2019 orphan drug report: the global market will reach 16 trillion yuan in 2024, and the new base will become the leader

Orphan drug (Ophan Drug) is a kind of medicine used for prevention, treatment and diagnosis of rare diseases Rare diseases are a general term for diseases with very low incidence rate, also known as orphan diseases According to the definition of the World Health Organization, diseases with a population of 0.65 ‰ - 1 ‰ of the total population can be defined as rare diseases However, according to the situation of our country, there are some differences in the recognition standards of rare diseases For example, the United States is defined as a disease with fewer than 200 thousand people; the European definition is a disease with a incidence rate of less than 1/2000 There is no clear definition of rare diseases in China However, the general consensus is that adults with a prevalence rate below 1/500000 and a disease incidence rate below 1/10000 in neonates can be defined as a rare disease It is estimated that as many as 7000 diseases have been defined as rare diseases, of which only about 400 rare diseases have corresponding treatment drugs approved Globally, the total number of patients with rare diseases has reached 300 million Evaluate Pharma, a pharmaceutical market research organization, recently released the report on orphan drugs in 2019 According to the report, scientific progress will boost the rapid growth of orphan drug market in the next six years (2019-2024) In addition to recent breakthrough cell and gene therapy products (such as yescarta and luxturna), many "once and for all" therapies for rare diseases will also enter the market, including zolgensma of Novartis and lentiglobin of Bluebird The explosive development of new technology makes its high price more concerned In addition, large pharmaceutical companies will continue to consolidate the position of orphan drug market The following are the specific contents of the report: 1 The global orphan drug market will grow rapidly, reaching US $242 billion in 2024 The report points out that in 2018, the global orphan drug market will be US $131 billion In the next six years (2019-2024), the market will grow rapidly at an annual compound growth rate of 12.3%, twice the growth rate of the non orphan drug market (6%) in the same period By 2024, it will reach US $242 billion (about RMB 1629.749 billion), and its share in the prescription drug market will break through 20% for the first time, reaching 20.3% 2 The cost of orphan drug treatment is 4.5 times higher than that of non orphan drugs The report points out that the strong growth of orphan drug market shows that the industry continues to invest in the favorable market of rare disease groups In addition, despite the increasing pricing pressure in major markets such as the United States, orphan drug products can still obtain a lucrative pricing premium Of the top 100 orphan drugs in sales in 2018, the average cost per patient per year was $150854, compared with $33654 for non orphan drugs However, in the past four years, the median price difference between orphan drugs and non orphan drugs has decreased by nearly 50%, indicating that the difference in treatment cost between two types of drugs is decreasing 3 Orphan drug products generate significantly higher income per patient than non orphan drugs report that drug revenue per patient increases as the number of patients decreases In 2018, a total of 10 orphan drugs earned more than $200000 per patient, including 4 for blood diseases and 2 for respiratory diseases In 2018, soliris and naglazyme were the top two orphan drugs with the highest income, each with more than $5 million per patient per year, although the two drugs had the least treatment Among them, naglazyme treated less than 200 patients and soliris treated less than 3000 in 2018 By contrast, Amgen's neulasta, a whitening drug, had more than 160000 people treated in 2018, but generated only $25000 in revenue per patient 4 Orphan drug market is more fragmented: in 2024, Xinji will become a leading enterprise in orphan drug market In 2024, global sales will reach 13.7 billion US dollars, accounting for 73% of its prescription drug sales, but its share in orphan drug market will drop by nearly 40% In 2024, orphan drug revenue from top 5 pharmaceutical companies is expected to exceed US $60 billion, accounting for about 26% of the whole orphan drug market, and the market share will decline by about 25% Key promoters of orphan drug market include AstraZeneca and Weicai, among which the penetration of AstraZeneca PARP inhibitor lynparza in the field of ovarian cancer will double the market share in the promotion period, and the penetration of Weicai lenvima in the field of liver cancer will also double the market share of the company In the future, another major feature of orphan drug market will be more fragmentation 5 Non oncology orphan drug market: Takeda and vertex will become the leaders in the non oncology orphan drug market Takeda will become the market leader in the acquisition of shire In 2024, the sales volume will reach 8.4 billion US dollars The company has about 30 drugs under research, covering multiple treatment fields Alexion, in fourth place, has only five drugs, focusing on hematology and musculoskeletal diseases Key drivers include GW pharmaceuticals, Bluebird biology, argenx and others 6 Hematology, central nervous system and respiration are the most important non oncology orphan drug treatment categories According to the report, in 2024, Bojian continued to lead the musculoskeletal category, vertex continued to lead the respiratory category, Pfizer replaced Tyva as the leader of the central nervous system category, and dermatology will become the smallest treatment category 7 Oncology orphan drug market: Xinji will become the leader in 2024, and its oncology orphan drug sales will reach US $12.6 billion Although the sales will remain stable in 2018-2024, the market share will drop by 50% Xinji, Aberdeen and Johnson & Johnson will occupy 30% of the market GlaxoSmithKline, rexahn and blueprint medicines have changed the most All three companies have no oncology orphan drug products in 2018, but they will occupy a certain market share in 2024, which means that the oncology orphan drug market will be more fragmented in the future 8 Top 10 orphan drug products in 2024: imbruvica and Revlimid will become the best-selling orphan drug products, accounting for 60% of the sales volume of top 10 pharmaceutical companies Other key drivers of the market include drugs such as vyndaqel and hemlibra 9 Evaluatepharma, the most valuable top 10 orphan drug product under research, analyzed the orphan drug under research (phase III to submitted) according to the net present value (NPV) The combined NPV of top 5 orphan drug under research was as high as $53 billion, of which vertex's triple therapy for cystic fibrosis ranked first In the research of orphan drugs, the development of drugs for oncology indications plays a leading role 10 Top 10 indications of orphan drugs: NHL, AML and pancreatic cancer are the first ones In the past 35 years (1983-2018), 70% of orphan drugs have been awarded the qualification of oncology Among them, NHL has awarded the most orphan drugs (270), followed by acute myeloid leukemia (220) and pancreatic cancer (189) In 2018, these three diseases were also awarded the most orphan drug qualifications, highlighting the significant unmet medical needs in these areas 1 Evaluatepharma orphan drug report 2019 2 The "one and done" era for orphan drugs: evaluate predictions 12% growth, driven by new technologies, but benefits go to big pharma