Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)

In recent years, with strong support from the national government, the diagnosis and treatment of rare diseases in China have achieved significant progress. However, only 5% of rare diseases worldwide have effective treatments, and poor drug accessibility remains a major challenge in the field of ra...

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Main Author: Rare Disease Drug Committee of the Chinese Pharmaceutical Association
Format: Article
Language:zho
Published: Editorial Office of Medical Journal of Peking Union Medical College Hospital 2025-05-01
Series:Xiehe Yixue Zazhi
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Online Access:https://xhyxzz.pumch.cn/article/doi/10.12290/xhyxzz.2025-0252
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author Rare Disease Drug Committee of the Chinese Pharmaceutical Association
author_facet Rare Disease Drug Committee of the Chinese Pharmaceutical Association
author_sort Rare Disease Drug Committee of the Chinese Pharmaceutical Association
collection DOAJ
description In recent years, with strong support from the national government, the diagnosis and treatment of rare diseases in China have achieved significant progress. However, only 5% of rare diseases worldwide have effective treatments, and poor drug accessibility remains a major challenge in the field of rare diseases, both in China and globally. Due to limited research data, insufficient clinical experience, and a lack of medication expertise, there is an urgent need for pharmacists to play an expanded role throughout the rare disease care process, alongside the establishment of systematic management and monitoring systems. To enhance the leading role of medical institutions in securing rare disease drug availability, improve pharmaceutical care quality, standardize service content and delivery models, and fully leverage pharmacists' expertise in rare disease management, the Rare Disease Drug Committee of the Chinese Pharmaceutical Association organized a panel of experts to develop the Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025). This consensus aims to establish a standardized pharmaceutical care framework tailored to China's healthcare context, ensuring the accessibility, appropriateness, efficacy, and safety of rare disease treatments.
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spelling doaj-art-66bd622caec04993b86e0c91fb2f8f522025-08-20T02:30:35ZzhoEditorial Office of Medical Journal of Peking Union Medical College HospitalXiehe Yixue Zazhi1674-90812025-05-0116365967210.12290/xhyxzz.2025-0252Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)Rare Disease Drug Committee of the Chinese Pharmaceutical AssociationIn recent years, with strong support from the national government, the diagnosis and treatment of rare diseases in China have achieved significant progress. However, only 5% of rare diseases worldwide have effective treatments, and poor drug accessibility remains a major challenge in the field of rare diseases, both in China and globally. Due to limited research data, insufficient clinical experience, and a lack of medication expertise, there is an urgent need for pharmacists to play an expanded role throughout the rare disease care process, alongside the establishment of systematic management and monitoring systems. To enhance the leading role of medical institutions in securing rare disease drug availability, improve pharmaceutical care quality, standardize service content and delivery models, and fully leverage pharmacists' expertise in rare disease management, the Rare Disease Drug Committee of the Chinese Pharmaceutical Association organized a panel of experts to develop the Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025). This consensus aims to establish a standardized pharmaceutical care framework tailored to China's healthcare context, ensuring the accessibility, appropriateness, efficacy, and safety of rare disease treatments.https://xhyxzz.pumch.cn/article/doi/10.12290/xhyxzz.2025-0252rare diseasesrare disease drugspharmaceutical carehospital
spellingShingle Rare Disease Drug Committee of the Chinese Pharmaceutical Association
Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
Xiehe Yixue Zazhi
rare diseases
rare disease drugs
pharmaceutical care
hospital
title Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
title_full Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
title_fullStr Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
title_full_unstemmed Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
title_short Expert Consensus on Rare Disease Pharmaceutical Care in Hospitals (2025)
title_sort expert consensus on rare disease pharmaceutical care in hospitals 2025
topic rare diseases
rare disease drugs
pharmaceutical care
hospital
url https://xhyxzz.pumch.cn/article/doi/10.12290/xhyxzz.2025-0252
work_keys_str_mv AT rarediseasedrugcommitteeofthechinesepharmaceuticalassociation expertconsensusonrarediseasepharmaceuticalcareinhospitals2025