Measuring symptoms as a critical component of drug development and evaluation in hematological diseases

Abstract

With the rapid development of new therapies for patients with hematological malignancies, there is an increasing need for patient report of symptom status during all phases of drug testing. The patient's perspective on new treatments reflects treatment tolerability as well as symptom benefit, and may assist patients and clinicians in choosing treatments. Inclusion of patient-reported outcomes, more common in solid-tumor than hematological trials, provides early information about symptoms to guide decisions about appropriate dosing and supportive care needs. We provide a historical overview of the use of patient-reported outcomes and symptom assessment in solid-tumor and hematological drug development, and offer recommendations about methodological issues in the monitoring of symptoms in the drug development process in hematological clinical trials.

Keywords: European Medicines Agency; US FDA; cancer clinical trials; drug development; patient-reported outcomes; recommendations; symptom assessment; symptom benefit; symptom burden.

Figure 1. Model of symptom burden as part of health-related quality of life

Within the HRQOL spectrum, symptoms are the component most closely related to the disease and treatment process (green oval). Symptom burden includes both symptom status and functional status (purple oval). HRQQL: Health-related quality of life. This figure can be viewed in full color at: www.future-science.com/doi/full/10.4155/CLI.13.108 With permission from © Cleeland CS (2006) and adapted from Wilson and Cleary (1995).