Benchmarking Gene Therapies for Sickle Cell Disease in Saudi Arabia Using Mendelow’ s Stakeholders Matrix

Abstract

Aim and Methods: This paper explores the potential of GTs for SCD in KSA, addressing their applicability, accessibility, and comparison with traditional treatments. It aims to benchmark GTs in KSA by analysing advancements in R&D, developing equitable distribution strategies, and comparing real-world data. A scoping review was conducted using major academic databases, with 29 relevant articles selected to assess the effectiveness and implementation of SCD GTs in KSA and globally. Findings: Traditional treatments for SCD have long relied on blood transfusions and pharmacologic options like HU, approved by the FDA in 1998. While these treatments have been essential, their limitations have led to the development of new therapies, including GTs. GT offers a curative approach by targeting the genetic root of SCD. Recently, FDA-approved gene therapies, such as Casgevy and Lyfgenia, have marked a significant advancement. These therapies use innovative genome editing techniques to replace defective cells. Although promising, the adoption of GT is tempered by concerns about cost, accessibility, and long-term efficacy compared to traditional treatments, which remain widely used due to their established effectiveness and broader availability. Practical Implications: GT has the potential to revolutionize SCD management, but challenges like accessibility, affordability, and infrastructure readiness, particularly in Saudi Arabia, require comprehensive policy reforms and supportive systems for successful implementation.