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.
Typically, for a patient and physician to be thinking of requesting RTT, it has already been determined, after a thorough investigation (ClinicalTrials.gov), that there are no available clinical trials for which the patient would be eligible. Expanded access programs (EAP) may have some similarities to clinical trials, but are coordinated by drug manufacturers through permission from the FDA. These programs are designed to enroll patients who are not candidates for a specific clinical trial but who may benefit from the treatment before the drug is approved by the FDA. Unlike formal clinical trials, there is no control group. Single-patient expanded access can be considered if there are no clinical trials or expanded access programs that apply to a specific patient. Typically, the physician requests this access from the FDA as the patient’s provider, under either emergency or non-emergency conditions. This process has historically been time-consuming for the physician (although recent changes in regulations have reduced the paperwork burden), and a successful access is not guaranteed. Further information on navigating the various treatment options can be found here.