China’s Center for Drug Evaluation (CDE) has officially included Eliglustat Tartrate Capsules in its patient-centric rare disease drug development pilot program, known as the “Care Program.” The move highlights China’s commitment to accelerating treatments for rare diseases, starting with Gaucher disease.

Policy Breakdown

• Drug & Indication: Eglystat Tartrate Capsules, developed by Beijing Kailai Tiancheng Medical Technology Co., Ltd., target Type I Gaucher disease, a rare genetic disorder.
• Development Stage: The drug is in the post-marketing (E stage) phase, focusing on patient experience data collection and preference analysis.
• Patient-Centric Approach: The pilot emphasizes direct patient input through surveys and data analysis to tailor future treatments.

Market Implications
The inclusion in the Care Program signals regulatory prioritization, potentially shortening time-to-market for rare disease therapies. Analysts at China Healthcare Innovation Institute estimate a 20–30% reduction in development timelines for drugs in the program.

Industry Impact
Pharmaceutical companies are encouraged to engage with patient communities early in the development cycle, aligning with China’s shift toward patient-centered regulatory frameworks.

Looking Ahead
The Care Program is expected to expand to include additional rare disease treatments, with a focus on leveraging real-world data for regulatory decisions. Stakeholders should monitor how patient feedback shapes clinical trial designs and approval processes.-China Health Reform Pulse

Policy Source: https://www.cde.org.cn/main/news/viewInfoCommon/a1b306badd16f4f4e16360e3ce4522ba