlines.

 

If the FDA allows the president’s medical team to obtain an investigational treatment, then don’t all patients deserve this same level of care, especially patients who are much sicker and have already exhausted all other available treatments? Today, Bob is preparing to enter hospice care knowing that there’s a treatment that was made available to him and that his doctors were recommending. Unfortunately, Washington bureaucrats had the power to decide when he should stop fighting for his life.

 

* 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 for the detailed medical account and timeline of the patient’s experience, which we have verified.

 

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