New legislation, known as the federal Right-to-Try bill, was passed by Congress and signed into law by the president in late May. While the new federal law creates uniformity, it’s important to know that 38 states* have passed Right-to-Try laws since 2014. These laws and now the federal law allow doctors with terminally ill patients to petition drug companies so that experimental drugs can be used under certain circumstances, even though those drugs have not been FDA-approved for use…and may never be.
WHAT MOST PEOPLE DON’T REALIZE : A person with a terminal condition and no available treatment options has more than the new Right-to-Try law to turn to for assistance. An existing program, known as expanded access, has already been achieving the same purpose with greater safeguards and consumer protection.
THE DOWNSIDES OF RIGHT-TO-TRY: The state and even the new federal laws are complicated and often come with strings attached. How these laws work: Doctors can ask pharmaceutical companies to give a terminal patient an experimental drug that has successfully gone through Phase 1 clinical trials, even if that patient is not enrolled in the formal clinical trial program. While patients seeking treatment under Right-to-Try laws can gain access to potentially lifesaving experimental treatments, they don’t get the FDA’s advice on dosage and safety (based on the information from clinical trials), as routinely occurs when using the existing expanded access program (described in more detail below).
Under Right-to-Try laws, a pharmaceutical company also can refuse to participate in the program. And in half the states with Right-to-Try laws, insurers can deny paying for hospice coverage should a patient require it after using a Right-to-Try drug. Some state laws also allow insurers to deny coverage for the costs to treat any complications that result from the drug.
BETTER ALTERNATIVE: In 2009, due to pressure to give patients access to experimental drugs, Congress amended the Federal Food, Drug and Cosmetic Act to create an expanded access (sometimes called “compassionate use”) program so that patients could get experimental treatments when enrollment in clinical trials is impossible. This program is effective and efficient. How it works: Patients and/or their doctors fill out a request form (taking less than an hour) and submit it to the FDA. In emergency situations, the FDA will reply within 24 hours. Overall, the agency approves 99% of all requests. Note: Patients usually find out about experimental treatments from their doctors but also can research such drugs online at ClinicalTrials.gov to see which ones have passed at least Phase 1 clinical trials.
But more importantly—and something that does not happen in Right-to-Try requests—the FDA will make safety changes before approving the request, adjusting factors such as dosage or frequency of treatment, based on the information already gleaned from testing and clinical trials. This is far safer for the patient and helps the doctor better use the drug. Unlike most state and the new federal Right-to-Try programs, the expanded access protocol typically includes the experimental drug free of charge to the patient.
Given the choice, I’d rely on the expanded access program to get a much-needed treatment!
*For a list of states that have Right-to-Try laws, go to RightToTry.org.