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Mr. Spock, Dr. Sheldon Cooper and the Rain Man (patient-reported outcomes)
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In this edition of HemOnc Today, we are focusing our cover story on the hot topic of value and outcomes.
Both ASCO and European Society for Medical Oncology have expended a great deal of time and energy trying to create numerical algorithms that provide data that allow oncologists to discuss with patients the benefits and drawbacks of various treatments in a detailed, comprehensible and accurate way.
As we have seen, this effort to balance the constructs of cost, price and time gained, as well as quality of life, reduction of tumor-related symptoms and toxicity of treatment, is really difficult. Anyone who has read the report from Lowell E. Schnipper, MD, and the ASCO Value in Cancer Care Task Force can appreciate that building a numerical algorithm around symptoms and side effects is not that hard, but expressing it in a way that can easily be evaluated and understood by all patients is much more challenging.
Steps forward
For decades, there has been an increasing trend to focus on patient perceptions of their reality in dealing with cancer and its innate or treatment-related consequences, and this clearly has been an important step forward. In past eons, patients were much more subjugated and subdued, and many clinicians were inclined to focus just on the disease process, considering the patient as somewhat of an annoying encumbrance, to be ignored as much as possible.
Fortunately, in our current era of greater enlightenment, we have moved away from that sad pattern, and the pendulum has swung way to the other end of its trajectory.
Decades ago, visionaries like Neil Aaronson, PhD, and Ian Tannock, MD, PhD, DSc, developed early versions of tools to measure patient perceptions and quality of life, and subsequent iterations have attempted to improve on their precision and reproducibility.
The NIH has developed its Patient Reported Outcomes Measurement Information System (PROMIS), which is focused on physical, mental and social well-being, and attempts to ensure comparability, reliability and validity, flexibility and inclusiveness.
The National Quality Forum, British National Institute for Health and Care Excellence, the Cochrane organization, FDA and others have all claimed a piece of the action and have developed official stances and modifiers of patient-reported outcomes (PROs). Even Wikipedia has a detailed section, noting that common PRO questionnaires are focused on symptoms and other aspects of well-being, functioning (disability), health status, general health perceptions, quality of life, health-related quality of life, and reports or ratings of health care.
There is a commonality of jargon and word salad of initials and acronyms, leading to a general consensus of what items should be measured to try to get a clearer definition of what matters to patients vis-à-vis their illnesses and the state of wellness.
Of rapidly increasing importance in the portrayal of a complete picture, in addition to the parameters listed above, we are beginning to understand broader issues, such as the impact of fiscal toxicity and the severe damage to family life and posterity occasioned by our financially chaotic health care morass.
When a weighting of the importance of each specific parameter to the patient is attached, the sum total can lead to the calculation of a quality-adjusted life-year. Thus, the more important parameters in the view of patients will have greater weight.
In recent years, other modifiers have been attached or inserted to try to make PROs even more robust. It is not rocket science to recognize that it is important to know and understand how patients feel about their situations, and that their perceptions of disease and treatment are crucial in fashioning management algorithms.
Long way to go
Mr. Spock from Star Trek; Dr. Sheldon Cooper, the famed physicist from Cal Tech in The Big Bang Theory, and the Rain Man, the autistic savant from the 1988 film of the same title, would be ideal subjects for reporting their responses to disease and its treatment. They would give unemotional and linear responses to questions, seeking only clarification to improve accuracy and detail.
By contrast, patients often do not give linear and unemotional answers to verbal or written questions. Part of the problem is that, in contrast to Lewis Carroll’s view, many patients don’t agree that “a word means what I want it to mean ... nothing more, nothing less.” Patients and family members often interpret phrases and connotations quite differently from the intent of their physicians, and their answers may not reflect the question in the mind of the investigator.
Further, it is well known that patients often try to be “good patients,” wanting to please the physician, or they fear that an honest confession of the level of symptoms or toxicity may lead to premature termination of treatment. Conversely, they may feel that an accurate description of tumor-related symptoms may lead to an escalation of therapy. Patients in pain or receiving large doses of analgesics may have inattention or clouding of consciousness, with a less clear focus on producing definitive PROs.
Finally, the tools themselves — due to the quest to increase the acquisition of detailed information — can become unduly long and unwieldy, and reporter fatigue may often contribute to reduced accuracy of response or a negative bias as the patient becomes irritated by the questionnaire itself.
Thus, while considering the importance of the patient and family in the attribution of value and structured outcomes, it is unwise to make the assumption that PROs are a simple, unfettered and linear reflection of truth.
We still have a long way to go before we really know how to measure and interpret PROs, and this index should not be given more weight than other objective measures.
That said, we should not forget that the dark period when physicians did not listen to patients should remain firmly embedded in the past.
Reference:
Schnipper LE, et al. J Clin Oncol. 2015;doi:10.1200/JCO.2015.61.6706.
For more information:
Derek Raghavan, MD, PhD, FACP, FRACP, FASCO, is HemOnc Today’s Chief Medical Editor for Oncology. He also is president of Levine Cancer Institute at Carolinas HealthCare System. He can be reached at derek.raghavan@carolinashealthcare.org.
Disclosure: Raghavan reports no relevant financial disclosures.