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Published on 2024-03-21 17:42:00
CRISPR technology has moved beyond being just a promising concept to becoming a practical reality. However, in order for this powerful gene-editing tool to achieve its full potential, experts and advocates stress the importance of connecting patients suffering from severe diseases with the therapies that could potentially help them.
During a panel discussion at the STAT Breakthrough Summit East in New York, experts highlighted the significance of lab-developed tests (LDTs) in facilitating this connection. These tests, conducted by certified labs using clinical samples, play a crucial role in guiding healthcare decisions and have the capability to identify genetic diseases.
Julia Vitarello, the founder and CEO of Mila’s Miracle Foundation, which is dedicated to finding a cure for Batten disease, emphasized that the technology to identify patients and customize personalized treatments already exists. However, she pointed out the lack of infrastructure and processes needed to connect millions of children suffering from terminal illnesses with the available technologies.
Access to LDTs remains a significant challenge, according to Lee Fleischer, a former director at the Centers for Medicare and Medicaid Services. He raised concerns about ensuring that the right labs are certified to perform this essential work in the future.
The landscape of lab-developed tests has evolved significantly since the FDA began subjecting medical devices to pre-market review in 1976. These tests, which are created, produced, and used within a single laboratory, now inform approximately 70% of healthcare decisions, as reported by the Centers for Disease Control and Prevention. In response to concerns about the accuracy of these tests, the FDA proposed a new rule in September 2023 to enhance oversight of LDTs. While some patient advocates welcomed the move, labs warned that it could hinder their ability to offer LDTs efficiently. The rule requires labs to submit tests designed to screen for rare genetic diseases to the FDA.
Fyodor Urnov, a professor at the University of California, Berkeley, and director of the Danaher-IGI Beacon for CRISPR Cures, shared that Berkeley’s Innovative Genomics Institute is actively working on creating a network of LDTs that can help connect patients with available CRISPR therapies. He stressed the importance of spreading the availability of these tests globally to ensure the future success of CRISPR technology.
Urnov emphasized the necessity of diagnosing patients and following up on their progress rigorously to gain recognition from CMS for the field to have a sustainable future.
In conclusion, connecting patients suffering from devastating diseases with potential therapies through lab-developed tests is crucial for realizing the full potential of CRISPR technology. By addressing the challenges related to access and oversight of LDTs, researchers and advocates can pave the way for more impactful and personalized treatments for patients in need.
