Rare diseases in focus at Sino-French Medical Forum in Beijing

Rare disease governance must go beyond laboratory breakthroughs, officials and experts said at the 2026 Sino-French Medical Forum, calling for deeper exchange and collaboration in diagnostics, research and development, and reimbursement to build a system in which innovation is not only accessible, but also affordable and sustainable for patients.

Co-hosted by the Embassy of France in China, Business France, the French Healthcare Alliance and the Chinese Hospital Association, the event was recently held at the China National Convention Center in Beijing. With a focus on health and innovation, this year's medical forum brought together more than 100 guests, including policy researchers, clinical experts, patient organization representatives and industry leaders from both countries, to share perspectives on innovation, rare diseases, prevention and early intervention, healthy longevity breakthroughs, care-system development and patient access to treatment.

Rare diseases are a public health issue that transcends national borders, said Floriane Bougeard, counselor for health, social affairs and labor at the Embassy of France in China. "We look forward to making the Sino-French Medical Forum a platform to further promote exchanges and cooperation between France and China in early diagnosis, diagnostic and treatment standards, clinical research and patient protection for rare diseases."

"China's rare-disease governance has made notable progress in recent years, yet tangible challenges remain in primary-care capacity, specialist talent reserves, innovative research and development and drug accessibility, and the development of a multitiered medical security system," said Li Linkang, executive chairman of the China Alliance for Rare Diseases, vice-president of the Chinese Hospital Association and deputy director of the office of National Rare Disease Treatment Cooperation Network.

Servier, a French pharmaceutical company engaged in China's healthcare sector since 1979, draws on global innovation and is committed to bringing innovative treatment options to patients with serious unmet medical needs, especially in rare diseases and hard-to-treat cancers.

"Patients with rare diseases and hard-to-treat cancers still face significant challenges in optimizing diagnosis and treatment pathways and improving access to innovative medicines," said Manuel Ruiz, industry co-president of the French Healthcare Alliance China and general manager of Servier China.

"Servier has accumulated global R&D expertise and practical experience in rare diseases and hard-to-treat cancers. Through high-quality exchange platforms, we look forward to promoting mutual learning between China and France in rare disease governance systems and innovative drug R&D, further supporting patient benefit," Ruiz added.

China's rare-disease governance system has gradually evolved into a multi-level, multi-stakeholder framework involving government bodies, medical institutions and wider society.

Recent data show that the National Rare Disease Treatment Cooperation Network has been expanded and strengthened, with 419 hospitals currently included. The national rare disease list has been expanded to cover 207 diseases. Meanwhile, the foundation for standardized rare disease diagnosis and treatment has been continuously consolidated, and the time to diagnosis for some patients has been shortened from six years to about four weeks.

Reimbursement coverage is also widening. The national medical insurance drug catalog now operates under a dynamic adjustment mechanism, bringing more high-value rare-disease medicines into coverage: more than 100 drugs for over 50 rare diseases are reimbursed, and patient access continues to improve. At the provincial level, more regions are piloting diversified support mechanisms — earmarked rare-disease funds, medical-assistance programs and policy-guided commercial health insurance — to ease the financial burden on families.

Next, China will steer its rare-disease governance toward four core goals: early diagnosis, access to effective medicines, affordable care and effective long-term management, Li said, adding that he hopes governments, hospitals, research institutions, pharmaceutical companies and public welfare organizations will work together to weave a stronger safety net, leaving no rare-disease patient behind as the Healthy China initiative takes shape.

Xiong Xianjun, a visiting professor at Peking Union Medical College, an expert on the rare disease professional committee of the Chinese Hospital Association and former director of the Department of Medical Service Management at the National Healthcare Security Administration, noted that diagnostic, treatment and healthcare-coverage challenges facing rare-disease communities have drawn growing attention.

The medical security system is entering a stage of high-quality development, he added. To better protect patients with rare diseases, the focus must shift from piecemeal approaches to institutional redesign: setting disease-specific payment benchmarks and integrating basic medical insurance, critical illness insurance, medical assistance, commercial health insurance and social charity resources into a durable multitiered safety net that eases the treatment burden on every patient.

Ma Wenbin, a professor at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, shared findings from a report on the burden of astrocytomas and oligodendrogliomas, which was jointly released by the Glioma Professional Committee of the China Alliance for Rare Diseases and Tianjin University in June.

The report puts the median age of onset at 40, with most patients experiencing seizures, cognitive impairment, depression and fatigue — symptoms that sharply erode quality of life. Surgery, radiotherapy and chemotherapy remain the mainstays of treatment, yet treatment-related complications continue to add to patients' physical, psychological and financial strain. In real-world care, patients also face cross-region treatment, rising costs, reduced social participation and loss of work capacity.

Ma called on all sectors of society to pay greater attention to patients with glioma, provide appropriate support through medical insurance payments and medication, improve clinical access to innovative therapies and jointly help patients and families reduce the long-term burden of the disease.

Sun Wenjun, founder of DI'AI Family, a leading patient advocacy group for glioma and neuroblastoma (childhood cancer), highlighted the full-cycle needs of rare-disease patients. He emphasized that a rare disease is not merely a medical event but a long-term life situation requiring comprehensive support — from information services and psychological counseling to clinical-research participation and policy advocacy.

"We need to shift from focusing on the disease to focusing on the person living with the disease," he said, noting that patient organizations serve as a bridge between patients, clinicians and policymakers.

Focusing on the full continuum of care for rare and hard-to-treat cancers such as gliomas, roundtable panelists exchanged views on rare-disease policy support, diagnostic and treatment collaboration, payment coverage and the development of patient support systems. They agreed that a more systematic support mechanism is urgently needed across early diagnosis and intervention, standardized care, access to innovative therapies and long-term security.

Panelists called on governments, hospitals, industry, patient organizations and civil society to work together to move from "drugs on the shelf" to care that patients can reach, afford and sustain. That is how innovation moves beyond headlines and begins to change lives.