A proposed definition of rare diseases for China: from the perspective of return on investment in new orphan drugs

Orphanet Journal of Rare Diseases

Jinxiang Han¹  Yazhou Cui¹ 

[1] Shandong Academy of Medical Science, Shandong Medical Biotechnological Center, Key Laboratory for Biotech Drugs of the Ministry of Health, Jinan 250062, China

关键词: Orphan drug;    China;    Definition;    Rare diseases;   

Others  :  1138510
DOI  :  10.1186/s13023-015-0241-x

 received in 2014-09-26, accepted in 2015-02-13,  发布年份 2015

【 摘 要 】

A prevalence threshold to define rare diseases is needed for orphan drug designation. Here, we propose a bottom-up approach to defining rare diseases for China, based on the minimum number of patients needed for the industry to make a reasonable profit on a new drug. To obtain this patient population size, we considered three factors: (1) the industry research and development cost per new drug; (2) the sales per new drug to recoup its research and development costs and generate profit; (3) the highest affordable cost for one patient’s treatment in a given healthcare system. Using this model, we estimate that, with the current level of innovation in the pharmaceutical industry in China, between 300,000 and 500,000 patients could be a reference threshold to define rare diseases. Compared with other proposals, this evidence-based definition is more useful for designing rare diseases and orphan drug policies for China.

【 授权许可】

   
2015 Cui and Han; licensee BioMed Central.

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