Patients with sickle cell dis ease (SCD) have sig nifi cant impairment in their quality of life across the life span as a consequence of serious dis ease bur den with several SCD - related complications. A number of dis ease - modifying therapies
are currently available, yet long - term clinical benefits in real - world set tings remain unclear. Over the past few years,
a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including:
(1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (con sen sus
measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient - reported out comes assessment is highly recommended, and patient - reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations
for Interventional Trials guide lines. Patient - centered out comes research (PCOR) approaches and meaningful stakeholder
engagement through out the process have the potential to optimize the execution and success of clinical trials in SCD
with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient - centered research approaches and stakeholder engagement. A pro posed hematology stake holder - engagement frame work for clinical trials is also discussed.
Sherif M. Badawy
Disease Category: Blood disorders
Disease Name: Sickle Cell Disease
Age Range: Unknown
Sex: Either
Nature of Intervention:
- Overview of literature
- Literature review