Evidence Generation Plan for Lovotibeglogene Autotemcel (lovo-cel) First Cell-based Gene Therapy for Sickle Cell Anemia

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Publication Date

7-24-2024

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Presentation: 19:02

Abstract

Sickle cell anemia (SCA) is a significant blood disorder that affects quality of life and can lead to a reduced life span. Individuals with SCA endure severe symptoms and complications, highlighting the urgent need for innovative therapies that promise improved outcomes. SCA affects approximately 20 million people globally, with around 100,000 cases in the United States alone. Recently, the FDA approved lovotibeglogene autotemcel, a promising gene therapy with potential curative benefits. The specific aim was to develop a strategic plan for generating evidence encompassing pre-launch, peri-launch, and post-launch activities for lovotibeglogene autotemcel. A comprehensive literature review was conducted using PubMed and SCOPUS, focusing on the latest research from 2022 to 2024 related to SCA and gene therapy. This review identified six key studies that shed light on the current gaps and competitive landscape for SCA treatments. These studies underscore that while gene therapies are a recent advancement in the SCA field, long-term follow-up is crucial to validate their safety and effectiveness. Notably, there is one primary competitor with a similar indication and approval date. For a successful product launch, the pre-launch phase should involve creating a budget impact model and preparing a pre-approval AMCP dossier. During the peri-launch and launch phases, it would be essential to assess budget impact from a payer perspective, considering both treatment-related and disease-related costs over a one-year horizon. In the post-launch phase, conducting real-world evidence studies would be important to demonstrate the therapy's effectiveness beyond clinical trials. Lovotibeglogene autotemcel offers a unique genetic approach to addressing the unmet needs in SCA treatment. To highlight its value, targeted strategies and further studies are warranted to address evidence gaps and meet business needs from a payer perspective.

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English

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