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.
Explore This IssueOctober 2018
Ethical Decision Making
The main ethical challenge here for clinicians is balancing the possible risks and benefits for the patient’s consideration, a task that must take into consideration all four of the ethical principles—self-determination (autonomy), beneficence, non-maleficence, and social justice. As with all ethical dilemmas, there are some generalizable precepts for all patients and some specific issues particular to each patient’s condition. Furthermore, in the present debate, the polarity seems to be primarily public opinion versus the medical-scientific community.
Self-Determination: Proponents of RTT posit that the federal regulations (FDA) and requirements for the patient and physician that were in place until recently have been excessively burdensome, and response time has been so long, that near-terminal patients who are seeking the opportunity to participate in a non-clinical trial have died before a decision was made. Some patients want direct access to the drug manufacturers without onerous governmental oversight, and feel it is their “right.” Opponents believe that there is no given “right” in the laws, because the drug companies have the final decision on providing the drug; therefore, RTT does not provide a guarantee for access to therapy, so patient self-determination may not be fulfilled.
Beneficence: Even with therapeutics that have passed FDA approval, there are always questions about benefits, especially for drugs given in terminal stages of a serious disease. Because the treating physician must bear the responsibility for assessing what is known about a drug or other therapy that may have only completed a Phase I trial, the clinician may be at a disadvantage when it comes to providing adequate information to the patient about potential benefits for their consideration. Early salutary reports from a Phase I study may not be sufficient data to support a patient’s interest in seeking an RTT therapy exception. Even after standard therapy has failed, there are acceptable alternative care methodologies, including hospice and palliative care in a terminal patient. The treating physician must honestly explain the possible benefit(s) to be derived from an experimental therapy that has not yet been approved by the FDA. This is also the case with expanded access programs and single-patient expanded access, although these programs have additional oversight from the FDA for comfort.