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Figure 16    How the biotech mega research institute can facilitate the development of an industrial cluster—
                        rare diseases as a case in point

                                                               Opportunities for
                                                            emerging technologies
                        Huge market opportunities                                                  Policy incentives and
                           with obvious gaps                                                       expedited approval


                              USD
                          194 billions                      Biotech mega research institute
                                                                           Attractive to
                                                       Commercialisation   enterprises                “List of Overseas
                                 5~7%                     of advanced      relocating               New Drugs Catering
                                                       research outcomes                               to Clinical
                                                                           to the Loop
                                                                                                      Urgent Needs”
                                                          Leading transnational pharmaceutical
                                    CNY
                                60-90 billions              giants such as Biogen and GSK
                                                                                                involving a number of orphan drugs
            Source: Boston Consulting Group

            Capitalising on advanced opportunities: the institute should   Taking rare diseases as an example (see Figure 16), studies have
            focus its attention on advanced scientific enquiries and resolving   identified enormous market opportunities, with the global market
            major challenges in medicine, such as emerging infectious   estimated to reach roughly USD 194 billions, and the Chinese
                                                                                                        51
            diseases like novel coronavirus diseases, common age-related   market at CNY 60–90 billions, by 2030.  Meanwhile, effective
            diseases like Alzheimer’s, rare diseases like albinism, and new   treatments exist for fewer than 5% of rare diseases only, suggesting
                                     50
            fields of study like epigenetics,  as well as disruptive technologies   huge unfulfilled demand. Consequently, many governments have
            such as next generation testing technologies, next generation   introduced policy incentives, including research grants, expedited
            genetic manipulation technologies and synthetic biology technologies.



            50   Epigenetics is the study of the relationships between phenotypes, diseases and genes, which can help predict the impact genetic defects may have for the invention of more targeted
              prevention and treatments.
            51   Statistics by the Boston Consulting Group show that the cumulative transactions made by the top 20 pharmaceutical companies of the world in the field of rare diseases amount to USD 218
              billions in 2020. Established pharmaceutical giants have been proactive in acquiring companies specialised in rare diseases: in 2018, Takeda Pharmaceuticals acquired rare disease giant
              Shire, Sanofi acquired Bioverative to gain a foothold in haemophilia, and Novartis acquired biotech company AveXis with expertise in gene therapies; in 2019, Roche acquired gene therapy
              pioneer Spark Therapeutics; in December 2020, AstraZeneca acquired rare disease giant Alexion Pharmaceuticals.
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