The approval process for advanced therapies such as cell and gene therapy is expected to be optimized

The approval process for advanced therapies such as cell and gene therapy is expected to be optimized

In recent years, with the rapid development of biomedical technology, advanced therapies such as cell and gene therapy (CGT) have become hot topics in the global medical field. Data from the entire network over the past 10 days show that regulators in various countries are actively optimizing the approval process to accelerate the launch of these innovative therapies. This article will combine structured data to analyze current hot topics and policy trends.

1. A global hot topic inventory (next to 10 days)

2. Key progress in the optimization of approval process

1.US FDA News: The FDA recently announced that it will expand the scope of "Advanced Therapy in Regenerative Medicine (RMAT)" recognition, allowing more early clinical data to support accelerated approval. According to statistics, 7 CGT products have been approved through the RMAT channel in 2023, an increase of 40% over 2022.

2.China's policy breakthrough: The "Technical Guiding Principles for Clinical Research and Development of Gene Therapy Products (Draft for Comments)" released by NMPA for the first time:

• "Basket Test" design allowed
• Accept alternative endpoint indicators
• Establish a fast track for breakthrough therapeutic drug procedures

3.Highlights of the new EU regulations: EMA launches an upgraded version of the "PRIME Program", shortening the gene therapy review cycle from 210 days to 150 days, and establishing a rapid response mechanism for expert consultation.

3. Industry impact data analysis

4. Expert views and industry outlook

1.Technology-driven approval reform"AI-assisted clinical trial data analysis allows regulators to accept more flexible evidence of efficacy," said Dr. Smith, Stanford University Medical Center.

2.Balancing safety and speed: Experts from the China Pharmaceutical Review Center emphasized: "Optimization is not to lower standards, but to achieve full-life cycle supervision through innovative mechanisms such as 'dynamic GMP certification'."

3.Future trend forecast:

• There may be the first "real-time approval" case in 2024
• Asia-Pacific will become a test field for approval reform
• General cell therapy product approval standards will be formulated separately

5. Corporate response suggestions

1. Establish an early communication mechanism and seize pre-evaluation opportunities
2. Invest in real-world evidence (RWE) generation ability
3. Pay attention to regional differences, such as China's special requirements for ethical review of gene editing

With the increasing global regulatory synergy, the average time to market for cell and gene therapy products is expected to be shortened to 8-10 months by 2025, which will significantly improve the accessibility of treatment for patients with rare diseases and cancer. The industry needs to continue to pay attention to policy changes and grasp the strategic development window.