Presentation (CTI)

OBJECTIVES: China's rare disease policies have expanded regulatory pathways, yet NMPA national registration does not fully capture functional availability through regional special-access programs. We quantified national-registration gaps, regional special-access availability, and FDA-to-NMPA approval lag for therapies mapped to China's 207 rare disease catalog conditions.
METHODS: We constructed a drug-indication/regulatory-record dataset linking FDA and NMPA dates and regional special-access evidence through 29 May 2026. The primary cohort comprised FDA approvals from 2001-2024; 2025-2026 approvals were described separately. Drug loss was operationalized as a national-registration gap: no NMPA national approval by cutoff. Functional availability required documented regional special-access evidence and was distinct from national registration. Kaplan-Meier analysis used FDA approval as time zero and NMPA approval as the event; unapproved records were right-censored, and China-first records coded as time-zero events. Lag decomposition used records with complete FDA/NMPA submission and approval dates.
RESULTS: Among 231 FDA-approved records, 121 (52.4%) had a national-registration gap: 110 with no observed NMPA submission and 11 pending approval. Of these, 11 had documented regional special-access availability and 11 were under NMPA review, leaving 99 with no national registration, regional pathway, or active review. KM-estimated median FDA-to-NMPA approval time was 3,462 days (9.5 years); cumulative NMPA approval was 21.9%, 33.0%, and 51.2% at 3, 5, and 10 years. Post-2016 records showed shorter median lag than 2001-2015 (2,925 vs 4,049 days; log-rank P=0.070). Among 110 records with complete dates, submission lag constituted most of mean approval lag (1,627/1,692 days), whereas review-time lag was smaller (65 days).
CONCLUSIONS: More than half of FDA-approved rare disease therapy records had a national-registration gap, but some were regionally accessible or under review; gaps therefore overstate complete unavailability. Approval lag was predominantly submission-side, supporting earlier China submission, global development inclusion, and targeted pathways for small-market rare disease therapies.