Market Access Challenges for Gene Therapies in Inherited Blood Disorders: A Retrospective Evaluation of HTA Outcomes for Sickle Cell Disease and Thalassemia

OBJECTIVES: To analyze Health Technology Assessment (HTA) outcomes for Sickle Cell Disease (SCD) and thalassemia gene therapies from 2019 onwards, understand disparities in acceptance rates, and identify key factors contributing to positive or negative decisions.

METHODS: Data extracted from the NAVLIN HTA Database, focusing on HTA decisions regarding gene therapies for SCD and thalassemia products made from 2019 onwards. Recommendations categorized as positive or negative. For negative decisions, appraisal documents will be reviewed to identify underlying reasons. Geographic variations and their impact on HTA outcomes will also be examined.

RESULTS: Significant disparities in HTA outcomes for SCD and thalassemia across different regions. Factors influencing negative decisions often include high treatment costs, insufficient long-term efficacy data, and regional variations in disease prevalence and healthcare infrastructure.

CONCLUSIONS: This analysis highlights the complexities and regional disparities in market access for gene therapies targeting SCD and thalassemia. Understanding these factors can inform strategies to improve HTA outcomes and facilitate access to innovative treatments for patients with inherited blood disorders.