Macau first: a scalable entry route for paediatric orphan drugs into Greater China
Written by Chris Zhou, vice president of partnerships, US and EU, ACA Pharma
Estimated reading time: 7 minutes
Access shouldn’t hinge on multi-year approvals when safety and efficacy are already established. By starting in Macau (on China’s south coast), manufacturers can deliver paediatric orphan therapies within months, de-risk compliance, and validate demand. This piece explains the model, the safeguards, and how it scales into Greater China
China alone has ~20 million people living with rare diseases, most of which present in childhood; ~80% are genetic and ~95% lack approved treatments. For families, the “diagnosis-to-therapy” journey is slow—diagnosis often takes ~4–5 years1—and conventional market entry can add more years due to local filings, pricing, and launch sequencing. For children, delay isn’t neutral; it can cost growth and function. Early, controlled access—under clear pharmacovigilance (PV)/quality assurance (QA) guardrails—bridges the time gap while originators prepare full registrations.
What slows companies down today
Most teams default to “registration-first” in Mainland China, then stall in a maze of country rules, heavy dossier admin, and PV/QA lift—all before a single patient sees a drug. Lean teams also absorb import permits, customs, cold-chain, and site-by-site onboarding, while fragmented demand, pricing/reimbursement variance, foreign exchange (FX), and compliance risks drain bandwidth. Net result: months to years lost to sequencing and setup, not patient care. A Macau-first lane cuts through this while preserving safety and quality.
Macau Fast-Track: months, not years
We start by leveraging your existing FDA/EMA/MHRA2 dossier as is—no need to update. We file in Macau and typically secure local approval in ~30–90 days. From there, we bridge into Mainland via the Greater Bay Area (GBA): a provincial-level filing that enables cash-pay supply to 45 GBA hospitals right after Macau approval. For orphan or clinically urgent products, we can extend access to top rare-disease departments in Beijing, Shanghai, Hainan, Tianjin, and Chongqing; for paediatric orphan drugs, to the top 44 pediatric centers nationwide.
Net effect: real patients on therapy in months rather than ~five years for a full, trial-heavy route. The Macau list is short (sub-10 items) and reuses what you already have, with less admin friction (e.g., no embassy notarisation). PV/QA, import, cold chain, and site onboarding are handled end-to-end, so originators preserve safety and quality while proving demand and building a scalable bridge into Greater China.
Group NPP hospital procurement
For originators who prefer no registration at all, Group NPP (named-patient procurement) provides a clean, supply-only route focused on paediatric orphan drugs. We activate a national network of the 44 top pediatric centers—which collectively see ~95% of paediatric rare-disease patients—so hospitals apply site-by-site import permits and we negotiate terms on a group basis.
Paperwork is minimal: a simple purchase agreement plus confidentiality, with no exclusivity and no distribution contract required. Because rare and ultra-rare cases concentrate at these centers, coverage is efficient and predictable; PV/QA, cold chain, and traceability sit on our standard platform. Typical timing from greenlight to first deliveries is ~three months, giving families access now while companies preserve optionality for future filings.
Case snapshot: four-year-old boy with DMD
A four-year-old boy with Duchenne muscular dystrophy (DMD) presented with biomarkers consistent with muscle damage, MRI (magnetic resonance imaging), evidence of progressive muscle loss and early cardiac strain.
Leveraging Macau Fast-Track, we registered deflazacort3 (marketed as Emflaza in the U.S.) and imported product without local clinical trials or NMPA approval in under 62 working days. Therapy initiated at 6 mg/day under hospital supervision with structured follow-up and PV/QA in place.
Crucially, the programme was framed not as an expensive “foreign import” but as a targeted deregulation to fast-track clinically needed, otherwise unavailable medicine for children—earning national media coverage4. This became the first paediatric orphan access via this route into China, demonstrating that months—not years—are achievable when safety and quality are already established and execution is disciplined.
Commercial model that de-risks originators
We are the buyer. We typically take title ex-works at your warehouse and handle export from the U.S., then import through our licensed entities across Greater China and Southeast Asia (including being the local MAH (marketing authorisation holder) where applicable). We manage end-to-end execution—customs, warehousing, distribution, marketing, promotion, and sales—and collect from the supply price. There’s no fixed-asset investment or field build-out for the originator; your obligation is manufacturing COGS (cost of goods sold). Contracting is straightforward with a single U.S. counterparty, and we typically prepay in USD, eliminating FX and capital-control risk for you.
When this pathway fits—and when it doesn’t
China delivers low drug prices and broad domestic coverage—but where therapies are absent, regulators have cleared lanes to fast-track truly innovative or high-need medicines, especially paediatric orphan drugs.
Best fit: products with established global safety/efficacy that fill a clear gap (rare disease, pediatric, etc.). For Greater Bay Area entry, the threshold is unmet need—even non-orphan products can qualify if the indication or clinical use meaningfully differs from available options.
Poor fit: commodities with multiple domestic generics or me-too products offering no clinical step-up; those won’t fast-track and shouldn’t pursue this route.
From early access to durable market entry
Early access isn’t a side path—it’s the smarter first lane. You generate revenue from day one, test real demand and pricing with the right centers, and iterate ops before committing to a full-scale launch. By “hacking” the NMPA (National Medical Products Administration) sequence—leveraging FDA/EMA/MHRA approvals to secure a rapid Macau decision, then bridging into the Greater Bay Area—you turn years of waiting into months of learning. In parallel, we can run filings and access routes in Hong Kong, Singapore, and Hainan’s pilot zone, so market validation and regulatory progress move together. The result: a de-risked, data-backed entry that builds toward durable, Mainland-wide adoption instead of a single, high-burn launch bet.
About ACA Pharma
ACA Pharma runs the fastest compliant route into China for paediatric orphan and other high-need therapies. We leverage Macau Fast-Track to bridge into the Greater Bay Area in months, while running parallel access in Hong Kong and Singapore. With state-owned distributor ties, a 100% execution success rate, and a ~80% market share for Macau registrations, we deliver both speed and certainty—then take you the rest of the way across Mainland China with end-to-end commercialization. We also operate across Southeast Asia, giving originators one U.S. counterparty and a single, proven path from first shipment to scale.
Learn more atwww.acapharma.netand on Linkedinhttps://www.linkedin.com/company/acapharma/
Chris Zhou is vice president of partnerships at ACA Pharma, which specialises in fast-track registrations, end-to-end commercialisation and early access programmes across China and SE Asia. He previously founded several businesses in technology and real estate. Earlier in his career he was a vice president in structured finance at Deutsche Bank and served in multiple groups within the investment banking division at J.P. Morgan. He holds a B.A. in Economics from the University of China
Connect with Chris
References
[1] https://english.nmpa.gov.cn/2024-07/19/c_1006358.htm; https://pmc.ncbi.nlm.nih.gov/articles/PMC10463573/; https://www.thelancet.com/journals/langlo/article/PIIS2214-109X%2824%2900056-1/fulltext
[2] FDA: Food and Drug Administration, EMA: European Medicines Agency, MHRA: The Medicines and Healthcare products Regulatory Agency
[3] https://acapharma.net/deflazacort-emflaza-launch-in-china-via-macau-fast-track-pathway/
[4] https://tv.cctv.com/2024/05/13/VIDEafTKXR6hoUWvBJvwHQ2w240513.shtml
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