title: The availability and affordability of orphan drugs for rare diseases in China.
abstract: BACKGROUND
  Orphan drugs are intended to treat, prevent or diagnose rare diseases. In recent years, China healthcare policy makers and patients have become increasingly concerned about orphan drug issues. However, very few studies have assessed the availability and affordability of orphan drugs for rare diseases in China. The aim of this study was to provide an overview of the availability and affordability of orphan drugs in China and to make suggestions to improve patient access to orphan drugs.
  
  
  METHODS
  Two components of the availability of orphan drugs were examined. Market availability was assessed by the extent to which orphan drugs were marketed in China with a comparison to orphan drugs in international markets, such as the U.S., EU and Japan. We conducted surveys and collected data from 24 tertiary public hospitals in China to measure hospital-level availability of orphan drugs. The affordability of orphan drugs was calculated using hospital dispensary prices and was expressed as days of average daily income required for the cost of a course of treatment. Affordability was also analyzed under the Chinese basic medical insurance system.
  
  
  RESULTS
  Orphan drugs approved in the U.S., EU and Japan had 37.8%, 24.6% and 52.4% market availability in China, respectively. Median availability of 31 orphan drugs surveyed at the 24 tertiary public hospitals was 20.8% (very low). Within a periodic treatment course, the average treatment cost of 23 orphan drugs is approximately 4, 843. 5 USD, which equates to 505.6 days of per capita net income for an urban resident with a middle income (187.4 days for a high-income urban resident) or 1,582.8 days's income for a rural resident with a middle income (657.2 days for a high-income rural resident). Except for homoharringtonine, 22 orphan drugs for 14 rare diseases were unaffordable for the most of residents in China. With 5% out-of-pocket expenses, only three generics could be afforded by middle-income residents, whereas seven drugs for high-income urban residents.
  
  
  CONCLUSIONS
  The Chinese government can take more responsibility for improving the availability and affordability of orphan drugs through setting up incentive policies and public platforms for sharing of orphan drug information. Control of the high price of orphan drugs, combined with a joint funding model from both government and private enterprise can efficiently reduce the economic burden of affected patients in China.
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. shwgong@hust.edu.cn.
authors.forename: Shiwei
authors.initials: S
authors.lastname: Gong
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. wyxiao1028@126.com.
authors.forename: Yingxiao
authors.initials: Y
authors.lastname: Wang
authors.affiliation: Department of Pharmaceutical Systems and Policy, School of Pharmacy, West Virginia University, Morgantown, WV, USA. pan.lucy@gmail.com.
authors.forename: Xiaoyun
authors.initials: X
authors.lastname: Pan
authors.affiliation: School of Health and Medicine Management, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, Hubei, China. zhangliang@mails.tjmu.edu.cn.
authors.forename: Liang
authors.initials: L
authors.lastname: Zhang
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. hys19810612@sohu.com.
authors.forename: Rui
authors.initials: R
authors.lastname: Huang
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. wyzcx@126.com.
authors.forename: Xin
authors.initials: X
authors.lastname: Chen
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. 18971627262@163.com.
authors.forename: Juanjuan
authors.initials: J
authors.lastname: Hu
authors.affiliation: Department of Pharmacy Business and Administration, School of Pharmacy, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, 430030, Hubei, China. xuyitjmu@hust.edu.cn.
authors.forename: Yi
authors.initials: Y
authors.lastname: Xu
authors.affiliation: Department of Endocrinology, Institute of Geriatric medicine, Liyuan Hospital, Tongji Medical College of Huazhong University of Science and Technology, Wuhan, Hubei, China. jinsi@hust.edu.cn.
authors.forename: Si
authors.initials: S
authors.lastname: Jin
mesh_terms: D002681:China; D005102:Health Expenditures; D006801:Humans; D009965:Orphan Drug Production / Q000706:statistics & numerical data*; D017149:Private Sector; D017150:Public Sector; D035583:Rare Diseases