Over the past year, state Right-to-Try (RTT) laws that claim to enable terminally ill patients to access unapproved, experimental drugs, biologics, and devices have swept the nation. As of early May, seventeen states have enacted RTT laws (most recently, Florida and Minnesota), and bills creating such laws are currently pending in over twenty state legislatures.
Although these laws have created an expectation that terminally ill patients will be able to quickly access potentially life-saving treatments by being exempted from the rules of the U.S. Food and Drug Administration (FDA), this expectation is, quite simply, false.
Federal preemption laws prevent states from creating workarounds that purport to supersede the extensive regulatory scheme enforced by the FDA. Contrary to the hype surrounding them, so-called Right-to-Try laws do not create any additional “rights” for patients. And, even if they did, RTT laws fail to address fundamental questions of patient welfare and public health inherent in considering whether, and which, individuals should have access to experimental treatments outside of clinical trials.
While each law has unique aspects, the RTT laws generally aim to permit patient access to an investigational treatment if: (1) the patient is terminally ill; (2) a physician recommends use of the treatment, (3) the patient provides informed consent; and (4) the treatment has completed a “Phase I” clinical safety/dose limitation trial. Both manufacturers and physicians would receive liability protection against claims arising from adverse events caused by the experimental treatments.
Manufacturers may choose to charge for the treatments, and health insurers are not required to cover the costs of the treatments or care resulting from their use. What the laws do not do is compel anyone or any company to fulfill a patient’s request for an experimental drug, device, or biologic. The result will be false hope and unmet expectations on the part of patients with no therapeutic options.
For decades, FDA has enforced comprehensive federal regulatory pathways for approval of medical therapies, including drugs, which are thus far the primary focus of the RTT movement. Section 505 of the Food Drug And Cosmetic Act (FDCA) prohibits the sale or distribution of a drug into interstate commerce until the drug is proven safe and effective. For new drugs, manufacturers must submit to FDA an extensive New Drug Application (NDA), which typically requires the completion of multiple “phases” of clinical trials to establish the drug’s safety and efficacy.
Recognizing that there may be circumstances in which individuals do not qualify to participate in a clinical trial but may benefit from treatment with an investigational drug, FDA has an “expanded access” pathway whereby an individual may access such products if: (1) the individual has a serious or immediately life-threatening condition and there is no satisfactory alternative therapy; (2) the potential benefit outweighs the treatment risks; and (3) providing the investigational drug will not interfere with the clinical trial’s process or otherwise compromise the product’s development.
It is well-established that the U.S. Constitution’s Supremacy Clause cements federal law as the “the supreme Law of the Land … any Thing in the Constitution or Laws of any state to the Contrary notwithstanding.” Therefore, state laws cannot permit manufacturers to provide patients with access to unapproved drugs when the FDCA mandates that “no person shall introduce or deliver for introduction into interstate commerce any new drug,” unless FDA has approved an application for such product or otherwise authorized use of the investigational drug through a clinical trial or an expanded access program.
If RTT laws are understood as actually providing a right to direct access to an investigational product without FDA approval or oversight, they would be “preempted” by federal law, meaning the federal laws are so powerful as to effectively nullify their state RTT counterparts. Indeed, several federal courts have already concluded that FDA’s comprehensive regulatory regime governing the manufacturing, approval, labeling, and distribution of drug products preempts state laws designed to legislate in this area. If challenged in court, we anticipate RTT laws will be similarly treated.
Although complete preemption is likely, there is one area of the RTT laws that may not be subject to nullification: Because the FDCA does not specifically regulate tort liability, which is typically a creation of state law, a court may conclude that liability protections in the RTT laws are not preempted by federal law. One of the reasons why manufacturers are hesitant to permit access to unapproved drugs under expanded access programs is the specter of tort liability. These liability protections may provide comfort to manufacturers when use of investigational drugs is authorized under FDA’s expanded access programs. However, potential liability is far from the only reason that a manufacturer may decline to make its experimental treatment available to a sick patient.
Public Health And Patient Welfare
Putting aside the invalidity of RTT laws and the false hope they create, these laws also fail to address more fundamental health and safety issues. RTT laws seek to grant vulnerable patients access to unproven and potentially harmful drugs or other medical products without any expert safeguards, such as scientific or ethical review by an Institutional Review Board or similar body.
Moreover, these laws undermine the clinical trial infrastructure regulated by FDA, which was developed to ensure that medical products are tested on a sufficiently diverse population in a scientifically sound manner to bring safe and effective products to market. By allowing anyone to obtain an experimental treatment outside the confines of a regulated clinical trial, RTT laws reduce incentives to participate in clinical trials, increasing the complexity of recruiting sufficient study participants.
Further, due to complex manufacturing requirements, there may be only limited amounts of investigational product available, so RTT laws, if they resulted in patients receiving investigational treatments outside of clinical trials, may divert resources needed for those clinical trials. As a result, the trials—upon which FDA approval ultimately depends—may be slowed. Although touting ostensibly laudable goals, RTT laws do not take any steps to navigate the thorny issues raised by use of experimental treatments, including potential delay in getting FDA approved products to the market, where they can benefit the public at large.
It is only a matter of time before courts nullify RTT laws. In the meantime, these laws offer vulnerable patients misplaced hope and do not attempt to minimize the serious health and safety risks inherent in treatment with unproven treatments. State legislatures still evaluating the merits of RTT laws should think twice. Patients facing terminal illnesses have enough issues to navigate without being given false hope through legislation that is ethically, legally, and clinically flawed.
See earlier Health Affairs coverage of Right-to-Try Laws.
The authors gratefully acknowledge the assistance of Anne Pierson Allen, an associate at King & Spalding LLP, for her research on this article. David Farber and Preeya Noronha Pinto are partners in the Washington, D.C. office of the FDA & Life Sciences Practice at King & Spalding.
Dr. Caplan and Dr. Bateman-House serve as non-voting, non-paid Chairperson and deputy chairperson of the Compassionate Use Advisory Committee (CompAC), an external, expert panel of internationally recognized medical experts, bioethicists, and patient representatives formed by NYU School of Medicine, which advises the Janssen Division of Johnson & Johnson about requests for compassionate use of some of its investigational medicines.
The views expressed in this post are those of the authors, and are not the views of their respective firms or institutions.