Treatment options in multiple myeloma (MM) are increasing with the introduction of complex multi-novel-agent-based regimens investigated in randomized clinical trials. However, application in the real-world setting, including feasibility of and adherence to these regimens, may be limited due to varying patient-, treatment-, and disease-related factors. Furthermore, approximately 40% of real-world MM patients do not meet the criteria for phase 3 studies on which approvals are based, resulting in a lack of representative phase 3 data for these patients. Therefore, treatment decisions must be tailored based on additional considerations beyond clinical trial efficacy and safety, such as treatment feasibility (including frequency of clinic/hospital attendance), tolerability, effects on quality of life (QoL), and impact of comorbidities. There are multiple factors of importance to real-world MM patients, including disease symptoms, treatment burden and toxicities, ability to participate in daily activities, financial burden, access to treatment and treatment centers, and convenience of treatment. All of these factors are drivers of QoL and treatment satisfaction/compliance. Importantly, given the heterogeneity of MM, individual patients may have different perspectives regarding the most relevant considerations and goals of their treatment. Patient perspectives/goals may also change as they move through their treatment course. Thus, the 'efficacy' of treatment means different things to different patients, and treatment decision-making in the context of personalized medicine must be guided by an individual's composite definition of what constitutes the best treatment choice. This review summarizes the various factors of importance and practical issues that must be considered when determining real-world treatment choices. It assesses the current instruments, methodologies, and recent initiatives for analyzing the MM patient experience. Finally, it suggests options for enhancing data collection on patients and treatments to provide a more holistic definition of the effectiveness of a regimen in the real-world setting.

Applied Cancer Research and Drug Discovery Translational Genomics Research Institute City of Hope Cancer Center Phoenix AZ USA

Department of Haematology Oxford University Hospitals NHS Foundation Trust RDM Oxford University NIHR BRC Blood Theme Oxford UK

Department of Haematology The Royal Marsden Hospital and Division of Molecular Pathology The Institute of Cancer Research London UK

Department of Hemato Oncology University Hospital Ostrava and Faculty of Medicine University of Ostrava Ostrava Czech Republic

Department of Hematologic Oncology and Blood Disorders Levine Cancer Institute Charlotte NC USA

Department of Hematology Cancer Center Amsterdam Amsterdam University Medical Center VU University Amsterdam Amsterdam The Netherlands

Department of Hematology University Hospital of Salamanca IBSAL CIC IBMCC Salamanca Spain

Department of Lymphoma and Myeloma MD Anderson Cancer Center Houston TX USA

Department of Medicine Division of Blood and Marrow Transplantation Moores Cancer Center University of California San Diego La Jolla CA USA

Department of Oncology Hematology and Bone Marrow Transplantation with Section of Pneumology University Medical Center Hamburg Eppendorf Hamburg Germany

Leeds Cancer Centre Leeds Teaching Hospitals Trust Leeds UK

Millennium Pharmaceuticals Inc a wholly owned subsidiary of Takeda Pharmaceutical Company Limited Cambridge MA USA

Myeloma Unit Division of Hematology University of Torino Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino Torino Italy

Plasma Cell Dyscrasias Unit Department of Clinical Therapeutics National and Kapodistrian University of Athens School of Medicine Athens Greece

Tisch Cancer Institute Icahn School of Medicine at Mount Sinai New York NY USA

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