February 28 is World Rare Disease Day. Here are some facts about rare diseases that you may not be aware of: There are as many as 6,000 to 8,000 known rare diseases worldwide, yet effective treatments are currently available for only 5% of them; the development cycle for a new drug targeting a rare disease typically exceeds 10 years; and even with charitable medication donation programs, the average annual treatment cost for full-dose regimens of certain rare diseases still ranges from RMB 1.05 million to RMB 2.92 million.

The absence of targeted therapies, exorbitant drug prices, and limited insurance coverage have left some patients with rare diseases sighing in despair over inaccessible treatments. With over 20 million individuals affected by rare diseases in China, many face challenges in medication accessibility, forcing them to resort to compromised measures such as abbreviated or subtherapeutic dosing regimens that undermine treatment efficacy. On the occasion of International Rare Disease Day 2021, JD Health launched its “Rare Disease Care Initiative,” officially unveiling the “JD Pharmacy Rare Disease Care Center.” In collaboration with the Beijing Pain Challenge Public Welfare Foundation, it also established the “JD Health Rare Disease Care Fund.” Leveraging its supply chain capabilities, the initiative aims to enhance access to high-quality medical resources and medications for rare diseases, while mobilizing social forces to innovatively explore multi-party payment models. Furthermore, JD Health will comprehensively partner with domestic and international pharmaceutical companies specializing in rare diseases, along with other industry stakeholders and social entities, to continuously develop a multi-tiered support system for rare disease care services.
It is reported that following the launch of JD Health’s Rare Disease Care Center, patients with rare diseases can access services including remote prescription renewals issued by specialists, online medication purchases, and home delivery. By leveraging JD Health’s extensive portfolio of rare disease medications and its “self-operated cold chain” logistics model, the initiative aims to alleviate challenges associated with seeking medical care and purchasing medications across different regions, thereby improving accessibility to essential treatments for a broader population of rare disease patients. Furthermore, the establishment of the Rare Disease Care Fund will address long-term challenges such as lifelong medication needs and cross-regional treatment, providing convenient medication support and charitable assistance for medication costs to patients facing financial hardship.
“JD Health will join hands with multiple partners to provide assistance to the rare disease community by ensuring drug supply, facilitating access to medical services, and promoting the establishment of a multi-party payment system,” said Xin Lijun, CEO of JD Health, at the launch ceremony of the JD Pharmacy Rare Disease Care Center and the JD Health Rare Disease Care Fund.

Addressing the challenges faced by patients with rare diseases and improving access to medical resources and medications in this field require a concerted effort from social resources and industry stakeholders. On the occasion of International Rare Disease Day 2021, JD Health joined hands with BeiGene, Biogen, Novartis, Pfizer, Bristol-Myers Squibb (BMS), Canaan Healthcare, Boehringer Ingelheim, GlaxoSmithKline (GSK), Nhwa Pharmaceutical, Takeda Pharmaceutical, and other pharmaceutical companies to jointly advocate for rare disease awareness under the campaign “Raising Our Voices for the ‘Rare’.” This initiative expressed their shared commitment to providing high-quality medications and services to patients with rare diseases and supporting their healthy lives by enhancing drug accessibility.
Promoting the research, development, and market launch of new drugs, as well as establishing affordable medication purchasing models for patients, are critical pathways to addressing the plight of individuals with rare diseases. During the roundtable forum at the launch event for the “JD Pharmacy Rare Disease Care Center” and the “JD Health Rare Disease Care Fund,” pharmaceutical industry experts engaged in in-depth discussions on the topic of “How to Improve Access to Rare Disease Medications Through Innovative Models.”

Chen Hu, Head of the Neuroscience Business Unit at Novartis Pharmaceuticals (China), stated in his address that continuously launching superior innovative medicines and ensuring patients can “afford quality drugs” are the two key factors for pharmaceutical companies to improve access to treatments for rare diseases. In recent years, Novartis has actively responded to national calls by intensifying its efforts in drug innovation, disease education, and channel expansion. The company is committed to advancing standardized diagnosis and treatment for multiple sclerosis (MS). Through medical insurance negotiations, charitable collaborations, precision therapy, and in-depth cooperation with the JD Health Rare Disease Care Center, Novartis is concentrating its resources to resolve the “last mile” challenge in medication access. These initiatives aim to enable China’s Class I innovative drugs for multiple sclerosis to benefit more patients and families, illuminating China and making care accessible to all.

As a renowned innovative pharmaceutical company both in China and globally, BeiGene’s Vice President and Head of Marketing for Greater China, Liu Yan, stated that for pharmaceutical companies, the first step in addressing the challenges of rare diseases is to resolve issues of accessibility. Currently, BeiGene is helping patients overcome the dilemma of having “no available treatments” through new drug development and the introduction of cutting-edge therapeutic solutions from abroad. Meanwhile, the company also aims to further enhance medication accessibility and affordability for patients by collaborating with JD Health’s Rare Disease Care Center, thereby benefiting a larger number of rare disease patients.

“Developing innovative drugs that meet the needs of patients with rare diseases in China and providing affordable medications to these patients is the greatest responsibility and mission of Chinese innovative pharmaceutical companies specializing in rare diseases,” stated Lu Yijun, General Manager of Canbridge Pharmaceuticals Inc. in China. He emphasized that drug supply, along with improvements to diagnostic and treatment systems and medical insurance policies, forms the foundation for enhancing the accessibility of rare disease treatments. Meanwhile, Canbridge Pharmaceuticals will collaborate with platforms possessing digital technology capabilities, such as JD Health, to provide greater support for patient consultation, treatment, and management.

Lu Shiwei, Head of Neuroscience Business at Sanofi Genzyme China, noted in his sharing that, in addition to accelerating the launch of innovative drugs in China, Sanofi is also actively promoting innovative diagnosis and treatment models through artificial intelligence and digitalization. The company aims to make unremitting efforts through multi-party collaboration to ensure that patients with rare diseases have access to medical care, available treatments, and insurance coverage for their medications. The launch of the JD Health Rare Disease Care Center will help further optimize the “last mile” delivery of rare disease medications, working together with pharmaceutical companies to bring hope and light to patients’ lives.

Song Faxian, Head of Oncology and Rare Disease Marketing at Pfizer Biopharmaceuticals Group China, stated that leveraging scientific solutions and digital intelligent tools to accelerate the resolution of diagnostic and therapeutic challenges in rare diseases is a key area for pharmaceutical companies to explore. It is essential to integrate efforts from all stakeholders to establish an end-to-end pathway for rare diseases with available treatments, ensuring accessibility to diagnosis, treatment, and standardized care. The launch of the JD Health Rare Disease Care Center has created a platform for collaborative service to rare disease patients, enabling the convergence of resources and efforts from various parties. Pfizer aims to seize this opportunity to further integrate its strengths in drug research and development and diagnostic and therapeutic capacity building, along with its global resources, to better serve the vast population of rare disease patients in China in collaboration with societal stakeholders.

It is evident that the response and participation of numerous pharmaceutical companies have created greater possibilities for improving the accessibility and affordability of treatments for rare diseases. However, given the large population of patients with rare diseases in China, low public awareness, and the late development of diagnosis and treatment networks, establishing a multi-party assistance model by leveraging comprehensive social forces remains the optimal approach to resolving the challenges faced by these patients in receiving diagnosis, treatment, and medication.
As the country further promotes the institutionalization and standardization of rare disease management, more social organizations, internet companies, and pharmaceutical enterprises are actively joining the construction of China’s rare disease prevention and control ecosystem. They are researching and exploring new pathways to promote standardized diagnosis and treatment of rare diseases, improving the level of disease diagnosis and treatment as well as new drug development, and striving to provide timely and efficient medical services for patients with rare diseases. As a result, more patients with rare diseases are expected to alleviate their burdens, access treatment, and embark on a healthy life.