Napoleon Bonaparte once said of China, “Let her sleep, for when she wakes, she will shake the world.” When it comes to rare diseases China has awoken and belongs to the top-10 countries based on scientific output. China doesn’t just stop at research.
China is quickly becoming a medical research powerhouse to reckon with. A recent literature search revealed that articles related to basic medical science and clinical research from China increased each year between 2000 and 2009 [Hu]. On average by 31% and 22%, respectively. In 2011, China was ranked fourth according to number of medicine-related publications in the SCImago Country Ranking (In 2000: rank 18). A truly remarkable achievement, although some concern has been expressed with regard to the quality and trustworthiness of the published research. China’s contribution to rare disease understanding is growing as well. A comparison of scientific output for 88 rare metabolic disorders and a dataset of 84 rare nervous system diseases between 1996-1998 and 2009-2011 revealed that China now belongs to the top-10 countries (see Figures below).
China’s growing role in (rare) medical research will certainly make a difference in our understanding of a number of rare diseases. However, to make a truly meaningful difference for the patient, disease understanding has to be translated into orphan drug development or some form of health care innovation. Therefore, it is important to know that China is not only making a difference in understanding disease, but is also focussing more and more on biopharmaceutical innovation [Rezzai]. A recent publication by Rezzai et al.revealed that China has built close to 20 high-tech parks with a life sciences component in the last fifteen years. R&D expenditures as a portion of its gross domestic product has doubled between 2002 and 2007 (~ US$102 billion in purchasing power parity). Finally, between 1999 and 2007 approximately US$200 Million of public funds was devoted to financing biotechnology companies. Perhaps China’s innovation power is best exemplified by SiBiono’s innovative gene therapy product, marketed as Gendicine for treatment of head and neck cancer since 2009.
The combined progress in research and innovation provides China the means to not only generate disease knowledge, but at the same time capitalize on this newly acquired knowledge in drug development. The first “Made in China” orphan drug approved in the EU or US may become a reality soon.
Note on Methodology: In brief, PubMed was used to determine for every “SCImago” top-15 country the total scientific output. For every disease (prevalence 0,1-50/100,000) a PubMed search string according to Heemstra et al.  was prepared. Via concatenation search strings were combined into one PubMed search string.
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