InFocus: Rare Disease Clinical Development in Hong Kong
2026
InFocus: Rare Disease Clinical Development in Hong Kong explores how the city is positioning itself as a globally relevant centre for orphan drug development, drawing on strong clinical infrastructure, accelerated regulatory pathways, and a growing population genomic database. With rare disease therapies now accounting for 35 percent of the global R&D pipeline and over 50 percent of FDA approvals, the stakes for getting this right are high.
The report examines the regulatory reforms driving this ambition, including the “1+” fast-track registration mechanism, the planned establishment of the Hong Kong Centre for Medical Products Regulation (CMPR), and the Named Patient and Compassionate Use programmes that are already getting innovative therapies to patients ahead of formal approval. It also spotlights the Hong Kong Children’s Hospital and the Hong Kong Genome Project, which together are building the diagnostic and data foundations that rare disease research demands.
Perhaps most significant is Hong Kong’s push to integrate with the 87-million-strong Greater Bay Area. Through initiatives such as the newly established GBA International Clinical Trial Institute and an ongoing NMPA pilot allowing designated mainland hospitals to use Hong Kong-registered drugs, the city is working to combine world-class clinical standards with the patient volumes that make rare disease trials viable at scale.
InFocus: Rare Disease Clinical Development in Hong Kong explores how the city is positioning itself as a globally relevant centre for orphan drug development, drawing on strong clinical infrastructure, accelerated regulatory pathways, and a growing population genomic database. With rare disease therapies now accounting for 35 percent of the global R&D pipeline and over 50 percent of FDA approvals, the stakes for getting this right are high.
The report examines the regulatory reforms driving this ambition, including the “1+” fast-track registration mechanism, the planned establishment of the Hong Kong Centre for Medical Products Regulation (CMPR), and the Named Patient and Compassionate Use programmes that are already getting innovative therapies to patients ahead of formal approval. It also spotlights the Hong Kong Children’s Hospital and the Hong Kong Genome Project, which together are building the diagnostic and data foundations that rare disease research demands.
Perhaps most significant is Hong Kong’s push to integrate with the 87-million-strong Greater Bay Area. Through initiatives such as the newly established GBA International Clinical Trial Institute and an ongoing NMPA pilot allowing designated mainland hospitals to use Hong Kong-registered drugs, the city is working to combine world-class clinical standards with the patient volumes that make rare disease trials viable at scale.
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