Mary is dying. Does she have any drug options left? - (7/2/2019)

By Dr. Ron Gasbarro

When your life hangs in the balance, should you have the right to save it? Mary has a terminal illness. She has exhausted her treatment options. She does not qualify for a clinical trial because of her dire condition. Her doctor tells her that an experimental drug exists which may help her. Nevertheless, there are a few caveats of which Mary should be aware. First, the drug has only passed a Phase 1 clinical trial. Hence, it has not been completely studied. What does this mean? 

The Food and Drug Administration (FDA) mandates that an experimental drug must undergo several phases before the FDA approves it for use in a certain disease. A Phase 1 clinical trial assesses whether the drug is safe for humans. If the drug does not kill the lab rat in a pre-clinical trial, then it can tried on a human in a Phase 1 trial. But note: A Phase 2 clinical trial is conducted to evaluate the effectiveness and safety of the drug. A Phase 3 trial involves thousands of patients and can continue to run for 2 to 5 years to see if any toxicities exist. Therefore, the drug Mary’s doctor suggested may neither work nor be safe. Should Mary try it? What does she have to lose? 

Wait, there’s more. The manufacturer of this experimental drug will not have to wade through a mountain of red tape for the FDA to approve the drug. That could take years – time that Mary does not have. In fact, the drug manufacturer is willing to make the drug available to Mary right away. The plan is now for the manufacturer, Mary’s doctor, and Mary to work together to get her the treatment she is counting on, without government mumbo-jumbo (i.e., ethical oversight for safety). 

Is this legal? If Mary’s drug is that promising, then why is it not available to the public? In 2018, the executive branch of the US government passed the Right-to-Try law. Right-to-Try protects the right of terminally ill patients to pursue treatments that have passed part of the FDA approval process but are not yet on pharmacy shelves. 

Even though the Right-to-Try Act is a Federal law, several traps can ensnare the patient. For example, the law will make it easier for unethical companies or prescribers to exploit desperate patients. The law would allow companies to charge as much as they want for an experimental drug that may not work and could be fatal. The law would prevent patients from suing the manufacturer if the treatment harms or kills them. In other words, the law will give patients more access to experimental treatments but without the safeguards that they would otherwise have. Increasingly, the Right-to-Try law sounds like it would benefit Big Pharma more than the patient. Not everyone is buying the legitimacy of this law. Major cancer organizations opposed the passage of the law, including the American Society of Clinical Oncology, the American Cancer Society Cancer Action Network, and The Leukemia & Lymphoma Society. 

The primary role of the FDA is to protect the public’s health by ensuring the safety and effectiveness of drugs, vaccines, medical devices, and food products. The law reduces FDA oversight of drug regulation. In a Phase 1 study, one gets a very low dose of the drug. If there are only minor side effects, the next few patients may get a higher dose. This process continues until doctors find a dose that is most likely to work while having an acceptable level of side effects. What is “an acceptable level?” What is tolerable to you may not be tolerable to Mary who is already suffering. Let’s turn the tables. If you were terminally ill and your doctor suggested an experimental drug, would you take it or not? 

Ron Gasbarro, PharmD, is a registered pharmacist, medical writer, and principal at Read more at


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