Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva Released in China

BeijingNovember 24, 2023PR Newswire -- Recently, the 2023 China Rare Disease Conference was held in Beijing. At the twelfth sub-forum of this year's conference, "Endocrine Genetic Metabolic Rare Diseases," the "Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva" completed by the China Rare Disease Alliance/Beijing Rare Disease Diagnosis and Treatment and Security Association, the Center for Pharmaceutical Policy and Economics Research at Tianjin University School of Pharmacy, and Ipsen was officially released.

Fibrodysplasia Ossificans Progressiva (FOP) is a rare autosomal dominant genetic connective tissue disease caused by mutations in the ACVR1 gene, leading to progressive ossification of muscles and connective tissues throughout the body.^[1],[2]FOP has been included in the "Second Batch of Rare Disease Catalog" jointly released by the National Health Commission and five other departments.^[3]Currently, research on FOP patients in China is mostly limited to small-sample case reviews, with almost no reports available on their quality of life or economic burden. Therefore, there is an urgent need to conduct sociological surveys on FOP patients in China to gather fundamental data.

"Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva" aims to comprehensively understand the basic characteristics, diagnosis and treatment status, and disease burden of patients with Fibrodysplasia Ossificans Progressiva in China, to support clinical practice and the formulation of relevant rare disease policies. It provides substantial real-world data support for the research and development approval of innovative drugs and the formulation of relevant medical security policies.

The survey was conducted using the online synchronous research system of China's Rare Disease Comprehensive Cloud Service Platform, collecting 67 questionnaires within two days, far exceeding the expected number. After data quality control, all questionnaires were included as valid responses for analysis, efficiently completing the sociological survey of patients with Fibrodysplasia Ossificans Progressiva (FOP).

Release Ceremony Venue (Provided by the China Rare Disease Alliance)

Associate Professor He Xiaoning from the School of Pharmaceutical Science and Technology at Tianjin University interpreted the "Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva (FOP)": "This study collected a total of 67 patient questionnaires. FOP patients experience early onset, with the average age of first onset being 4.9 years. The current state of diagnosis is poor, with an average age at diagnosis of 8.0 years, leading to an average diagnostic delay of 3.1 years, with the longest delay reaching up to 18 years. The level of disability is high, with 98.5% of patients becoming disabled due to the disease. 95.5% of patients require care from family or friends. Currently, 59.7% of patients are not receiving drug treatment. In the past year, 61.2% of patients have neither visited outpatient clinics nor received inpatient treatment. Almost all patients (97.0%) currently exhibit heterotopic bone formation. As many as 92.5% of patients suffer from comorbidities or complications associated with FOP. Overall, the health-related quality of life for patients is poor."^[4]The disease burden of FOP patients is extremely heavy. We hope to promote the research, development, and approval of innovative drugs, improve the medical security system for rare disease patients, and reduce the disease burden to improve the current survival status of FOP patients.

Li Lin Kang, Executive President of the China Rare Disease Alliance, stated that the release of "The Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva (FOP)" will raise public awareness about FOP patients, provide insights for improving diagnostic and treatment services for rare diseases, and offer reference for policy formulation. Accelerating drug approval processes and enhancing medical security systems will ensure that patients with rare diseases receive proper medical care and are adequately covered.

Professor Gu Xuefan, Director of the Department of Pediatric Endocrinology, Genetics and Metabolism at Xinhua Hospital affiliated with Shanghai Jiao Tong University School of Medicine, and Director of the Rare Disease Research Center, introduced that newborn screening is an important measure for preventing rare diseases. Over the past 20 years, with the gradual popularization of tandem mass spectrometry screening for multiple inherited metabolic disorders in China, more than 40 types of inherited metabolic disorders can now be screened and diagnosed.

Professor Xia Weibo, Chief of the Endocrinology Department at Peking Union Medical College Hospital, introduced that most rare endocrine diseases are related to genetics. Clinical awareness is low, diagnosis is difficult, and there is a lack of basic and clinical research as well as effective treatment drugs, resulting in very low cure rates. The Rare Disease Registry System is a crucial tool for understanding rare diseases, especially rare endocrine diseases. Patients with FOP experience heterotopic ossification, have high disability rates, and poor clinical prognosis. The release of this survey report can provide data references to assist in the formulation and implementation of national health policies.

Professor Wu Jing, Vice Dean of the School of Pharmaceutical Science and Technology at Tianjin University, stated that the report conducts a baseline survey and examination from multiple dimensions, including the basic characteristics of diseases, current status of diagnosis and treatment, sociology, and economics. It comprehensively understands the disease burden and living conditions of patients, thereby effectively promoting the development of various safeguard measures and providing empirical evidence for future policy adjustments.

Ipsen, as a mid-sized international biopharmaceutical company, focuses on innovative drugs in the fields of oncology, rare diseases, and neuroscience. The Ipsen Shanghai Innovation Center will continue to introduce innovative treatment solutions to meet the urgent medical needs of patients in China. With the release of the "Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva," Ipsen will continue to fully promote its efforts.Waving SelfLeverage the advantages in the rare disease field, exert corporate influence, provide innovative drugs and treatment solutions for rare disease patients, and improve the quality of life for patients.

Executive Chairman of the China Rare Disease Alliance, Li Lin kang, Chief Physician of the Pediatric Endocrinology and Genetic Metabolism Department at Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine and Director of the Rare Disease Research Office, Professor Gu Xue fan, Vice Dean of the School of Pharmaceutical Science and Technology at Tianjin University, Professor Wu Jing, Director of the Guangzhou Women and Children's Medical Center, Professor Liu Li, Chief of the Endocrinology Department at Peking Union Medical College Hospital, Professor Xia Wei bo, Chief Physician of the Endocrinology Department at Peking Union Medical College Hospital, Professor Zhu Hui juan, Director of the Endocrinology and Genetic Metabolism Department at Beijing Children's Hospital and Director of the Rare Disease Research Office, Professor Gong Chun xiu, Deputy Chief of the Endocrinology Department at the Second Hospital of Hebei Medical University, Professor Zhang Song yun, Associate Professor of the School of Pharmaceutical Science and Technology at Tianjin University, He Xiao ning, and Head of Angel’s Coral (FOP) Care Home, Mr. Zhang Jian peng, jointly attended the Endocrine Genetics and Metabolic Rare Diseases Sub-forum of this year’s conference and the release ceremony of the "Sociological Survey Report on Patients with Fibrodysplasia Ossificans Progressiva."

References

[1] Akesson LS, Savarirayan R. Fibrodysplasia Ossificans Progressiva. 2020 Jun 11. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet].

[2] Zhang, JM., Li, CF., Ke, SY. et al. Analysis of clinical manifestations and treatment in 26 children with fibrodysplasia ossificans progressiva in China. World J Pediatr 16, 82–88 (2020).

[3] Notice of the National Health Commission of China on Publishing the Second Batch of Rare Disease Catalog (No. 26 [2023] of the National Health Commission)

[4] "Sociological Survey Report on Patients with Progressive Osseous Heteroplasia"

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