At first glance, the new Right to Try law sounds like a victory for desperately ill patients who have run out of treatment options. Signing the legislation on May 30, 2018, President Trump declared that “patients with life-threatening illnesses will finally have access to experimental treatments that could improve or even cure their conditions.”
However, many leading patient advocacy groups, including the American Cancer Society Action Network, Susan G. Komen, and the American Lung Association, oppose the law because of lack of U.S. Food and Drug Administration (FDA) oversight and the possibility that it could feed false hopes of vulnerable patients. Earlier this year, those groups and dozens more voiced their opposition in a letter to Congress, warning the legislation would “remove FDA’s … important safety measures.”
Before the federal law was enacted, 40 states had already passed their own Right to Try laws. Like the new law, state laws allowed certain patients to bypass the FDA and gain access to drugs that had already completed phase 1 clinical trials. Phase 1 trials test drugs for the first time on a small number of healthy people who don’t have the targeted condition to identify proper dosages and any significant side effects.
Are you a candidate?
You may qualify for an experimental drug if you:
- Have a life-threatening illness
- Have no other treatment options
- Can’t participate in a clinical trial
The issue, like so many these days, has become deeply polarized. Now that Right to Try is law, doctors are trying to determine how to advise their seriously ill patients in a balanced, realistic, and compassionate way about what the legislation means for them.
Several key points are important to know, especially if you’re facing a life-threatening illness.
- The new law offers potentially easier access to investigational drugs but at potentially greater risk. Right to Try allows doctors to approach pharmaceutical companies without FDA approval. But, critics say, with no FDA oversight, patients may be at greater risk of being harmed by unproven therapies. According to an article in JAMA Internal Medicine in March, two-thirds of drugs that pass phase 1 clinical trials subsequently fail their phase 2 trials, and even more fail phase 3 trials.
- Patients already had access to investigational drugs. The FDA has long offered expanded access—also called compassionate use—to experimental drugs. Requests are typically processed within a few days or immediately for emergencies. That’s not enough, supporters of the new law say. They insist that critically ill patients shouldn’t need to go through the FDA when time is of the essence.
- Pharmaceutical companies can say no. The new law gives patients and doctors the right to ask for a drug in development, but it doesn’t require manufacturers to provide it. A company may turn down a request for reasons such as a limited supply or concerns over its safety.
- Doctors who steer patients to experimental drugs and the pharmaceutical companies that dispense them won’t be held liable if something goes wrong. Drug makers can’t be held accountable if the treatment is later found to be unsafe or unforeseen complications arise.
- Insurers aren’t required to cover investigational drugs, associated medical costs, or treatment for any adverse effects. You may end up paying for everything out of your own pocket. Under some state laws, you may give up your entitlement to future health insurance coverage. Be sure to ask your insurer about costs and coverage before consenting to treatment.
- Patients’ hospice and home health care insurance may be in jeopardy. Under some earlier state laws, insurers could stop payments for hospice and home care when patients tried investigational treatments. You’ll need to check with your health insurer to see whether this holds true under the federal law.
- Right to Try won’t release a flood of promising new drugs. Critics worry that people have been given a false sense that many terminally ill patients are being denied all kinds of wonder drugs, which will now become available. Certainly, there are potentially lifesaving drugs in the pipeline. If a treatment is promising, researchers and pharmaceutical companies are as anxious as anybody to get it approved and into the hands of patients. But testing a new medication, and learning how best to use it, takes time. The new law won’t change that.
Finding an experimental drug
If you are terminally ill, your doctor might suggest an experimental drug. But your doctor may be unaware of all drugs under investigation that could help you. You might want to do your own research by:
- Visiting clinicaltrials.gov. You and your doctor can search for applicable experimental drugs by using the search terms “expanded access” or “compassionate use.”
- Checking the websites of health organizations, which may report on drugs that are currently under research.
- Searching the websites of drug manufacturers for their policies if you’d like to learn more about a specific drug.
The bottom line
If you (or a family member) have a life-threatening illness and have exhausted all treatment options, work with your doctor to identify experimental therapies—as well as clinical trials—that may be worth trying. Carefully weigh all potential benefits and risks. It’s important to understand that the treatment outcome is unpredictable and can possibly do more harm than good.
This article first appeared in the September 2018 issue of UC Berkeley Health After 50.