Unrealistic optimism in early phase new drug studies

In a thoughtful posting in the New York Times, Dr. Pauline Chen, a well respected observer of the doctor-patient relationship, points out an ethical dilemma that faces cancer researchers working in drug development conducting early phase (I) new drug studies. Ethicists have identified that patients in these studies often have unrealistic optimism about their personal outcomes despite a detailed and thorough informed consent process. They argue that optimistic bias may prevent truly informed participation in these studies.

Oncologists practicing in the United States have been aware of this type of issue for many years. Partly as a result of the unrealistic optimism phenomenon, they have received a somewhat undeserved bad rap of encouraging patients to take treatment even when the chance of benefit is slim. Faced with a patient who has exhausted the treatments that have a “reasonable” chance of benefit, the oncologist must navigate the treacherous waters of fully informing the patient of available options which inevitably include both palliative care as well as anti-cancer treatments with small chances of success. Although palliative care has been shown to be associated with longer survival and better quality of life than chemotherapy in this setting, patients perceive that this option has no “home run” potential (although most experienced oncologists can remember a patient or two who survived months or even years beyond reasonable expectations while receiving hospice care).

Despite extensive discussion about the active nature of palliative care, patients often equate this option with “giving up.” The value of receiving a treatment intended to kill cancer cells is over estimated since patients have unrealistic optimism about their personal outcomes. The oncologist, who plays the role of both the medical expert and the patient advocate, is faced with the medical expert task of more aggressively countering this unrealistic expectation while pursuing the patient advocacy task of preserving the hope that is often an important underpinning of the patient’s quality of life. Even in the hands of the most skilled oncologist, the patient often opts for more chemotherapy with the hope and high expectation of achieving a uniquely positive outcome. The family either is reluctant to challenge their loved one’s hope and optimism or actually shares them.

What is the solution? One of the proposed remedies is the introduction of the palliative care expert and concept early in the course of treatment, well before all reasonable anti-cancer therapy has been exhausted. Perhaps, with earlier involvement of the palliative care specialist, patients will be able to apply their unrealistic optimism to a program focused on quality of life rather than killing cancer cells. The recently adopted approach of discussing all of the reasonable treatment options and the expected disease course prior to beginning initial therapy may also be helpful by creating the expectation that the goal of therapy will change after a pre-specified number of courses of anti-cancer treatment. Although this may help ease the transition to quality-of-life-directed care for some patients, I suspect that many will reevaluate their options when the scheduled end of anti-cancer treatment arrives and will choose to act on their unrealistic optimism and seek additional chemotherapy or a phase I study to believe in.

Perhaps the best solution for those who require unrealistic optimism to maximize their quality of life is one that medical oncologists have sought for years—a non-toxic, inexpensive, convenient oral targeted treatment with at least occasional instances of unexpected success. If quality of life through personalized medicine is our goal, is it unreasonable for us to target the hope and unrealistic optimism “receptors” in these challenging patients?

Richard Leff is Chief Medical Officer of Conisus.

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