AstraZeneca Reinforces Global Leadership in Building a Comprehensive Rare Disease Diagnosis, Treatment, and Support Ecosystem

February 29 is the 17th...Rare Disease Day, this year's theme is "Share Your Colours," which not only reminds the industry to pay attention to the living conditions of rare disease patients but also calls on the whole society to take action together to provide comprehensive support and assistance for rare disease patients.

Patient-centered drug development has become an industry consensus, and this is even more evident in the field of rare diseases. AstraZeneca’s patient insights clearly lead the way. During this year's International Rare Disease Day, the global management team of AstraZeneca’s Rare Disease Division gathered in China for a packed schedule, demonstrating their deep concern for Chinese patients and the importance they place on the Chinese market.

After meeting and exchanging ideas with experts in Beijing and Shanghai, the global management team of AstraZeneca's Rare Disease Division also engaged in discussions with local rare disease enterprises to explore collaboration opportunities, aiming to establish a stronger partnership with China’s rare disease ecosystem. At the Rare Disease Day exchange event held on February 29, the global management team gathered with relevant patient organizations, focusing on the medication challenges faced by rare disease patients in China, listening to the voices of Chinese rare disease patients, and emphasizing AstraZeneca's commitment to helping the rare disease community regain hope and a new life.

Hu Yiqing, Vice President of AstraZeneca China and Head of the Rare Disease Business Unit, stated that adhering to a patient-centered approach and to meet the needs of patients with rare diseases, AstraZeneca has been making relentless efforts. "We not only ensure the comprehensive and rapid introduction of innovative medicines globally but also, through cooperation with multiple parties, including the government, ensure that patients can access these medicines because only then can the true value of these innovative drugs be realized. In the future, AstraZeneca will continue to support all partners in exploring multi-level security models to help reduce the medical burden on patients with rare diseases and enhance their healthcare security rights."

Rare diseases are a major health challenge faced globally and one of the emerging therapeutic areas that AstraZeneca is most concerned about at the global level.

The world's largest rare disease database (Orphanet) shows that there are currently over 7,000 known types of rare diseases globally. Among them, rare diseases with onset at birth or during childhood account for 50% to 70%.¹ . With a large population base, China has more than 20 million patients with rare diseases.² , one of the largest patient populations with rare diseases globally. However, until now, there are still many unmet needs for patients with rare diseases in China and even globally.

As a leading global pharmaceutical company, AstraZeneca is accelerating its layout in the rare disease sector both globally and in China. In 2021, AstraZeneca officially entered the rare disease field through the acquisition of Alexion Pharmaceuticals and swiftly established a Rare Disease Business Unit in China in September of the same year. Shortly after, in 2022, AstraZeneca acquired LogicBio Therapeutics, a clinical-stage gene company, focusing on developing novel gene therapies for pediatric patients with rare diseases. In July 2023, AstraZeneca reached a collaboration agreement with Pfizer to advance next-generation genomic medicines, enhancing complementary pipeline assets and developing innovative technologies.

On the other hand, through the collaboration of R&D resources, AstraZeneca, based on the integration of 11 molecular product lines worldwide, is advancing more than 20 clinical development programs targeting various indications. These include the development of small molecule inhibitors for the alternative pathway of the complement system, with research coverage spanning multiple disease areas such as hematology, nephrology, central nervous system, metabolism, and ophthalmology. In the next five years, it is expected that more than 10 new products and new indications will be approved, helping more patients with rare diseases regain their brilliance in life.

As a leader in the healthcare industry, AstraZeneca not only continuously provides high-quality innovative products for patients with rare diseases but also is committed to promoting the accessibility and affordability of rare disease drugs from multiple aspects, safeguarding the survival hope and healthy life of Chinese patients with rare diseases.

In the Chinese market, in just two years, AstraZeneca China's Rare Disease Division has introduced two innovative drugs, covering five indications. Starting from January 1 this year, the new version of the "National Basic Medical Insurance, Work-related Injury Insurance, and Maternity Insurance Drug Catalog (2023)" was officially implemented, with several rare disease drugs listed, including those developed by AstraZeneca for the treatment of adult and pediatric paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), adult anti-acetylcholine receptor (AChR) antibody-positive refractory generalized myasthenia gravis (gMG), and for the treatment of children aged 3 years and above with symptomatic, inoperable plexiform neurofibromas (PN) associated with Neurofibromatosis Type 1 (NF1).

According to the statistics from the Illness Challenge Foundation, more than 100 drugs related to the treatment of rare diseases have been included in the national medical insurance catalog.³ , patients with rare diseases have more medication options and their medication burden is further reduced. In the industry's view, the support from the medical insurance payment end will effectively improve the accessibility of innovative drugs for patients suffering from related rare diseases, thereby achieving health benefits and quality of life improvement, and largely reducing the economic burden of treatment on patients' families.

It is foreseeable that the rare disease business will become an important driver for AstraZeneca to achieve a new round of performance growth. When talking about the reasons and advantages of developing the rare disease business, Hu Yiqing believed: "Currently, about 90% of known rare diseases are still in a situation where there is no available treatment. AstraZeneca is committed to continuously bringing innovative drugs to patients in the field of rare diseases, which can save their lives and improve their quality of life. This is also the reason and mission for us to develop the rare disease business. In the field of rare diseases, AstraZeneca has the R&D strength to transform the complex biology of the complement system into innovative drugs. We have more than 15 years of relevant development and diagnosis and treatment experience at the global level, accumulating profound expertise. In addition, we have also rapidly entered the non-complement therapeutic areas in recent years and achieved many significant results."

In recent years, China has continuously introduced multiple policies to support the research and development of drugs for rare diseases, including the establishment of a national collaborative network for the diagnosis and treatment of rare diseases, the release and updating of the "Rare Disease Catalog," priority review and approval for rare disease drugs, reduction of import tariffs, and the inclusion of rare disease drugs in medical insurance through negotiations and adjustments to the National Reimbursement Drug List. These efforts aim to improve the level of diagnosis and treatment for rare diseases, enhance the accessibility of rare disease drugs, and address the issue of drug security for patient populations.

From no available treatment, to having treatments accessible, and then to being able to afford them, the changes in the medical environment experienced by rare disease patients fully reflect the attention and care given to this group at the national level. With numerous favorable policies in place, pharmaceutical companies are firmly committed to expanding their presence in the rare disease sector in the Chinese market. They are also working hand-in-hand with all parties to further boost the confidence in making treatments accessible for rare disease patients. Undoubtedly, AstraZeneca is one of the most active representative companies in this field.

Currently, Qingdao is becoming the "bridgehead" for AstraZeneca to advance its rare disease business in China. In April 2022, AstraZeneca and the Qingdao Municipal Government signed a memorandum of cooperation during the Boao Forum for Asia, announcing the joint development of a leading rare disease treatment center in China. This collaboration aims to promote an integrated diagnosis and treatment model for the rare disease field, implement innovative full-course management solutions, and build an innovative ecosystem centered around rare disease treatment.

Subsequently, AstraZeneca further partnered with the Qingdao government to deepen cooperation in the field of rare diseases, establishing the "Qingdao-AstraZeneca International Innovation Research Center for Rare Diseases." Through collaboration with Qilu Hospital of Shandong University (Qingdao), the center integrates medicine, industry, academia, and research to support initiatives such as building a rare disease data platform, establishing project funds, creating research institutes and industrial alliances, and setting up an international exchange platform for rare diseases. Additionally, through policy support and pilot explorations in the rare disease field, it aims to foster a regional innovation ecosystem for rare disease diagnosis and treatment, further establishing an international demonstration hub for rare diseases.

In the research of rare diseases and standardized diagnosis and treatment, AstraZeneca is continuously making efforts. In June 2022, AstraZeneca's Global R&D China Center launched the "AstraZeneca R&D China Translational Medicine Research Fund" project to support and encourage Chinese clinical doctors and experts to carry out basic medical research and related translational studies. In November of the same year, AstraZeneca signed a biotech strategic cooperation agreement with CanSino Biologics. The two parties will collaborate on the quadrivalent meningococcal conjugate vaccine and complement inhibitors in the field of rare disease diagnosis and treatment, further improving the ecosystem for rare disease diagnosis and treatment in China.

In terms of promoting patient medication security, AstraZeneca is further deepening its cooperation with Shandong Province by signing a strategic cooperation framework agreement with the Shandong Provincial Medical Security Bureau. Both parties will strengthen cooperation in the medical security system for rare diseases in areas such as drug launches, medical insurance, and commercial insurance.

In addition, AstraZeneca hopes to leverage the "Hong Kong-Macao Medicine and Device Connect" pilot policy in the Guangdong-Hong Kong-Macao Greater Bay Area to promote the rapid access of globally innovative orphan drugs, further enhance the early availability of clinically urgent medicines, enrich medical institutions' approaches to disease management, and bring hope to patients in need. At the same time, AstraZeneca will continue to strengthen cooperation with the Chinese Medical Association, the China Rare Disease Alliance, and provincial rare disease quality control units; it will also sign a project agreement with the Beijing Kangmeng Charitable Foundation to consolidate public welfare efforts and provide multi-level diagnostic and treatment support for rare disease patients.

Not only that, but against the backdrop of significant progress in the research and development of innovative drugs for rare diseases in China, AstraZeneca is also strengthening its cooperation with the local pharmaceutical industry. Hu Yiqing stated that, to drive the global advancement of rare disease medicine, AstraZeneca has fully integrated the advantageous resources of Alexion Pharmaceuticals in the rare disease field. Relying on its leading scientific innovation capabilities and forward-looking layout in the rare disease area, AstraZeneca is making every effort to accelerate the introduction of global innovative rare disease medicines into China while actively promoting the localization of China’s rare disease diagnosis and treatment ecosystem. "In the future, we will continue to collaborate with governments, medical institutions, and partners across China to improve the accessibility of innovative drugs for rare diseases and contribute to building a robust ecosystem for the diagnosis, treatment, and support of rare diseases—working together towards a future where rare diseases are no longer untreatable."

For media use only.

Billions of Dollars in State-Owned Assets Integration! CR Pharmaceutical, Sinopharm Group, and China General Spark a Wave of Mergers and Acquisitions

BeiGene's Zanubrutinib Breaks Through "One Billion Dollars"! Global Commercialization, How to Create the Next Blockbuster?

BMS Grants Zai Lab 10-Provincial Sales Rights for O Drug, Mabwell Biotech Regains Full Rights! Is a Major Upheaval Brewing in China's PD-1 Market?

www.yyjjb.com.cn

Insight into Industry Trends

Long press to followPharmaceutical Economy Report

"Pharmaceutical Economy Report"

Academic Official Account

Focus on the Frontier of Oncology Academia

Press and hold to followImmunity Time

"Pharmaceutical Economy Newspaper"

Terminal Official Account

Record Major Events in the Pharmaceutical Industry

Press and hold to follow21st Century Pharmacy