Title page

Contents

Summary 1

What Are Gene Therapies? 2

Sickle Cell Disease in the United States 2

Gene Therapies for Treating Sickle Cell Disease 4

Policy Approaches to Increase Gene Therapy Treatment 6

How CBO Would Estimate the Budgetary Effects of Increased Use of Gene Therapy Treatment for Sickle Cell Disease 7

Figure 1. Number of SCD-Related Emergency Room Visits, by Type and Payer, 2021 3

Boxes

Box 1. Evaluating the Timing of Federal Budgetary Effects of Gene Therapies Compared With Other Health Interventions 8