China's groundbreaking strides in rare disease management are turning heads globally, and AstraZeneca's Marc Dunoyer is among those applauding the nation's efforts. But here's where it gets controversial: can China's ambitious healthcare reforms truly bridge the gap between innovation and accessibility for the millions affected by rare diseases?
As the Chief Strategy Officer of AstraZeneca and CEO of its rare disease division, Alexion, Dunoyer highlights China's "remarkable progress" in addressing rare diseases, positioning the country as a rising star in global biomedical innovation. In a recent interview with China Daily, he emphasized, "China's focus on health equity, innovation, and child welfare naturally extends to those living with rare conditions." And this is the part most people miss: China's holistic approach isn't just about treatment—it's about building an entire ecosystem that spans policy, diagnosis, therapy, and insurance coverage.
Over the past few years, China has quietly constructed a robust rare disease framework, aligning with the Healthy China 2030 strategy. At the 2025 China Conference on Rare Diseases in Beijing, startling figures emerged: the national rare disease catalog now includes 207 conditions, with nearly 100 therapies covered by the national medical insurance system. The collaborative diagnosis and treatment network has expanded to 419 designated hospitals, and over 620 hospitals have joined the national rare disease reporting platform. Here’s the kicker: In 2024, China's medical insurance fund allocated a staggering 8.6 billion yuan ($1.2 billion) to rare disease drug reimbursements, accounting for 7.7% of total pharmaceutical spending.
Dunoyer points out that this isn't just about healthcare—it's about preventing families from spiraling into poverty due to medical expenses. "Expanding access to diagnosis and treatment also fuels innovation in cutting-edge fields like gene and cell therapy," he explains. But here's the catch: While China's progress is undeniable, affordability remains a stubborn challenge. Dunoyer notes that families affected by rare diseases face financial burdens up to six times greater than those managing chronic conditions. "It's unrealistic to expect patients to bear these costs alone," he asserts.
What’s truly fascinating is China's potential to pioneer its own solutions. Dunoyer believes the country is uniquely positioned to develop innovative financing mechanisms and sustainable payment models, ensuring long-term patient access while fostering innovation. AstraZeneca, one of the first multinational pharmaceutical giants to invest heavily in China's healthcare sector, has already launched three breakthrough therapies across five rare disease areas, two of which are now on the national reimbursement drug list.
The company’s commitment doesn’t stop there. With over 10 global Phase III clinical trials underway in China and support for 139 Centers of Excellence nationwide—including a pilot program for neurofibromatosis type 1 in Qingdao—AstraZeneca is doubling down on its $2.5 billion investment in a new global strategic R&D center in Beijing. "Chinese innovation is no longer just a local phenomenon—it's shaping global medicine," Dunoyer remarks.
Yet, despite these leaps forward, Dunoyer cautions there’s still a long road ahead. Globally, therapies exist for only 5-10% of known rare diseases, leaving 90% without solutions. Here’s a thought-provoking question: Can China’s focus on equity, innovation, and pediatric health not only transform lives within its borders but also catalyze the next wave of medical breakthroughs worldwide?
China’s journey in rare disease management is more than a national endeavor—it’s a blueprint for global healthcare transformation. But as we celebrate its achievements, let’s not forget the challenges that remain. What do you think? Is China’s approach to rare diseases a model for the world, or are there critical gaps that still need addressing? Share your thoughts in the comments below.
