Pfizer Launches 'Dual-Dimensional Target Achievement' Initiative to Advance Hemophilia Care in China

On November 7, 2023, during the Sixth China International Import Expo, Pfizer Rare Disease hosted an event themed "Science Wins, Innovation Leads." The event focused on innovative exploration of hemophilia treatment models and the upgrading of therapeutic drugs. In collaboration with multiple parties, Pfizer aimed to promote the practice of "homogeneous diagnosis and treatment, localized management." For the first time in the industry, the concept of "double-dimensional standards, freedom with a method" for hemophilia diagnosis and treatment was proposed, continuously advancing clinical diagnosis and treatment development and comprehensive patient care until the goal of "breaking free from the constraints of the disease" is achieved, thereby "restarting a life of freedom and illuminating a better life" for hemophilia patients in China, providing experience and a model for the construction of a rare disease diagnosis and treatment system in China.
This event has received significant attention in the field of hemophilia diagnosis and treatment in China. Mr. Li Lin kang, Executive President of the China Alliance for Rare Diseases, Professor Yang Ren chi, Director of the Thrombosis and Hemostasis Treatment Center at the Hematology Hospital of the Chinese Academy of Medical Sciences, Professor Sun Jing, Director of the Hematology Department at Nanfang Hospital affiliated with Southern Medical University, Professor Wu Jing sheng, Director of the Hematology Department at the First Affiliated Hospital of the University of Science and Technology of China, and Mr. Guan Tao, Chairman of Beijing Hemophilia Home Rare Disease Care Center, were among the many guests invited to attend this event, where they engaged in in-depth exchanges and shared experiences.
"The CIIE provides Pfizer with a high-quality, open platform to introduce and showcase its global innovative therapies and treatment concepts in the rare disease field. Pfizer is always committed to exploring and accelerating the introduction of breakthrough innovations in the rare disease area, benefiting countless patients and families affected by rare diseases," said Mr. Song Faxian, General Manager of Pfizer China's Rare Disease Division. "At this year’s CIIE, Pfizer, together with industry partners, advocates for and practices a new goal of personalized hemophilia treatment, demonstrating Pfizer’s global exploration and Chinese practice in innovative hemophilia treatment solutions, shaping a 'China Solution' for hemophilia diagnosis and treatment through innovation. 'Science Will Win, For Love, For Rare' is Pfizer's unwavering commitment to rare diseases in China. Pfizer will further enhance the level of diagnosis and treatment of rare diseases, including hemophilia, allowing patients with rare diseases to have a better future."

Faxon Song, General Manager of Pfizer China Rare Disease Business Unit, Speaking at the Event

"Double Criteria Achievement" Helps Hemophilia Patients Restart a Life of Freedom

Hemophilia is an X chromosome-linked recessive hereditary bleeding disorder with a high disability rate, and patients suffer from lifelong coagulation dysfunction. Data shows that the incidence of hemophilia in China is about 2.73/100,000, with a disability rate as high as 70%. Two-thirds of patients will experience joint and muscle disabilities after reaching adulthood, affecting their normal life and work abilities. In recent years, factor replacement therapy has gradually matured. Guided by the treatment goal of "controlling bleeding and protecting joints," patients can maintain normal joint function to the greatest extent, avoid fatal bleeding, and achieve self-care and independent living. The development of diagnostic and treatment standards has also driven the treatment demands of hemophilia patients, evolving from "survival" and "pain-free" to "disability-free," gaining the ability for independent living, and looking forward to "breaking free from the shackles of the disease" and returning to normal social life as soon as possible.

At this year's CIIE, Pfizer, in collaboration with the China Rare Disease Alliance, leading figures in hemophilia diagnosis and treatment, and patient organizations, has taken the lead in launching the concept of "Dual Goals, Freedom with Strategy." This reaffirms the critical importance of the two core factors affecting the prognosis of hemophilia patients: "reducing bleeding" and "joint protection," while emphasizing the integration of joint protection throughout the entire treatment process.

(From left to right) Mr. Song Faxian, General Manager of Pfizer China Rare Disease Division, Ms. Qian Yun, Vice President of Pfizer China and Head of Market Access, Mr. Guan Tao, Chairman, Professor Yang Renchi, Mr. Li Lin Kang, Chairman, Professor Sun Jing, Professor Wu Jingsheng, Dr. Cao Junyang, Chief Medical Officer of Pfizer China, and Ms. Guo Jin, Head of Communications at Pfizer China, jointly launched "Dual Control, Freedom in Reach - Beyond Bleeding."

"In addition to avoiding sudden bleeding events in patients, patients and their families should also pay attention to joint damage caused by subclinical bleeding that is not easily detected," said Professor Yang Renchi. "Achieving 'dual-dimensional target achievement' requires establishing a Shared Decision-Making (SDM) system between hemophilia doctors and patients, regularly evaluating the degree of bleeding and joint condition, dynamically adjusting treatment goals and plans, and helping patients achieve better disease management and quality of life. This is also an essential path toward realizing the ultimate treatment goal for hemophilia: 'standardized diagnosis and treatment, localized management.'"

Leading Innovation Breakthroughs: Exploring a New Phase in Hemophilia Diagnosis and Treatment in China

The treatment of hemophilia is a comprehensive system that requires an integrated solution centered on patients. It integrates all related nodes in the whole chain of hemophilia management to address challenges in areas such as medication, diagnosis and treatment, payment, nursing, psychological support, and patient education. Through comprehensive care at all levels, it aims to further improve the quality of life for hemophilia patients. Precisely because of this, caring for rare diseases requires the collective wisdom and more efforts from the whole society.

Mr. Li Linkang, Chairman of the China Alliance for Rare Diseases, stated: "In recent years, the China Alliance for Rare Diseases has been committed to enhancing the diagnosis and treatment capabilities for rare diseases, conducting many pioneering efforts in building and improving the hemophilia treatment system. Significant progress and encouraging achievements have been made in the development of hemophilia centers, greatly advancing the process of 'standardized treatment and localized management.' With the joint efforts of the entire industry, hemophilia care in China is entering a fast track toward a new stage of treatment. We hope to leverage the platform and opportunity of the CIIE (China International Import Expo) to draw more attention from all stakeholders to the latest advancements in hemophilia treatment, accelerate the introduction of innovative therapies, increase awareness of the needs of hemophilia patients, and encourage everyone to join in promoting the advancement of hemophilia care, ensuring that patients with rare diseases no longer feel alone."

The development of hemophilia diagnosis and treatment has posed new requirements for traditional treatment methods, and the clinical demand for drug innovation is also increasing. Currently, Pfizer is actively exploring non-factor therapy and gene therapy for hemophilia, aiming to meet the clinical needs of some hemophilia patients whose symptoms cannot be alleviated after receiving factor replacement therapy. Pfizer's non-factor therapy has a unique mechanism of action, helping patients re-establish the balance between bleeding and coagulation. It adopts a fixed dose and is administered subcutaneously once a week, treating either type A or type B hemophilia. At present, China has joined the global research and synchronous development, hoping to benefit domestic patients as soon as possible.

Mr. Song Faxian, General Manager of Pfizer's Rare Disease Division in China, stated: "‘Enabling every rare disease patient to embrace a new life’ is Pfizer Rare Disease’s steadfast commitment. In the field of hemophilia, Pfizer is dedicated to bringing innovative scientific solutions to more rare disease patients, actively exploring ways to address key links in the entire diagnosis and treatment chain, and working with multiple parties to establish and improve the hemophilia diagnosis and treatment system, helping Chinese hemophilia patients achieve the vision of ‘Zero Worries in Treatment, Zero Restrictions in Life’ more quickly. At the same time, Pfizer is actively promoting the accelerated introduction of more innovative products and treatment concepts into China, mobilizing all sectors of society to jointly build an ecosystem for hemophilia diagnosis and treatment, and making relentless efforts to establish hemophilia diagnosis and treatment standards as a model for rare disease treatment in China."