Sickle Cell Disease (SCD) is the most common inherited blood disorder in the United States and affects millions of people worldwide. Although treatment options have remained limited, multiple new drug therapies are currently in development and rapid advancement in gene therapy for SCD holds the promise of a cure. The timing is ideal to determine how best to assess the impact of these new therapies and create alignment across experts and stakeholders on which study outcomes are most important.

The goal of this project is to reach agreement on a core set of outcomes to be used in clinical trials of new therapies for SCD. We will bring together individuals with SCD and their family members, clinicians who care for SCD patients, representatives from companies developing new SCD therapies, regulators (e.g. FDA), healthcare payers (e.g. private health plans and Medicaid), and other stakeholders and use a structured consensus process to reach agreement on a core outcome set for SCD clinical trials.