Recently, the U.S. Food and Drug Administration (FDA), has begun to support the reporting and use of patient-reported outcome measures (PROMs) of symptoms as secondary end points in clinical trials for oncology treatment. These outcomes measures are likely to become increasingly important for oncology patients as they will hopefully become part of the drug label.
There have been a number of patient-reported outcome questionnaires developed. These measure patient symptoms, treatment adherence, function and health-related quality of life, and satisfaction with treatment regimens. These measures have the potential of becoming an important part of daily clinical practice. They can encourage better communication between clinicians and patients and even guide adjustments in treatment decisions.
So, what are these Patient-reported outcomes (PROs)? They are simply subjective status reports of the patients participating in clinical trials that help describe patient’s current disease- or drug-related condition or recent experiences. Items like patient pain scale reports and health-related quality of life (HRQOL) self-reports about the physical, emotional, and social impact of a disease or therapy are examples of PROs that many of us already know. PROs often correlate with objective test outcomes, however they do offer insight into a patient’s status that are not easily measured with objective clinical tests or medical imaging
Many cancer clinical trials already include PROs as secondary outcome measures; however it is rare that the FDA allows these symptom outcomes to be a part of drug labeling! In non-oncology drugs almost 25% of FDA approved treatments have label information about medication effects on patient symptoms or function.
“That disparity is surprising, given how common symptoms and functional impairment are in patients with cancer and how toxic oncology drugs can be,” reports Ethan Basch, MD, of the Cancer Outcomes Research Program at the University of North Carolina at Chapel Hill, NC. “As an oncologist, when I sit with patients to discuss starting a new chemotherapy regimen, their first questions are often, ‘how will it make me feel?’,” Dr. Basch wrote in a recent commentary for The New England Journal of Medicine.4 “Regrettably, this information is generally missing from US drug labels and from published reports of clinical trials…of cancer drugs.”
The inclusion of this information is important. Given the number of newly approved oncology treatments (in prostate cancer these include drugs such as Provenge, Zometa, Xtandi, Jevtana etc.) the issue of side effects becomes much more important in making good clinical decisions. Patients want to be able to weight the positive possibilities (increase in overall survival) against the potential effects of the treatment. We, as survivors of any cancer have the right to participate in our clinical decisions, but these decisions must be fully informed. Knowing about the side effect experiences of people in the clinical trials is every bit as important to us as knowing survival advantages.
Joel T Nowak, M.A., M.S.W.