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Volume 13 Issue 2
Mar.  2022
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Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, National Rare Diseases Committee. Expert Consensus on the Application of Multi-criteria Decision Analysis in Clinical Comprehensive Evaluation of Orphan Drugs(2022)[J]. Medical Journal of Peking Union Medical College Hospital, 2022, 13(2): 235-254. DOI: 10.12290/xhyxzz.2022-0013
Citation: Multi-disciplinary Team for Rare Diseases, Peking Union Medical College Hospital, National Rare Diseases Committee. Expert Consensus on the Application of Multi-criteria Decision Analysis in Clinical Comprehensive Evaluation of Orphan Drugs(2022)[J]. Medical Journal of Peking Union Medical College Hospital, 2022, 13(2): 235-254. DOI: 10.12290/xhyxzz.2022-0013
shu

Expert Consensus on the Application of Multi-criteria Decision Analysis in Clinical Comprehensive Evaluation of Orphan Drugs(2022)

Funds: 

Fundamental Research Funds for the Central Universities 3332021003

CAMS Innovation Fund for Medical Sciences CIFMS 2021-I2M-1-003

Youth Research Fund of Peking Union Medical College Hospital pumch201911755

More Information

  • Corresponding authors: ZHANG Bo1, 3, E-mail: zhangbopumch@163.com
    ZHANG Shuyang2, 3, E-mail: shuyangzhang103@163.com
    1. Department of Pharmacy, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
    2. Department of Cardiology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China
    3. State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China

  • Received Date: January 08, 2022
  • Accepted Date: February 24, 2022
  • Issue Publish Date: March 29, 2022
    Graphical Abstract
    • Abstract
  • Comprehensive drug evaluation is an important technical tool for decision-making on drug supply. Traditional health technology of evaluation provides methodological references for comprehensive clinical evalu-ation of drugs. However, there is a lack of sufficient data from clinical trials for orphan drugs. It is difficult to evaluate the clinical and economic value of orphan drugs with the criteria of ordinary drugs. Particularity of orphan drugs makes it difficult to use the evaluation methods of traditional health technology. Multi-criteria decision analysis explores the comprehensive value of drugs from various aspects. It promotes the transparency and scientific thinking of evidence-based decision-making, and improves the quality of decision-making, and thus provides a potential solution to the comprehensive clinical evaluation of orphan drugs. However, its application in the comprehensive evaluation of orphan drugs in China is still in its infancy and exploratory stage, and there is a lack of methodological guidelines. In order to improve the construction of a diversified system for comprehensive clinical evaluation of orphan drugs in China, the Multi-disciplinary Team for Rare Diseases of Peking Union Medical College Hospital and the National Rare Diseases Committee organized experts in related fields to formulate this consensus, aiming to provide methodological standards for comprehensive clinical evaluation of orphan drugs.
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    • References (55)
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