6th Anniversary of Hemophilia Patient Assistance Program and Launch of White Paper Project Held in Beijing

BEIJING, July 5, 2021 /PRNewswire/ -- On July 4, 2021, the 6th Anniversary and White Paper Launch Ceremony for the "Bleeding Under Control, Life Unbound" Hemophilia Patient Assistance Program, hosted by the China Primary Health Care Foundation and supported by Takeda China, was officially held in Beijing.

Distinguished guests including Hu Ningning, Vice Chairman of the China Primary Health Care Foundation; Prof. Yang Renchi, Director of the Thrombosis and Hemostasis Center at the Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences; Prof. Wu Runhui from Beijing Children's Hospital, Capital Medical University; and Liu Yan, Head of the Hemophilia and Rare Diseases Business Unit, Takeda China, attended the ceremony. Together with numerous hemophilia patients and their families present at the event, they jointly drew a blueprint for freedom, witnessed a new era of freedom, and ushered in a new chapter in hemophilia treatment in China.

Ultra30,000 visits,NearRMB 150 Million, Empowering Hemophilia Patients' "Free Life"”

Hemophilia is a rare bleeding disorder in which a deficiency or insufficiency of a specific clotting factor prolongs clotting time, resulting in a tendency to bleed easily.^[1][2]. Currently, there are approximately 136,000 patients in China, with hemophilia A accounting for approximately 80%–85% of the total.^[3]。Recurrent bleeding without timely treatment can lead to joint deformities and even disability.^[4], severe bleeding symptoms may even be life-threatening.^[5]。

“Hemophilia patients only need to receive long-term, regular, and standardized treatment to achieve the ‘zero-bleed’ treatment goal and return to a normal life.”Hu Ningning, Vice Chairman of the China Primary Health Care Foundationstated, "Six years ago, we launched and implemented the 'Bleeding Has Limits, Life Has Freedom—Hemophilia Patient Assistance Program'. As the program progressed, we continuously expanded the assistance coverage, strengthened support measures, and streamlined procedures, enabling more hemophilia patients to receive timely and effective treatment."

“Blood"Limitless, Free Life" Hemophilia Patient Assistance Program was launched and has been in operation since June 1, 2015, aiming to help more patients alleviate their financial burden and regain a free life. As of May 31, 2021, the program has provided assistance to over 2,800 patients, recording more than 30,000 assistance visits, and has distributed a total of nearly RMB 150 million in financial aid. The assistance covers over 100 cities across 25 provinces, autonomous regions, and municipalities in China.

Hu NingningDeputyChairmanEmphasized, “The prevention and control of major diseases, including rare diseases, hold strategic significance for advancing the ‘Healthy China’ initiative. In the future, we will collaborate with diverse stakeholders across society to actively explore multi-tiered coverage schemes, support the improvement of service and support mechanisms for the prevention and treatment of rare diseases such as hemophilia, minimize the financial burden on patients and their families to the greatest extent, strengthen patients’ confidence in treatment, help them return to normal life, and contribute to the comprehensive advancement of the ‘Healthy China’ initiative.”

Achieving the "Zero Bleeding" Treatment Goal: Widespread Adoption of Standardized Prophylaxis Is Imperative

In recent years, alongside rapid social and economic development, both society and the government have attached great importance to rare diseases and their patient populations, and relevant policies have been continuously refined. Consequently, China’s diagnostic and therapeutic capabilities for hemophilia, as well as the accessibility of high-quality medical resources, have made significant strides. However, it cannot be overlooked that due to low public awareness of the disease, inadequate standardized prophylactic treatment, and certain limitations in existing therapeutic modalities, patients with hemophilia continue to face significant challenges, including low diagnosis rates, poor treatment adherence, and high disability rates.

Through nearly a decade of efforts by the China Hemophilia Collaboration Group, approximately 18,000 patients were registered in its network from 2007 to 2019; the proportion of patients receiving prophylactic treatment nearly doubled, reaching 16.2%, though it remains below global levels; according to a 2018 survey by the World Federation of Hemophilia, China's per capita FVIII consumption was 0.026 IU, two orders of magnitude lower than that in developed countries, indicating insufficient prophylaxis; among Chinese patients with hemophilia A, the rates of joint bleeding and joint arthropathy reached as high as 61% and 36%, respectively.^[6], not only severely impacts the daily lives and work of patients and their families, but also frequently leads to the plight of "poverty caused by illness" and "relapse into poverty caused by illness".

“Standardized treatment is an important therapeutic approach to help patients with hemophilia achieve ‘zero bleeding’ and return to a normal life.”Prof. Yang Renchi, Director of the Center for Thrombosis and Hemostasis, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciencesstated, “Patients with hemophilia have a lifelong tendency to bleed following minor trauma, and those with severe disease may experience ‘spontaneous bleeding’ even in the absence of obvious trauma, necessitating lifelong treatment. Therefore, in actively promoting the widespread adoption of standardized treatment, the latest domestic and international guidelines indicate that higher trough concentrations yield superior therapeutic benefits, while individualized therapy guided by pharmacokinetic (PK) software offers more definitive clinical efficacy. Concurrently, advancing the establishment of hemophilia centers in China will help further elevate the national diagnostic and treatment standards for hemophilia, improve the hemophilia prevention and care system, and expedite the realization of the clinical objective of ‘localized management and standardized diagnosis and treatment.’”

Prof. Runhui Wu, Beijing Children's Hospital, Capital Medical Universitystated: "Children account for the majority of hemophilia patients in China. For hemophilia prophylaxis, individualized prophylactic regimens should be tailored to pediatric patients based on factors such as age, bleeding phenotype, pharmacokinetic (PK) characteristics, and lifestyle. This approach aims to minimize bleeding risks, preserve joint health, and promote the establishment of pediatric hemophilia centers in China, thereby enabling more Chinese children with hemophilia to grow up healthily, free from disease-related distress and financial burdens, and successfully return to school."

Centered on Patient Needs, Ushering in a New Chapter in Hemophilia Treatment

On the 6th anniversary of the “Bleeding Under Control, Life Without Limits” Patient Assistance Program, the Chinese Primary Health Care Research Foundation has officially launched its inaugural 《Hemophilia White Paper》 project. Leveraging real-world survey data, the project will gain an in-depth understanding of the current diagnosis and treatment landscape for hemophilia patients in China. Centered on the unmet medical needs and disease burden of hemophilia patients, it aims to provide a valuable reference for the general public and healthcare professionals, promote the diagnosis and treatment management as well as disease education for hemophilia patients in China, further enhance attention and care from all sectors of society towards hemophilia patients, and bring hope for a better life to more individuals living with hemophilia.

Guan Tao, Chairman of Beijing Hemophilia Home Rare Disease Care Centerstated at the event: "As a hemophilia patient, I deeply empathize with the challenges and aspirations of fellow patients. I hope that all sectors of society will join hands to help more hemophilia patients access standardized prophylactic treatment and achieve the therapeutic goal of 'zero bleeding'. We will also return to daily life with a more positive attitude and create value for society."

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Notes:

1.      Orphanet. Rare disease registries in Europe. Orphanet Report Series: Rare Disease Collection, May 2017. Available at: http://www.orpha.net/orphacom/cahiers/docs/GB/Registries.pdf Last accessed October 2017.

2.      World Federation of Hemophilia. What is hemophilia? Available at: http://www.wfh.org/en/page.aspx?pid=646 Last accessed October 2017.

3.      The Prevalence of Hemophilia in China: a systematic review and meta-analysis, Southeast Asian J Trop Med Public Health. 2014 Mar;45(2):455-66

4. Wu Jing, Chinese Journal of Hospital Administration, June 2020, Vol. 36, No. 6

5. Zhao Yupei, Zhang Shuyang, et al. Guidelines for the Diagnosis and Treatment of Rare Diseases (2019 Edition). People's Medical Publishing House, 2019, 10(01): 177-178.

6.      An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies. Available at: https://onlinelibrary.wiley.com/doi/epdf/10.1111/hae.14217