The fundamental premise behind RTT is the concern of libertarians and patient advocates that the bureaucratic processes inherent in the FDA regulations regarding entry into clinical trials or applying for exceptional access have been onerous, time-consuming, and often approved too late for a terminally ill patient to receive the experimental treatment. From the general public standpoint, the FDA is a “black box,” and its processes and regulations are poorly understood, giving rise to frustrations regarding individual patient access to experimental therapy. Paperwork for both the patient and physician has been voluminous, which has discouraged many patients from even beginning the process. Proponents of RTT have touted state and federal laws as giving patients the “right” to have access to experimental therapies—some have suggested that RTT is akin to “right to die” laws in several states—as part and parcel of medical self-determination and a “right to live.” To date, 41 states have passed RTT laws in addition to the recent federal law.
Explore this issue:October 2018
RTT laws provide a mechanism for patient and physician to identify an experimental therapy (usually a drug) that is pertinent to the patient’s disease, and to jointly request the opportunity to try that therapy outside of FDA-approved clinical trials. The manufacturer must agree to the request, and would work with the patient’s physician to develop a specific treatment program for that patient. Who bears the costs of the treatment is usually determined on a case-by-case basis, and this determination may have an impact on the patient’s decision to proceed. There is an informed consent document that must be signed by the patient, and it is the physician’s responsibility to provide the clinical risks and benefits information—which may be problematic for an experimental treatment that has only been tested in a Phase I study. The physician is nominally required to receive approval from an Institutional Review Board for the treatment plan, which may or may not be expedited by the Board. The physician is required to provide outcomes data to the drug manufacturer for the course of therapy. Of note, the drug manufacturer is under no obligation to provide the experimental therapy to a patient via this pathway. The RTT laws explicitly provide protection for both the treating physician and the drug manufacturers from liability of death and serious outcomes from treatment through this access system.
There are a number of plausible alternatives to RTT for the patient to consider, including clinical trials, expanded access programs, and single-patient expanded access.