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

Abstract

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.

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

“…Therefore, further efforts are needed to address the issue of pharmaceutical treatment costs holistically [51].…”

Economic Burden of Major Diseases in China in 2013

“…The majority of researches on economic burden has mostly focused on a single disease or localized diseases (7)(8)(9)(10)(11), but an integrated study of multiple diseases is rare in China. Recent studies only estimated the economic burden of lung cancer (12), seasonal influenza (13), chronic diseases ( 14), children with asthma (15) and Alzheimer's disease (16) in China, and the economic burden of Alzheimer's disease in Zhejiang Province (17), hepatitis E-infected patients in Jiangsu Province (18) and rare diseases in Shanghai (19) etc. Therefore, we intend to estimate the overall EBD and then, to separately measure the EBD between urban and rural areas in China in order to provide evidence for relevant policy makers.…”

Demographics, in-hospital analysis, and prevalence of 33 rare diseases with effective treatment in Shanghai

“…Firstly, receiving an accurate diagnosis may be a long-drawn process driven by the lack testing modalities and existing knowledge and information due to the rarity of the condition. Having overcome the hurdle of accurate diagnosis, the patient may face the obstacle of lack of treatment, or if the treatment is not included in a government established health insurance scheme—a financially ruinous self-funded cost of treatment [ 4 ]. From governance and health care perspective, while certain regions are well-advanced in many aspects of rare diseases management, in many others there is a lack of comprehensive policy, or a national rare disease plan and legislative framework that is specifically aimed to address the complex web of issues surrounding rare diseases is still in development or yet to be fully implemented [ 5 ].…”

“…In our previous work [ 4 ], we examined the specifics of the economic burden and described the direct medical costs related to rare diseases in Shanghai. Herein we take a holistic view and focus on rare disease epidemiology in Shanghai.…”