Ethical Challenges of ‘Right to Try’ Laws for Clinicians

Non-Maleficence: Long thought to be the primary responsibility of the physician (“do no harm”), non-maleficence addresses the potential negative effects of experimental drug therapy that the patient needs to consider in an informed consent. All of the potential side effects and complications cannot be known, for those drugs and devices that have only passed Phase I trials and may be in Phase II or III have limited discoverable data on adverse effects, including death. Each terminal or near-terminal patient may have unique organ compromise or risks that could enhance the potential for catastrophic side effects. Explaining this inadequate data effect to a patient and their family can be unsettling for them and stressful for the physician. Additionally, electing to petition for a RTT may disqualify the patient from subsequent hospice or palliative care.

Social Justice: Depending on the state, RTT laws may require manufacturers to provide the experimental therapy without cost to the patient (i.e., Texas), but in other states, the patient may be held responsible for the cost of the therapy. Raising funds for treatment takes time, and some patients do not have sufficient time remaining in their lives. Additionally, those patients with lesser resources and inadequate healthcare access may be at a disadvantage with respect to ability to secure physician support to apply to RTT and to pay for the drug if required.

Both proponents and critics of RTT tend to agree that there is a failure of communication between the scientific community and the public with respect to the issues and policies associated with clinical trials, expanded access, and right to treat—this must be improved. There should also be additional clarification of the risks to clinical trials for catastrophic adverse outcomes associated with patients in the RTT access pathway, for drug manufacturers may be reluctant to jeopardize their clinical research by providing experimental drugs to terminal patients. Conversely, there is justifiable concern in public discourse that the federal government has been slow to streamline the processes for expanded access for patients who do not qualify for clinical trials, and the RTT movement has stimulated positive changes in procedures and policies.

In the balance, only the patient and treating physician, working together, can develop the best approach for terminal illness care if the patient wishes to consider an exception to FDA clinical trials. The physician must become as knowledgeable about the available data as possible in order to counsel the patient and family in their deliberations. Serious discussions should focus on both the objective information on the patient’s condition and how that might affect experimental therapy, and the subjective disclosure of the patient’s personal wishes for the remainder of life. Supportive care, palliative care, and hospice remain the gold standard for end-of-life care for terminal patients, with physicians providing empathy, honesty, and compassion along the path. Patient self-determination must be balanced with the prospects of benefit, risks of catastrophic adverse events that could further shorten meaningful life, and an equality of care access for all terminal or near-terminal patients as appropriate.