1. Quality of Life (QOL) & Patient Reported Outcomes (PRO) Lori Minasian, MD Chief, Community Oncology and Prevention Trials Research Group, DCP, NCI, NIH, DHHS

2. What Is A Patient Reported Outcome? A Patient Reported Outcome (PRO) is a measurement of any aspect of a patient’s health status that comes directly from the patient, without interpretation of the patient’s responses by a clinician or research associate

3. What Is Quality of Life? Quality Of Life is an assessment by a person of all aspects of life Health Related QOL (HRQL) is an assessment of how illness and its treatment affect a person’s well being (≥ 3 domains: physical, mental, and social functioning, etc).

4. PRO ≠ QOL ≠ HRQL Evaluation of impact of illness or treatment on physical, emotional, & social aspects of QOL Evaluation of all aspects of life Anything reported by the patient Quality of Life QOL Patient Reported Outcomes PROs Health-Related Quality of Life HRQL

5. Wilson and Cleary’s HRQOL Conceptual Model Biological and Physiological Variables Overall Quality of Life General Health Perceptions Symptoms Status Functional Status Characteristics of the Individual Characteristics of the Environment

6. Mayo Clinic Example: What is Your Overall Quality of Life? Hospice patients 7.6 Advanced cancer patients 7.2 Recovering surgical patients 6.6 Healthy volunteers 8.2 Medical students 4.4

7. Why Measure QOL in Cancer Trials? To determine a winner if treatment outcomes equivalent To determine the effect of cancer or its treatment on other aspects of life (ripple effect). To obtain patients’ perspective of therapy and its impact. QOL assessment can provide powerful data on symptoms and toxicity. But, what is the relevant information to be obtained from QOL?

8. What Question Are You Asking? How Does that Information Help? Answers may differ with disease stage What’s applicable in the adjuvant setting may not be applicable in metastatic disease or prevention setting Questionnaires bring the patient’s perspective. Impact of therapy on ability to function Impact of toxicity (short or long term) Will that information help physicians & patients make better decisions regarding therapy? Clinical significance

9. Key Questions When Incorporating QOL/PRO Outcomes in Clinical Trials: What is the purpose of the QOL/PRO assessment? How will a QOL/PRO assessment add to the information from the trial? What are the key QOL or symptom specific issues identified by the investigators? Will existing QOL/PRO tools be appropriate for the trial?

10. “How will a QOL or PRO assessment add to the information from the trial?” GOG 179: Cisplatin with or without topotecan in advanced cervical cancer C+T = > PFS and OS with More Toxicity No significant QOL; stable over time Use of reliable assessment instrument longitudinally allows favoring combination chemo to single-agent in this population

11. “Will QOL information help us make better decisions regarding therapy?” GOG172: Pts who received higher dose IP therapy, compared to those with conventional dose IV therapy experienced better survival

12. Patient-Reported FACT-O Scores (1)

13. Patient-Reported FACT-O Scores (2)

14. “Is It Possible to Obtain Patient’s Perspective of therapy and its Impact?” QOL data useful in interpreting treatment implications and influencing decision-making Illustrates complex relationship between treatment efficacy and toxicity (e.g., 179) More precise estimation of toxicity (e.g., Ntx) Supports development of more acceptable treatment alternatives (e.g., 172)