Current progress in the management of rare diseases and orphan drugs in China

Gong, Shiwei; Jin, Si

doi:10.5582/irdr.2012.v1.2.45

Cited by 13 publications

(9 citation statements)

References 5 publications

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“…Due to the large number of rare disease-affected patients in China, the shortage of orphan drugs for these patients has become a major health problem. Patients with rare diseases in China face a double dilemma [ 8 ].…”

Section: Introductionmentioning

confidence: 99%

The availability and affordability of orphan drugs for rare diseases in China

Gong

Wang

Pan

et al. 2016

Orphanet J Rare Dis

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BackgroundOrphan 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.MethodsTwo 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.ResultsOrphan 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.ConclusionsThe 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.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-016-0392-4) contains supplementary material, which is available to authorized users.

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Section: Introductionmentioning

confidence: 99%

The availability and affordability of orphan drugs for rare diseases in China

Gong

Wang

Pan

et al. 2016

Orphanet J Rare Dis

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“…With a 5% patient co-pay, only three generic orphan drugs were considered affordable by middle-income Chinese patients [6]. Over 100 commercial health insurance companies exist in China for those who are able to afford an additional policy to supplement government insurance [12]. However, chronic rare diseases are not well defined for coverage, with approximately 10–15 rare diseases that are covered.…”

Section: Financial Considerations For Orphan Drug Developmentmentioning

confidence: 99%

Challenges in orphan drug development and regulatory policy in China

Cheng

Xie

2017

Orphanet J Rare Dis

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While regulatory policy is well defined for orphan drug development in the United States and Europe, rare disease policy in China is still evolving. Many Chinese patients currently pay out of pocket for international treatments that are not yet approved in China. The lack of a clear definition and therefore regulatory approval process for rare diseases has, until now, de-incentivized pharmaceutical companies to pursue rare disease drug development in China. In turn, many grassroots movements have begun to support rare disease patients and facilitate drug discovery through research. Recently, the Chinese FDA set new regulatory guidelines for drugs being developed in China, including an expedited review process for life-saving treatments. In this review, we discuss the effects of these new policy changes on and suggest potential solutions to innovate orphan drug development in China.

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“…The healthcare policies are getting more and more favorable for the rare disease treatment. The SFDA also initiated orphan drug green channel projects initiated in 2012 (Gong and Jin 2012). Orphan drug is granted to apply for fast tract review, sample size reduction, and clinical trial waiver, even though there is still lack of detailed description.…”

Section: Rare Disease/orphan Drugmentioning

confidence: 99%

Approaching China's Pharmaceutical Market

Lu¹

2015

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Current progress in the management of rare diseases and orphan drugs in China

Cited by 13 publications

References 5 publications

The availability and affordability of orphan drugs for rare diseases in China

The availability and affordability of orphan drugs for rare diseases in China

Challenges in orphan drug development and regulatory policy in China

Approaching China's Pharmaceutical Market

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