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Now is the time to make patient-reported outcomes mandatory component of clinical trials
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The need to demonstrate value for the escalating cost of health care would seem to be obvious, yet the metrics by which value is determined are not always standardized.
There is a burgeoning amount of data that supports the numerous ways in which patient-reported outcomes (PROs) are value-added metrics, including providing significantly more toxicity and symptom data and more accurate data for subjective symptoms than clinician or Common Terminology Criteria Adverse Events (CTCAE) reporting. In fact, the more subjective the symptom, the lower the clinician–patient agreement of ratings of presence, frequency and severity.
For these reasons, and for over 2 decades, the medical community has embraced the fact that the most valid measure of a patient’s pain is the patient’s self-report. Yet, there has been slow progress in requiring PRO metrics for other wholly or largely subjective symptoms (eg, peripheral neuropathy, cognitive dysfunction, fatigue) for clinical trials and clinical assessments.
In addition, PROs provide other essential information for the assessment of value of treatments such as independent prognostication of survival across cancer sites and issues related to patient adherence. For example, it has been the PRO data on tamoxifen and aromatase inhibitors that have informed our understanding of the high nonadherence rates of these otherwise highly efficacious therapies.
The need for standard inclusion of PRO measures becomes even more pressing as we move forward with clinical trials of precision medicines, many of which are oral and have multiple side effects. It is urgently important that we understand the full value of these costly treatments. Regardless of the effect size of clinical trial results, there is no value to a drug patients refuse to take.
In fact, it seems unethical to ask patients to participate in clinical trials and never ask them directly to rate, at minimum, their symptoms and possibly their quality of life. If there were a “Patient Declaration of Independence,” it may begin something like this: “When, in the course of human events, it becomes necessary for a human being to seek a clinical trial for the life-changing diagnosis of cancer, the Laws of Nature and of Nature’s God entitle them to a voice in the process.” It is PRO metrics that gives them that voice.
And now, with the development, validation and release of the NCI–sponsored PRO–CTCAE — the PRO companion to the CTCAE — there is no reason or excuse to exclude the patient voice and to use their experiences to inform value for care.
I wrote an editorial published in 2007 in Journal of Clinical Oncology titled, “Should patient-reported outcomes be mandatory for toxicity reporting in cancer clinical trials?” Then, as now, the resounding answer is yes and the time is immediately.
References:
Atherton PJ, et al. J Pain Symptom Manage. 2015;doi:10.1016/j.jpainsymman.2015.04.016.
Bruner DW. J Clin Oncol. 2007;doi:10.1200/JCO.2007.13.3330.
Fang F, et al. Int J Radiat Oncol Biol Phys. 2004;58:1394-1404.
Flores LT, et al. Gastrointest Cancer Res. 2012;5:119-124.
Jensen MP. J Pain. 2003;4:2-21.
Movsas B, et al. J Clin Oncol. 2009;doi:10.1200/JCO.2009.23.7420.
Roychowdhury D, et al. J Clin Oncol. 2003;doi:10.1200/JCO.2003.04.166.
For more information:
Deborah Watkins Bruner, RN, PhD, FAAN, is Robert W. Woodruff professor of nursing and director of faculty mentorship at Nell Hodgson Woodruff School of Nursing and associate director for mentorship, education and training at the Winship Cancer Institute of Emory University. She can be reached at deborah.w.bruner@emory.edu.
Disclosure: Bruner reports no relevant financial disclosures.