An Evidence-based Evaluation on Orphan Drug Policies_Chinese Journal of Evidence-Based Medicine

Authors：

 CHENMin ^1,2 ,  ZHANGLing-li ^1,2 , LIYou-ping ³ , QUANShu-yan ^1,2,4

1. Department of Pharmacy/Evidence-Based Pharmacy Center, West China Second University Hospital, Sichuan University, Chengdu 610041, China;
2. Key Laboratory of Birth Defects and Related Diseases of Women and Children(Sichuan University), Ministry of Education, Chengdu 610041, China;
3. Chinese Evidence-Based Medicine Center, West China Hospital, Sichuan University, Chengdu 610041, China;
4. West China School of Pharmacy, Sichuan University, Chengdu 610041, China;

Corresponding author：

ZHANGLing-li, Email: zhlingli@sina.com

Keywords：

Drug policy; Rare disease; Orphan drug; Evidence-based evaluation

DOI：

10.7507/1672-2531.20150138

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Objective To learn the development and implementation of orphan drug policies, in order to provide decision-making references for the establishment of orphan drug policy according with China's national conditions. Methods We electronically searched databases including CBM, CNKI, VIP, EMbase, PubMed, Web of Knowledge, National Library of Medicine, CRD database, The Campbell Library, The Cochrane Library and the drug administration websites of USA, Canada, UK, Ireland, the Netherlands, Germany, Spain, France, Australia, New Zealand, China, India, South Korea, Japan, and South Africa to collect studies about orphan drug policy. The search date was up to February 2014. Two reviewers independently screened literature, and extracted data. Then, all included orphan drug policies were summarized and a comparative analysis was performed. Results A total of 110 studies were included. USA, Singapore, Japan, Australia, European Union, Chinese Taiwan and South Korea had introduced orphan drugs incentive policies. South Africa, India, Canada, New Zealand and Chinese Hongkong were producing orphan drugs policy frameworks. The main items of orphan drug policy included marketing exclusivity, tax incentives, technical assistance, grant funding, expedite approval process and prolong re-evaluated time. Conclusion In mainland China, there is no orphan disease management policy. China should establish specific organization and working procedures, promote orphan drug policy related legislative work, clarify the definition and prevalence of orphan diseases, provide incentive mechanism to promote the research and development of orphan drugs, provide enterprises to develop compensation mechanism to safeguard the rights and interests of patients, as well as establish patients register network platform to track the processes of the diseases.

Citation： CHENMin, ZHANGLing-li, LIYou-ping, QUANShu-yan. An Evidence-based Evaluation on Orphan Drug Policies. Chinese Journal of Evidence-Based Medicine, 2015, 15(7): 802-809. doi: 10.7507/1672-2531.20150138 Copy

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Chinese Journal of Evidence-Based Medicine

An Evidence-based Evaluation on Orphan Drug Policies

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