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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  • http://www.biopharmphysicians.com/blog/ Steven Rauscher

    Perhaps we all need to work on changing the language we use in discussing cancer treatment.

    Much of the discussion on cancer, especially upon first diagnosis, revolves around battling the disease. It is difficult to shift the discussion later and introduce words such as palliative care and hospice without an association with resignation or giving-up. In her comments on the current Room for Debate discussions in the New York Times (http://nyti.ms/j7d8iR), Maggie Mahar points out that “20 percent of patients with solid tumors receive chemotherapy during their final two weeks of life”.

    Perhaps all clinicians should discuss supportive care from the very beginning when cancer is first diagnosed. That would mean bringing in a palliative care consult to provide supportive care to improve quality of life throughout treatment, regardless of the outcome of that treatment.

    Steven Rauscher
    http://bit.ly/hP6srm

  • http://www.evimed.com Renee

    It is interesting that you coin the term ‘optimistic bias’ for patients in relation to fully understanding the risks and of the open trial they are about to embark; however, the patient is and will always have optimistic bias. They simply want to live, live to be with dear family and friends. From a psychological perspective, no matter how much logical intervention and palliative care consultation is provided, many patients will want to battle till the end, or until close to the end. Palliative care is then becomes a feasable option for them. They have to be optimistic, perservere or feel obligated to mentally fight to just get through the treatments.

  • Mary

    Unfortunately, too often “optimistic bias” prevents quality of life issues from being properly addressed, early on. We, as patients, are so terrified of “cancer” that we stop living in the name of treatment. I choose not to do that and refused all adjuvant therapy for my first more aggressive cancer. The absolute benefit was not good enough for me to give up such a large chunk of my life to cytotoxic treatments and their long term side effects. I saved my medical insurance a ton of money, money that did not find its way to Big Pharma! This leads to another issue, I have. Too often the results of studies are given in relative benefits and we, as consumers, tend to think in absolutes. This is a very deceiving tactic and I don’t understand why this deception is allowed to stand.