(Shanghai, China, September 27, 2020) - Today, a training on rare disease registration and diagnosis & treatment standards was held successfully by the National Rare Diseases Collaboration Network in Shanghai. During the conference, VISEN Pharmaceuticals (“VISEN”) signed a formal strategic cooperation agreement with China Alliance for Rare Disease. During the next 5 years, the two parties will implement a series of initiatives related to achondroplasia (ACH) to improve diagnosis and treatment of ACH in China, raise public awareness of rare diseases, especially ACH, and promote the overall level of diagnosis and treatment of rare diseases in China, and ultimately optimize the treatment and quality of life of Chinese patients who suffer from rare diseases.
After signing the contract, the two parties will conduct a multicenter patient registration study (ApproaCH) among Chinese patients with achondroplasia, including the establishment of a cohort of Chinese ACH patients, the completion of investigation reports on ACH patient status, and the conduction of ACH patients burden of diseases (BOD) studies. The two parties also will facilitate the development of “Chinese Clinical Expert Consensus on Achondroplasia (or Guidelines)," implement training and experience exchange activities for medical staff to improve the diagnosis and treatment of rare diseases and organize popularization and education activities to raise the awareness of such diseases among patients. In addition, the two parties will focus on in-depth research on the pathogenesis of ACH, prenatal screening and diagnosis, disease diagnosis and clinical management, the understanding of the risk of the disease, and its prognosis, with an aim to provide the basis for decision-making on clinical diagnosis, treatment and prevention.
Li Linkang, Executive Director of China Alliance for Rare Disease and Vice President of Chinese Hospital Association,
signed a strategic cooperation agreement with Pony Lu, CEO of VISEN.
Li Linkang, Executive Director of China Alliance for Rare Disease and Vice President of Chinese Hospital Association, Zhao Lin, Deputy Secretary-General of China Alliance for Rare Disease, Professor Gu Xuefan, Director of Shanghai Children's Rare Disease Diagnosis & Treatment Center and chief physician of Xinhua Hospital, Pony Lu, CEO of VISEN, and Dr. Yang Jun, CMO of VISEN attended the signing ceremony.
Integrate high-quality resources to facilitate the development of prevention and treatment of rare diseases in China
Since the 18th National Congress of the Communist Party of China, the CPC Central Committee and the State Council have upheld "people-centered” concepts and attached great importance to the management of rare diseases. The National Health Commission (NHC), National Medical Products Administration, National Healthcare Security Administration and other relevant national administrations have adopted a series of measures to improve the diagnosis and treatment of rare diseases, the evaluation and approval of drugs, as well as drug availability, to promote the development of healthcare for rare diseases in China. Although rare diseases are receiving more attention, there have always been medical challenges, with low prevalence, large variety, heavy BOD, difficulty in diagnosis and treatment, and other characteristics. Take achondroplasia as an example, of which there is no known cure due to lack of epidemiological data and effective diagnosis and treatment methods.
“In recent years, China has made rapid progress in clinical research, diagnosis and treatment innovation, and drug availability of rare diseases. China Alliance for Rare Disease has played an organizational role as a learned society in integrating the high-quality resources from clinical diagnosis and treatment of rare diseases, medical insurers, pharmaceutical enterprises, and public welfare organizations. It helps promote breakthroughs in medical research on rare diseases, improve the level of prevention and protection from rare diseases, and enhance collaborative innovation in clinical, scientific research and orphan drug development for rare diseases,” said Li Linkang, Executive Director of China Alliance for Rare Disease and Vice President of Chinese Hospital Association. "Through our cooperation and joint efforts, we hope that both parties fully utilize our respective advantages and work together to facilitate the registration, standard-setting and capacity enhancement of rare diseases, enrich the epidemiological data of rare diseases in China, and provide a reference for decision-making in formulating policies in medical insurance, medicine and medical care, therefore promoting the development of rare disease prevention and protection in China to realize our goal of benefiting patients."
Raise awareness and care to fill the gaps in diagnosis and treatment, so that the rare disease patients no longer “walk alone”
Achondroplasia is a kind of congenital dysplasia due to endochondral ossification defect, with a prevalence rate of 1:25,000 among live births. It affects the linear bone growth. The clinical manifestations are disproportionately short stature and a series of skeletal and neurological complications. Patients are under great pressure both physically and psychologically, and it is difficult for them to integrate into society, facing difficulties in employment, marriage, daily life and other aspects1. Also, poverty caused by illness is common among patients with achondroplasia and their families.
"Due to the lack of sufficient clinical research and relevant education in China, the public has low awareness of achondroplasia. At the same time, medical practitioners also need to improve their recognition and awareness of the disease," Professor Gu Xuefan, Director of Shanghai Children's Rare Disease Diagnosis & Treatment Center and Chief physician of Xinhua Hospital, indicated. "At present, there is no fundamental solution for patients with achondroplasia, and Chinese doctors lack the relevant expertise and clinical experience in this area. To make matters worse, patients with achondroplasia are likely to suffer from serious complications, such as hydrocephalus and obstructive sleep apnea, which result in a higher risk of death. As the disease imposes a huge burden to patients and their families, it is extremely important to establish a consensus among medical experts on this disease to fill the gaps in diagnosis and treatment."
“VISEN Pharmaceuticals is committed to becoming a specialist in endocrine-related disease therapy, and the aim of our collaboration with China Alliance for Rare Disease is to realize this ambitious dream and our commitment to our patients,” said Pony Lu, CEO of VISEN. “In the future, VISEN will work deeply with China Alliance for Rare Disease, and at the same time, introduce the world's leading treatment methods and innovative drugs to China, so that more Chinese patients can benefit earlier from the world's most advanced and reliable treatment solutions. In doing so, we will help Chinese patients who suffer from achondroplasia, make a real contribution to the development in the prevention and treatment of rare disease in China, and accelerate the realization of a 'Healthy China 2030'."
About VISEN Pharmaceuticals
VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the world's advanced and reliable treatment solutions.
In 2018, VISEN Pharmaceuticals (“VISEN”) was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Ventures), to develop and commercialize Ascendis Pharma’s endocrinology rare disease therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.
About China Alliance for Rare Disease
China Alliance for Rare Disease (hereinafter referred to as "the Alliance") is a nationwide, non-profit and cooperative exchange platform, approved by Medical Administration of NHC, initiated by Peking Union Medical College Hospital, China Pharmaceutical Innovation and Research Development Association, Chinese Hospital Association, and Chinese Research Hospital Association, and jointly and voluntarily established by medical institutions, colleges and universities, scientific research institutes, enterprises and other organizations that have the professional ability to diagnose and treat rare diseases.
The alliance aims to help build a healthy China and continuously meet people's growing needs for a better life. It adheres to the people-centered concept of development and gives full play to the advantages of medical institutions, universities, scientific research institutes, and enterprises. The joint work between industry leaders, complementary advantages, and multilateral cooperation will reinforce the clinical research of rare diseases, optimize the standardized system for diagnosis and treatment of rare diseases, strengthen the health education of patients and medical staff, continuously promote the level of clinical diagnosis and treatment, and push forward the development of rare disease-related biomedical medicine industry, thereby improving the quality of life of patients with rare diseases.
 Pauli RM. Orphanet J Rare Dis. 2019;14(1):1-49