Q: My patient is terminally ill. There may be a treatment, but it isn’t FDA approved. Do patients have a right to try investigational treatments, and what are the ethical implications of administering potentially harmful medications?
A: There are multiple pathways for terminally ill patients to obtain investigational medications. The intent is to enable dying patients to receive investigational treatments outside of clinical trials and before approval by the U.S. Food and Drug Administration (FDA). Right-to-try legislation and compassionate use are the common routes. Right-to-try legislation empowers patients to bypass the FDA approval process and go directly to drug manufacturers, but it doesn’t exist in every state. Compassionate use regulations were established by the FDA to provide dying patients with faster access to experimental treatments. Compassionate use is available in all states and is physician initiated.
Right-to-try laws and compassionate use regulations enable patients to obtain treatments that have completed phase 1 clinical trials, which evaluate safety but not efficacy. Patients using the right-to-try or compassionate use loopholes subvert the clinical trials process.
Drug developers and manufacturers are reluctant to provide access to investigational treatments. Concerns include negative publicity and impact on future FDA approval from treatment failures and patient harm, effect on supply of investigational agents for clinical trials, and decreased availability of appropriate patients to participate in clinical trials. The FDA has developed a stringent approval process to protect the public from treatments that do more harm than good.
Ethically, the challenge is to balance a patient’s desire for hope and potential for benefit with the obligation to do no harm. Multiple provisions in the American Nurses Association Code of Ethics for Nurses with Interpretive Statements inform this issue.
Provision 1 states: “The nurse practices with compassion and respect for the inherent dignity, worth, and unique attributes of every person.” Establishing a trusting relationship, considering patients’ needs, and respecting their values and right to self-determination would seem to require nurses to advocate for, and assist patients to obtain investigational treatments.
Provision 2 states: “The nurse’s primary commitment is to the patient…” This would also indicate the nurse should advocate for access to investigational treatments above concerns for the clinical trials process. However, this is somewhat conflicting, as Provision 2 also identifies the patient as a population and community. There is a risk to the population of patients in need of safe and effective treatments when the clinical trials process is subverted for individual, unapproved use.
Provision 3 states: “The nurse promotes, advocates for, and protects the rights, health, and safety of the patient.” This provision addresses the importance of informed consent for participation in research. It would apply for access to investigational treatments outside the research process. Special concern for vulnerable patients is also adressed in Provision 3. It describes the ethical obligation to protect health and safety, which seems to indicate that investigational treatments should not be provided.
Provision 4 states: “The nurse has authority, accountability, and responsibility for nursing practice; makes decisions; and takes action consistent with the obligation to promote health and to provide optimal care.” Nurses bearing primary responsibility for the nursing care, their judgments, decisions, and actions need to reflect on the provision of investigational treatments because the harms that can result are unknown. Creating false hope for terminally ill patients and families can be harmful when it prevents preparation for the dying process.
Ultimately, it’s a delicate balance between patient autonomy and informed consent, on the one hand, and the requirement to promote safety and prevent harm on the other, which must be considered in right to try and compassionate use of investigational medications. Nurses, supported by their code of ethics, should evaluate each situation individually and determine the ethically appropriate course of action.
Response by Donna Casey, DNP, MA, RN, NE-BC, FABC, chair of the ANA Ethics Advisory Board, and Danielle Sawyer, BS, paralegal and second-year student at Delaware Law School.
February 2018 Frontline FINAL