How Far Have We Come? Challenges to Orphan Drug Access in China, 2011-2017

Guan, Xiaodong; Zhang, Jingyuan; Man, Chunxia; Ni, Bingyu; Shi, Luwen

doi:10.1016/j.xphs.2019.01.012

Cited by 17 publications

(28 citation statements)

References 31 publications

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“…The low availability of anticancer medicines is likely multifactorial, such as inadequate investment in research and development [38], insufficient incentive on maintaining stocks and inefficient procurement systems [47]. Our findings are contrary to the research in Pakistan that showed OBs (52.5%) were more available than LPGs (28.1%) in both public and private facilities [39].…”

Section: Discussioncontrasting

confidence: 80%

“…Most surveys focused on the essential medicines for adults [24,25,26,27,28] and children [29,30,31,32]. Some others focused on medicines for cardiovascular patients [33,34], antidiabetics [35,36], and orphans [37,38]. A few studies have been conducted to evaluate the cost, availability, and affordability of anticancer medicines [39,40,41], but did not cover China.…”

Section: Introductionmentioning

confidence: 99%

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Availability, Price and Affordability of Anticancer Medicines: Evidence from Two Cross-Sectional Surveys in the Jiangsu Province, China

Zhu

Wang

Sun

et al. 2019

IJERPH

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Objectives: With the increasing incidence of cancer, poor access to affordable anticancer medicines has been a serious public health problem in China. To help address this issue, we assessed the availability, price and affordability of pharmacotherapy for cancer in public hospitals in the Jiangsu Province, China. Methods: In 2012 and 2016, anticancer medicine availability and price information in the capital and five other cities was collected. A total of six cancer care hospitals, 26 tertiary general hospitals and 28 secondary general hospitals were sampled, using an adaptation of the World Health Organization/Health Action International methodology. Data was collected for the anticancer medicines in stock at the time of the surveys. Prices were expressed as inflation-adjusted median unit prices (MUPs). Medicine was affordable if the overall cost of all the prescribed anticancer medicines was less than 20% of the household’s capacity to pay. We used generalized estimating equations to estimate the significance of differences in availability from 2012 to 2016 and the Wilcoxon rank test to estimate the significance of differences in MUPs. Multivariate logistic regression was computed to measure predictors of affordability. Results: From 2012 to 2016 there was a significant decrease in the mean availability of originator brands (OBs) (from 7.79% to 5.71%, p = 0.012) and lowest-priced generics (LPGs) (36.29% to 32.67%, p = 0.009). The mean availability of anticancer medicines in secondary general hospitals was significantly lower than the cancer care, as well as in tertiary general hospitals. The MUPs of OBs (difference: −21.29%, p < 0.01) and their LPGs (−22.63%, p < 0.01) decreased significantly from 2012 to 2016. The OBs (16.67%) of all the anticancer medicines were found to be less affordable than LPGs (34.62% for urban residents and 30.77% for rural residents); their affordability varied among the different income regions. From 2012 to 2016, the proportion of LPGs with low availability and low affordability dropped from 30.77% to 19.23% in urban areas and 34.62% to 26.92% in rural areas, respectively. Generic substitution and medicine covered by basic medical insurance are factors facilitating affordability. Conclusion: There were concerning decreases in the availability of anticancer medicines in 2016 from already low availability in 2012. Anticancer medicines were more affordable for the patients in high-income regions than the patients in low-income regions. Governments should consider using their bargaining power to reduce procurement prices and abolish taxes on anticancer medicines. Policy should focus on the special health insurance plan for low-income patients with cancer. The goal of drug policy should ensure that first-line generic drugs are available for cancer patients and preferentially prescribed.

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Section: Discussioncontrasting

confidence: 80%

Section: Introductionmentioning

confidence: 99%

Availability, Price and Affordability of Anticancer Medicines: Evidence from Two Cross-Sectional Surveys in the Jiangsu Province, China

Zhu

Wang

Sun

et al. 2019

IJERPH

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“…Several hindering factors contributing to the low availability of orphan drugs in China include the low market availability of orphan drugs in China [17,54]. Compared with the earliest launch time globally, the average delay in the market authorization of orphan drugs for rare diseases in China was 7.7-9 years [17,54]. Imiglucerase received China's marketing authorization in 2008 [17].…”

Section: Discussionmentioning

confidence: 99%

Analysis of the Situation of Unmet Health Care Need Among Extremely Rare Disease of Gaucher

Shan

et al. 2020

Preprint

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BackgroundThe diagnosis and health care of patients with rare diseases present a tremendous challenge worldwide. This study estimated the health service utilization, cost of illness, and patients with Gaucher disease (GD)’s/caregivers’ health-related quality of life in China.MethodAn online retrospective survey of patients with GD and their caregivers was conducted during May-June 2018. Socio-demographic, health service utilization, disease-related expenses, social support, sleep quality (Pittsburgh Sleep Quality Index [PSQI]), and the Short Form Health Survey (SF-36) were investigated. Using self-reported information, we estimated the annual cost of illness, including direct medical, direct non-medical, and indirect medical costs. ResultsForty patients and their 49 caregivers were surveyed. The patients’ onset age of GD was 9.3±10.9; their disease course was 3.5±3.1 years. 21 (42.9%) patients had ≥2 caregivers, but 35 (71.4%) caregivers reported have no experience as a caregiver. 79.6% caregivers have stopped working, and 87.8% changed weekly working schedule. Before final diagnosis, patients visited 3.9±3.1 (max=20) hospitals and took 1.2±1.7 (max=6.6) years for confirmed diagnosis. On average, 5.0±9.6 misdiagnoses occurred, and the per-patient diagnoses cost was USD ($) 7,576. After GD confirmation, 8 (16.3%) patients received no treatment, 40 (81.6%) received pharmacotherapy, 10 (20.4%) received surgery, 38 (77.6%) received outpatient service (8.8±9.1 times/annually), and 37 (77.5%) received inpatient service (4.0±3.5 times/annually). Annual per-patient cost was USD ($) 49,925 (95% confidence interval: 29,178, 70,672). Average direct medical cost was $41,816, including pharmaceutical ($29,908), inpatient ($7,451), and outpatient ($1,838). Productivity loss per-caregiver was $1,980, and their Zarit Burden Inventory score was moderate-severe (48.6±19.6). Both patients/caregivers reported lower social support (32.4±7.4, 34.9±7.6), two times higher PSQI (7.9±2.9, 8.7±3.6), and half lower SF-36 (41.3±18.6, 46.5±19.3) than those reported for healthy Chinese individuals.ConclusionThis study finds out that fill the unmet need for extremely rare diseases is very challenging. The high misdiagnosis rate, together with delayed diagnosis, substantial costs, and deteriorated health-related quality of life of GD patients as well as their heavy care burden, calls for extreme attention from policymakers in China. Further efforts of government and society are urgently demanded, including pharmaceutical reimbursement, screening newborns, developing precise diagnostic tools, and training doctors.

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“…Since 2011, drugs for Pompeii disease, Gaucher’s disease, mucopolysaccharidosis, and Fabry disease have been covered by the Children’s Hospitalization Fund in Shanghai [24]. Additionally, further efforts will be needed to address holistically the issue of drug treatment costs which will continue to impose significant economic burden [25] on Chinese patients, families, and society in general.…”

Section: Discussionmentioning

confidence: 99%

Analysis of economic burden and its associated factors of twenty-three rare diseases in Shanghai

Cai

Yang²,

Genchev

et al. 2019

Orphanet J Rare Dis

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BackgroundIt is estimated that at present there are over 10 million rare disease patients in China. Recently an increased focus from policy perspective has been placed on rare diseases management. Improved disease definitions and the releases of local and national rare disease lists are some of the steps taken already. Despite these developments, few Chinese rare disease-related epidemiology and economic studies exist, thus hindering assessment of the true burden of rare diseases. For a rare disease with an effective treatment, this is a particularly important aspect due to the often-high cost associated.ObjectiveThe goal of this study is to address the data scarcity on the subject of rare diseases economic impact in China. We aim to address an existing knowledge gap and to provide a timely analysis of the economic burden of 23 rare diseases in Shanghai, China.MethodsWe utilized the data from the Health Information Exchange system of Shanghai and employed statistical modeling to analyze the economic burden of rare diseases with an effective treatment in Shanghai.ResultsFirst, we described the actual direct medical expenditure and analyzed its associated factors. Second, we found age, disease type, number of complications, and payment type were significantly associated with rare disease medical direct costs. Third, a generalized linear model was employed to estimate the annual direct cost. The mean direct medical cost was estimated as ¥9588 (US$1521) for inpatients and ¥1060 (US$168) for outpatients, and was over ¥15 million (~US$2.4 million) per year overall.ConclusionOur study is one of the first quantifying the economic burden of an extensive set of rare diseases in Shanghai and China. Our results can serve to inform healthcare-focused policy making, contribute to the increase of public awareness, and incentivize development of rare-disease strategies and treatments specific to the Chinese context.Electronic supplementary materialThe online version of this article (10.1186/s13023-019-1168-4) contains supplementary material, which is available to authorized users.

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How Far Have We Come? Challenges to Orphan Drug Access in China, 2011-2017

Cited by 17 publications

References 31 publications

Availability, Price and Affordability of Anticancer Medicines: Evidence from Two Cross-Sectional Surveys in the Jiangsu Province, China

Availability, Price and Affordability of Anticancer Medicines: Evidence from Two Cross-Sectional Surveys in the Jiangsu Province, China

Analysis of the Situation of Unmet Health Care Need Among Extremely Rare Disease of Gaucher

Analysis of economic burden and its associated factors of twenty-three rare diseases in Shanghai

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