Research from Analysis Group and IHBDH Furthers Understanding of Hemophilia in China

Date:February 9, 2021
Location:Boston, MA
Contact:Eric Seymour
978-273-6049

Consultants from Analysis Group, a global leader in health economics and outcomes research (HEOR), and the Institute of Hematology & Blood Diseases Hospital (IHBDH), a clinical and research institute of the Chinese Academy of Medical Sciences and Peking Union Medical College, published a pair of studies in the journal Haemophilia advancing the understanding of hemophilia in China. The study results provide critical information on patient characteristics, treatment strategies, and other clinical information needed to aid regulatory decision making, enhance the quality of care, and improve patient outcomes.

There is an unprecedented effort underway to develop robust, real-world evidence for hematological conditions. These studies are some of the first to leverage China’s first comprehensive blood disease research platform, the National Longitudinal Cohort of Hematological Diseases in China (NICHE), a multi-disease cohort designed to study an array of hematological conditions such as hemophilia, acute myeloid leukemia, lymphoma, and multiple myeloma, among others.

The first study, “An overview of patients with haemophilia A in China: Epidemiology, disease severity and treatment strategies,” analyzes medical histories from 17,779 patients from 166 member hospitals of the Hemophilia Treatment Center Collaboration Network of China between 2007 and 2019. Researchers found that a high proportion (44%) of patients received only episodic, on-demand treatments, while only 16% received prophylactic clotting treatments. Further, nearly 60% of patients had joint bleeding, and 36% had joint arthropathy, indicating that their treatments were inadequate.

A possible contributing factor to these patterns may have been that prophylactic clotting treatments are rarely reimbursed for adult patients. And even though these treatments are reimbursed for pediatric patients, parents do not always accept the use of prophylaxis due to the lack of awareness and different health beliefs. In contrast, prophylaxis use for adult patients in the UK during the same period was 78%, and 75% in the US. These large gaps reiterate the need to improve hemophilia A treatment accessibility and prophylactic care affordability in China. The full study can be found in the November 2020 issue of Haemophilia.

The second study, “Real‐world analysis of haemophilia patients in China: A single centre’s experience,” was conducted to assess the evolution of real-world hemophilia A/B care between 2004 and 2018 for 428 patients treated at China’s largest hematology center. This is the first study in China to use cost data to enhance the understanding of the evolution of hemophilia care.

Researchers found a significant improvement in the delay between diagnosis and treatment. This result may be associated with a series of assistance programs, including a 2007 policy change to include hemophilia in the outpatient reimbursement scheme for special diseases; the addition of recombinant FVIII treatment for on-demand use to the National Reimbursement Drug List in 2009; and a 2009 charity assistance project to provide financial support to patients with hemophilia in 29 regions in China. Since then, significant improvement in the treatment of children and adults with hemophilia have been observed. For example, the annual factor consumption of both on-demand and prophylactic treatments increased in both 2009–2013 and 2014–2018, compared to 2004–2008. The full study can be found in May 2020 issue of Haemophilia.

An Analysis Group team led by Managing Principal Eric Wu, Vice President Jipan Xie, and Manager Jia Zhong supported renowned experts from the State Key Laboratory of Experimental Hematology, the National Clinical Research Center for Blood Diseases, the Institute of Hematology & Blood Diseases Hospital, the Tianjin Laboratory of Blood Disease Gene Therapy, the CAMS Key Laboratory of Gene Therapy for Blood Diseases, and the Chinese Academy of Medical Sciences and Peking Union Medical College.

For more information about the NICHE research platform, please visit www.niche-study.org. To learn more about Analysis Group’s HEOR capabilities, visit www.analysisgroup.com/healthoutcomes

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About Analysis Group’s HEOR Practice

Founded in 1981, Analysis Group is one of the largest international economics consulting firms, with more than 1,000 professionals across 14 offices. Analysis Group’s health care experts apply analytical expertise to health economics and outcomes research, clinical research, market access and commercial strategy, and health care policy engagements, as well as drug safety-related engagements in epidemiology. Analysis Group’s internal experts, together with its network of affiliated experts from academia, industry, and government, provide our clients with exceptional breadth and depth of expertise and end-to-end consulting services globally.