On Equipoise and Emerging Technologies

Abstract

TO THEEDITOR: Recently, Goitein and Cox 1 published an editorial in this journal questioning the ethics of conducting randomized controlled trials in the face of what they consider to be overwhelmingly convincing surrogate evidence that proton therapy is superior to conventional x-ray therapy in terms of dose and volume of normal tissue irradiated. They consider blind the insistence on level I evidence by many in the radiation and broader oncology community, asserting that it is not possible to view the two modalities with equipoise. I would suggest one main response to this argument. The traditional use of the term “equipoise” in suggesting a clinical trial has been from the standpoint of the treating physician, for whom it is uncertain which trial arm will prove to be superior. I believe, in general, that this term should be redefined from the perspective of the ultimate autonomous being in charge of patient care: the patient himself. However, because a cancer diagnosis puts the patient under duress for his life, the next most interested party is composed of those involved by virtue of paying the bill. Today, the patient in particular and the public at large clearly believe that financial concerns cloud the judgment of the physician and hospital, both of whom certainly stand to benefit from increased use of new and expensive technologies. Recently, there have been several articles in the mainstream media attesting to this fact. Although the physician may observe that patients will knock down the door to obtain access to the latest technology, he does not as immediately hear the grumbling distrust of the taxpayers and policyholders who actually pay the costs and who do not directly live under the duress of the cancer diagnosis. Most radiation oncologists agree that certain pediatric cancers, for example, represent an area where little expense should be spared, and where the patients stand to benefit greatly from proton therapy over x-rays. However, the broad availability of protons will inevitably lead to their use in less clear circumstances, such as in prostate cancer, where another relatively recent addition to our arsenal has already significantly reduced bladder and rectal toxicity. Powering studies in pediatric tumors to prove the decreased clinical incidence of adverse effects will surely not present a challenge, once one accepts the assertion that public perception finds neither physician nor patient disinterested enough to even consider equipoise. Conversely, powering the prostate study to prove that the added expense of protons will result in a statistically significant decrease in clinically relevant late adverse reactions will be appropriately difficult. It is incumbent upon all of us to insist on level I evidence to justify changes in patient care. We must have the courage to generate highquality, disinterested, and unbiased data. This is especially true in the current era, when changes in practice result in higher reimbursement rates, and when the United States taxpayer is increasingly held hostage to spiraling medical costs. We must broaden our perspective to realize that we are not the only ones whom we must convince with evidence. As long as they entrust us with their lives and with their pocketbooks, the public has a right to hold us to our own highest standards. Only in this way do we hope to retain the trust and respect of the patient, the public, and each other.