Gene editing therapies like CRISPR face regulatory hurdles due to novel risks: off-target edits, immunogenicity, long-term germline effects, and manufacturing scalability. Dose determination is complex (e.g., vector genomes vs. editing efficiency), with undefined potency assays linking CQAs to outcomes. Small populations complicate trial design, requiring adaptive endpoints and real-world evidence.
Global variances: FDA/EMA emphasize RMAT/PRIME for accelerated access, but differing GMP standards, environmental assessments, and follow-up durations (15+ years) impede harmonization. High costs ($2M+/patient) and vector shortages strain infrastructure. Multinational trials encounter import/export barriers.
Solutions: reliance models (e.g., EMA-FDA work-sharing), innovative trials (basket designs), post-approval surveillance. Progress: 10+ approvals since 2017.
References: PMC (2021), PMC (2023). Word count: 210.
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