What do pharmaceutical companies look forin the rare disease field?

　　Rare diseases, also known as orphandiseases, are diseases with a very low incidence and a very small totalpopulation affected, and are characterized by a large number of species,genetic predominance, difficulty in diagnosis, seriousness, low treatability,and expensive treatment. Currently, more than 7,000 rare diseases have beenidentified worldwide, but only 10% of them have corresponding treatments.

　　Compared with other drugs, drugdevelopment institutions often lacked the motivation to develop rare drugs inthe past due to high R&D cost and narrow audience, making rare drugs"abandoned orphans", which is the reason why they are called"orphan drugs". However, today's huge unmet need is one of thedriving forces for drug companies to step up their efforts in rare diseases.

　　"For AstraZeneca, the potential ofrare diseases is that we feel that this is something that has enough of amission." Wang Lei, AstraZeneca's global executive vice president andpresident of international business and China, previously said in a mediainterview including a surging news reporter that the number of individualdiseases of rare diseases is rare, but all together is not rare, and many raredisease groups are children, the field is more worthy of attention.

　　Meng Lili Lian, chief expert of SichuanTianfu Health Industry Research Institute, said in an interview with Punch Newsthat the significant progress in physiology and pathology as well as the largeamount of research on rare diseases themselves have objectively provided muchbetter conditions and opportunities for rare disease drug development than inthe past, which is an important reason for many pharmaceutical companies to beamong this field of rare disease drugs. At the same time, the issue of raredisease drugs has become a concern for the government, and relevant investmentand resources are tilted to this field.

　　For companies, the "marketexclusivity period" for new drugs for rare diseases is a more directpolicy benefit.

　　In May 2022, the State DrugAdministration issued the "Regulations of the People's Republic of Chinaon the Implementation of the Drug Administration Law (Draft Revision for PublicComments)", which clearly encourages the development and innovation ofdrugs for children and rare diseases, and grants a market exclusivity period ofno more than 12 months and 7 years for the first approved new varieties, dosageforms and specifications for children and approved new drugs for rare diseasesrespectively.

　　As mentioned in its semi-annual report,Hengrui Pharmaceutical has several products in the field of pediatric drugs andrare disease drugs, and will actively communicate with regulators on drugdevelopment plans during the R&D process to improve the efficiency ofclinical development of related drugs, and strive to enjoy the support ofsubsequent market exclusivity period and other preferential policies whileaccelerating the solution of patients' unmet treatment needs.

　　Of course, the investment in rare diseaseshas also brought the drug company performance returns. 2021 financial reportshows that AstraZeneca's rare disease area contributed a total of $3.07billion, accounting for about 8% of the year's revenue share. Takeda's fiscalyear 2021 data shows that its revenue contribution from rare disease revenue of611.2 billion yen is in second place. Beihai Kosei's half-yearly report showedthat it earned 34.728 million yen in the first half of the year, up 184.8%year-on-year, one reason being the increase in sales of already approved raredisease drugs.