The Right to Try: Giving Terminally Ill Patients the Opportunity to Try Experimental Treatments

September 28, 2015

This is the third in a four-part series of articles on consumer-focused health care. These articles are adapted from a Health Law Update presentation Attorney Tyler Wilkinson gave at the State Bar of Wisconsin’s annual Health, Labor and Employment Law Institute in August 2015.

Patients with terminal illnesses may not always have conventional medical options. As a result, patients in this situation often look to alternatives, including experimental medications that are not yet proven or publicly available. That’s the problem: potentially life-saving drugs are not always available to people most in need of those drugs. To address this problem, more than 20 states, including Wisconsin, have introduced “Right-to-Try” laws in the past year in the hope of providing terminally ill patients access to experimental medications.

Here’s how the proposed laws work: under current FDA regulations, a drug company must obtain regulatory approval to use experimental drugs on humans in clinical trials. The regulations strictly control who is given access to the experimental drug to ensure that the drug is used safely and to monitor side effects. One exception to the strict rules on who can use experimental drugs is the “compassionate use” exemption. Under this exemption, the FDA can allow drug companies to give experimental drugs to terminally ill people outside of the clinical trial. In short, the FDA gives drug companies permission to choose to give the experimental drug to terminally ill patients outside of the controlled clinical trial.

Therein lies the rub. The FDA does not order the drug company to give the drug; it gives them permission to do so. Ultimately, the drug company makes the decision on whether to allow a patient access to an experimental drug. Drug companies may be hesitant to give an experimental drug to a terminally ill patient before the drug has been tested through a robust clinical trial process. Drug companies fear that expanded use of an untested drug can cause adverse effects for the patient, which ultimately could derail the regulatory process or lead to a lawsuit.

This is where the “Right-to-Try” laws come into play. These laws provide a limitation of liability under state law to drug companies, drug manufacturers, pharmacists and physicians who are involved in helping a qualified patient obtain an experimental drug. A patient is eligible under the law if the patient is terminally ill, has considered all conventional treatment options, has received a recommendation for the experimental drug from his doctor and has given written informed consent for the use of the experimental drug. Contrary to the title, the “Right-to-Try” laws do not give the patient a right to use the experimental drug; it only removes some fear of liability for the drug companies.

Wisconsin’s proposed law has popular and bipartisan support, but it is not without criticism. Commenters argue that these laws empower terminally ill patients and provide them with choices when it comes to end-stage diseases. Others are not so sure, and question the ethics of a drug company deciding whether to allow access to an experimental, but potentially life-saving, treatment. Health care providers also question the safety of giving untested drugs to the most vulnerable patients. Still others fear that these laws create false hope for terminally ill patients who obtain a compassionate use exemption but who are denied by a weary drug company.

We are fairly confident the Right-to-Try bill will become a law in Wisconsin, but we are mindful of the questions the new law will raise. Will the FDA follow the states’ leads and grant more compassionate use exemptions? What criteria will drug companies use to determine which patients are eligible for experimental drugs? Will physicians be willing to recommend experimental drugs to terminally ill patients, even without the attendant fear of liability? Is this entire thing a “cruel sham” on dying patients as some commentators have argued?

These questions will be answered only with time. But the nation-wide push for “Right-to-Try” laws is another example of how patients are becoming more empowered to take control of their health care and treatment.

For more information about "The Right to Try: Giving Terminally Ill Patients the Opportunity to Try Experimental Treatments," contact Tyler K. Wilkinson at twilkinson@axley.com or 608.283.6783.