Indiana Governor Mike Pence signed into law Indiana’s new Right to Try legislation on March 24, but this law (and similar legislation proposed and enacted in other states) may have already catalyzed expanded access for terminally ill patients to potentially life-saving experimental drugs. Indiana’s Right to Try Law (House Enrolled Act 1065) is based on model legislation from the Goldwater Institute and is similar to legislation introduced in over 20 states since 2014. Indiana’s law establishes a state-sanctioned process to make experimental drugs, biologics and devices more easily accessible for terminally ill patients than under existing federal compassionate use guidelines of the Food and Drug Administration (“FDA”). In response to this rising tide of state legislative activity and growing popular outcry for faster, simpler access to life-saving treatments, the FDA proposed in February 2015 a dramatically simpler and less burdensome process for terminally ill patients to access experimental treatments.
The FDA and the courts have struggled for years to balance a terminally ill patient’s right of access to try experimental treatments against the FDA’s legislative mandate to protect the broader public by approving only those drugs, devices and biologics that have been shown by substantial evidence to be both “safe and effective.” In 1979, the U.S. Supreme Court declined to grant terminally ill cancer patients’ access to Laetrile, a drug not recognized as safe and effective under FDA law. The recent movie Dallas Buyers Club dramatically portrays the poignant struggle of AIDS patients in the 1980s to get potentially life-saving experimental treatments not yet approved by the FDA process. In response to activism by those AIDS patients, the FDA expanded and liberalized its rules to expand access to experimental drugs for terminally ill patients in 1987. More recently, the Abigail Alliance for Better Access to Experimental Drugs sought to stop the FDA from limiting access of terminally ill patients to potentially life-saving experimental drugs. The federal courts struggled with this case for several years but ultimately upheld the FDA law and the limitations of its drug approval process and declined to recognize a terminally ill patient’s fundamental right of access to experimental drugs in this context.
As an alternative to the FDA’s current process, Indiana’s law is available for patients who are diagnosed as terminally ill by a treating physician, who do not have a comparable FDA-approved treatment available, and who provide informed, written consent after proper disclosure of the potential risks. Indiana’s process grants this “qualified patient” access to drugs, devices and biologics that have completed FDA Phase I review (testing for safety only on a small number of patients) but have not been approved by the FDA for general use. Further, if the experimental treatment is provided in a hospital setting, the hospital governing board (or committee) must approve the treatment.
While Indiana’s new law shields the treating physician and the drug maker from potential state law liability, it leaves open the possibility of liability or further consequences under federal law. For example, it is unclear what consequences a drug maker and its experimental treatment may face from an adverse event or otherwise under the federal drug approval process. Since the ultimate goal of national market access rests with the same federal agency (FDA) whose compassionate use guidelines are supplanted by this state law, most drug makers may be reluctant to risk problems with the FDA. Importantly, Indiana’s law does not require a drug maker to make its experimental treatment available to qualified patients. With that cloud of uncertainty and potential risks, it is unclear whether drug makers will voluntarily choose to participate under this state law program.
Further, Indiana’s Right to Try Law raises the legal question of whether it is preempted by existing federal law granting the FDA authority over experimental drug regulation. The U.S. Constitution’s supremacy clause generally provides that federal laws (such as laws establishing the FDA and its drug approval process) are the supreme law of the land, thereby preempting any contrary state law.
Still, this new Indiana law and the wave a similar legislation introduced at statehouses across the country has undoubtedly increased popular pressure on the FDA to change its compassionate care program to help terminally ill patients. In February 2015, the FDA published new guidance on expanded access to experimental treatments for compassionate care and introduced a new, simpler form to request approval. The FDA estimates this new, simplified process will take physicians around 45 minutes to complete, while the prior process took an estimated 100 hours. In this way, Indiana’s new law has already helped catalyze change for terminally ill patients.