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    Home > Active Ingredient News > Drugs Articles > Double the growth rate of orphan drug market: Top 10 product shuffle new base medicine will become the "leader"

    Double the growth rate of orphan drug market: Top 10 product shuffle new base medicine will become the "leader"

    • Last Update: 2017-03-31
    • Source: Internet
    • Author: User
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    Source: pharmaceutical economic news 2017-03-31 pharmaceutical industry's growing interest in rare diseases seems to have no sign of abating, and there are good reasons for that Recently, a new orphan drug research report published by evaluatepharma company found that in recent years, the pharmaceutical industry has been increasing investment in research and development of rare diseases, resulting in the situation that the share of orphan drugs in the total global drug sales has been increasing The main factors driving this change are that the regulatory environment for orphan drugs is constantly improving, and the advantages of exclusive marketing rights and pricing make such drugs more profitable than non orphan drugs It is worth noting that the compound growth rate of orphan drugs in the top 25 non tumor areas is expected to reach 18% in the next six years, which is almost three times the market average Esbrit and NovoSeven may fail in the list of best-selling orphan drugs Evaluatepharma, a new list of spinraza and exondys 51, points out that in 2016, orphan drugs generated a total of US $114 billion in sales, accounting for 16.4% of the global non generic drug market By 2022, this figure will rise to US $209 billion, accounting for 21.4% of the total drug market In other words, in just 10 years, the share of orphan drugs in the drug market will double If these sales forecasts are fulfilled as scheduled, the compound annual growth rate of orphan drugs will reach 11.1% in the next six years, about twice that of non orphan drugs Lisa Urquhart, editor and report writer of evaluatepharma, said that small biotech companies have a significant presence in the treatment of rare diseases, but in this year's report, large pharmaceutical companies have regained their dominance in this area The definition of orphan drugs used in the report is that they are first approved for a rare disease or for more than one indication, but they account for more than 25% of the sales of orphan indications In a narrow sense (excluding oncology, only those drugs that have long been used to treat rare and life-threatening diseases), by 2022, the top 25 orphan drugs will generate a total sales of 37.4 billion US dollars, accounting for about 3.8% of the global drug sales Of course, this data does not include blockbuster drugs with multiple approved therapeutic uses, because most of them are not for rare diseases It is appropriate to consider orphan drugs in a narrower scope, because it can better understand the overall cost of these high-cost orphan drugs in treating diseases such as cystic fibrosis and rare lysosomal diseases So far, there has been little or no pricing pressure on orphan drugs in these areas, which account for at least a single digit percentage of global drug sales It is worth noting, however, that the market sometimes faces resistance from drug payers, probably because they spend a lot of money on a small number of patients Soliris, a super orphan drug developed by alexion, is still a major product in this field Soliris's sales reached $3.7 billion, ranking 27th on the list of all pharmaceutical products With the drug expected to be approved for treatment of generalized myasthenia gravis, soliris sales are expected to rise to $5.1 billion by 2022 Based on this, evaluatepharma predicts that soliris will become the 9th best-selling drug in the pharmaceutical industry Comparing the 2015 orphan drug report of evaluatepharma, we can find that the ranking of top orphan drugs has also changed dramatically For example, according to the previously generally agreed forecast data of the market, by 2020, Roche and Novo Nordisk will both be at the forefront of the drugs, espriet and novo seven But now, they are likely to disappear from the 2022 ranking, probably because of the competitive changes in the indications they are targeting In recent months, in the field of orphan drugs, Biogen and Sarepta have successively launched spinraza, which is used to treat spinal muscular atrophy, and exondys 51, which is used to treat DMD The former is expected to be among the top 10 orphan drugs by 2020, while the latter will be among the top 30 However, from the current situation, in the United States, drug payers have taken measures to restrict the use of these two drugs BMS, Novartis, Roche and Johnson & Johnson, which lead the orphan drug market, are closely followed by reports that celgene is expected to surpass all competitors in orphan drug sales by 2022, thanks to its sales of Revlimid, a blood cancer drug Sales of the drug are expected to increase from nearly $7 billion in 2016 to more than $13.5 billion in 2022 As early as 2005, the drug has been approved for orphan indications, used to treat myelodysplastic syndrome Since then, it has been approved for orphan indications such as non Hodgkin's lymphoma and multiple myeloma In addition, Bristol Myers Squibb, Novartis, Roche and Johnson & Johnson will follow the new base medicine in this ranking list In the report, evaluatepharma predicted that opdivo, Bristol Myers Squibb's checkpoint inhibitor, would become the best-selling orphan drug in the European market in 2022 Last year, orphan drugs cost an average of $140443 per patient, compared with $27576 for non orphan drugs Using median prices, evaluatepharma analysts found that in 2016, each patient spent 5.5 times more on orphan drugs than on non orphan drugs Although there are strict definitions, orphan drugs are still one of the most popular areas of drug development In terms of public policy, the legal framework for them is generally considered to be a great success However, the risk in the future is that orphan drugs may soon face the same pricing pressure common to non orphan drugs The report comes at a time when US Senator Charles Grassley has promised to study the US orphan drug law The orphan drugs act, enacted in 1983, provides development impetus and exclusive protection for pharmaceutical companies willing to participate in the treatment of rare diseases, but Grassley said the "unexpected use" of the law may cost taxpayers a lot  
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