• The United States (US) Food and Drug Administration (FDA) convened meetings on patient-focused drug development to identify the symptoms/effects of sickle cell disease (SCD) impacting daily life that are most important to patients.
• Drug developers are now expected to present direct evidence of the benefits of SCD therapies on these symptoms/effects. Such evidence has to be generated by measuring standardised patient reported outcomes (PROs).
• The majority of research reports on the generic instruments assessing quality of life (QoL) in SCD patients. However, there is a paucity of data on the available SCD-specific QoL/PRO instruments.
• The present review aimed to identify and summarise published data on the available SCD-specific PRO instruments.
Authors: Jatin Gupta; Ankit Ghildiyal