Quality of Life, Clinical Trials, and Clinical Practice

Quality of Life, Clinical Trials, and Clinical Practice

Nagi S. El Saghir, MD, FACP, FASCO

@NagiSaghir
Oct 30, 2018

I had the honor and pleasure to co-chair the European Society for Medical Oncology (ESMO) Metastatic Breast Cancer Proffered Paper Session with Dr. Frédérique Penault Llorca at the ESMO 2018 Congress (#ESMO18) in Munich, Germany. We called that session historical because we included several abstracts that dealt with quality of life (QOL) aspects and patient reported outcome (PRO) measurements, as part of major recent clinical trials involving new drugs, CDK4/6 inhibitors and PARP inhibitors for patients with metastatic breast cancer. Dr. Vesna Bjelic-Radisic presented an update on the pioneering European Organization for the Treatment of Cancer (EORTC) quality of life questionnaires (EORTC-QLQ) for health related quality of life (HRQOL) assessment, and Dr. PA Fasching, Dr. Nadia Harbeck, and Dr. Hope Rugo (@HopeRugo) presented results of PROs on three major MONALEESA-3, MONALEESA-7, and EMBRACA clinical trials. Discussant Dr. Leslie Fallowfield put the presentations in perspective and expressed her hope that this session will bring QOL issues up to mainstream attention. She said that when QOL issues are mentioned, if they are at all, they are found at the bottom of a manuscript published in a major medical journal, or as a supplement, or as a follow-up poster presentation, or a publication in a lesser impact journal. This session promises a change.

Quantity of life gained is usually assessed by classical disease-free survival (DFS), progression-free survival (PFS), and overall survival (OS), and measured in years, months, and even weeks. Quality of life (QOL), on the other hand, relates broadly to general health, personal life, study, work, hobbies, leisure, family, friends and other general aspects. Health related quality of life (HRQOL) refers to effects of disease symptoms, treatment side effects on the patient’s physical health, as well as mental life, emotional life, and wellbeing. HRQOL is best measured from both the patient’s point of view, and that of the physician’s. The European School of Oncology (ESO)/ESMO guidelines for advanced breast cancer stated in 2011 that each treatment regimen (except anthracyclines) should be given until progression of disease or unacceptable toxicity, and that what is considered unacceptable should be defined together with the patient.1 

The term patient reported outcome (PRO) is introduced to make sure that the patient point of view regarding toxicity and effects on his/her life are well addressed. This is done by patient reported outcome measurement (PROM), done either via questionnaires or interviews.

The scientific community, medical journal editors, and regulatory authorities usually focus on measurements of survival, response rates, and adverse events when assessing new drugs. Recently, the medical community, including ASCO and ESMO, recommends the incorporation of quality of life measurements as part of the endpoints of clinical trials. Examples are EORTC-QLQ questionnaires, the National Institute of Health’s (NIH) Patient-Reported Outcomes Measurement Information System (PROMIS), Functional Assessment of Cancer Therapy-General (FACT-G), ESMO Magnitude of Clinical Benefit Scale, and ASCO framework for assessing the relative value of cancer therapies that have been compared in clinical trials that was recently updated in the Journal of Clinical Oncology. This framework defines value as a combination of clinical benefit, side effects, and improvement in patient symptoms or quality of life in the context of cost. ASCO hopes to make this updated framework the basis for a software tool that we can use to assist shared decision-making with our patients.

Patient QOL issues are always at heart for all clinical oncologists. QOL measurements should become a major aspect of new drug assessment, especially that statistically significant results may mean only a few months increase in quantity of life. PROM will help us decide whether statistically significant results are clinically meaningful. PROM should also be assessed in post-marketing to better assess the impact of new drugs, as well as various aspects of financial toxicity on both the quality of life of patients and on health systems worldwide.

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