Medical network - March 31, the pharmaceutical industry for rare diseases growing interest seems to be no signs of abating, and it has a good reason. EvaluatePharma company has published a new orphan drug study found that in recent years, increasing r&d investment in the pharmaceutical industry for rare diseases, the resulting situation is that the orphan drug occupied in the total amount of global drug sales share continue to improve. 

 

Driving this change are the main factors, orphan drug regulatory environment constantly improve, facing market exclusive rights and pricing advantages make the drugs to more profitable than the orphan drug. It is worth noting that the top 25 in the field of non tumor orphan drug compound growth rate over the next six years is expected to reach 18%, it is almost 3 times the market average. 

 

Best-selling orphan drug ranking changes 

 

Esbriet, NovoSeven, or will this Spinraza, Exondys 51 new list 

 

EvaluatePharma, according to a report in 2016 orphan drug combined generated sales of $114 billion, 16.4% of the generic drug markets around the world. By 2022, that figure will rise to $209 billion, in the pharmaceutical market share reached 21.4%. That is to say, in just 10 years, orphan drug in the drug market share will be doubled. 

 

 

 

If the sales forecast in time, then, orphan drug in the next six years, the compound annual growth rate will reach 11.1%, is about twice the orphan drug. 

 

EvaluatePharma editing and writing Urquhart (Lisa Urquhart), said the small biotech companies in the field of rare diseases treatment power to be reckoned with, but in this year's report, the big drug companies to occupy the dominant position in the field. 

 

The report with orphan drug is defined as the first drug approved for use in a rare disease, or more than one approved indications of drugs, but the drug in an indication of the purposes of the sales of more than 25%. 

 

In narrow perspective (not including oncology, involves only used to treat a rare, life-threatening disease for a long time those drugs), by 2022, the top 25 orphan drug will create combined sales of $37.4 billion, in the global drugs accounted for about 3.8% of sales. 

 

Of course, this figure does not include those with approved treatment for many purposes of blockbuster drugs, for most purposes and not for rare diseases. From a more narrow range to test is orphan drug should be carefully, because it can better understand these high-priced orphan drug used to treat rare lysosomal diseases such as cystic fibrosis and the overall cost of disease. 

 

So far, in the field of these treatments, orphan drug pricing pressure is small, or didn't, they account for at least the global pharmaceutical sales single-digit percentage. However, it is worth noting that the market pressure drug payer sometimes, the reason may be that, they are spent a lot of budget in a fraction of patients. 

 

 

 

Alexander elder brother pharmaceutical (Alexion) developed super orphan drug Soliris is still the main product of this field. Soliris sales of $3.7 billion, ranked 27th in all pharmaceutical products sales. As it is expected to be approved and used to treat myasthenia gravis body type, by 2022, Soliris sales is expected to rise to $5.1 billion. Accordingly, EvaluatePharma prediction, Soliris will become the pharmaceutical industry's ninth largest selling drugs. 

 

EvaluatePharma 2015, after comparing the orphan drug report can be found that the top ranking between orphan drug also appears surprising changes. According to the market, for example, of a common forecasting data, to 2020, Roche (Roche) and Novo Nordisk (Novo Nordisk) drug Esbriet and NovoSeven will be in the forefront, but now, they may disappear in the list of 2022, probably because their indication for competitive changes have taken place. 

 

In recent months, in the field of orphan drug, best health (Biogen) and Sarepta company launched a drug used to treat spinal muscular atrophy Spinraza and used for the treatment of duchenne muscular dystrophy (DMD) drug Exondys 51. The former is expected to enter the top 10 in 2020 orphan drug category, while the latter will enter the top 30 orphan drug act. However, from the point of the current situation, in the United States, the drug payer both of the two take the measures to limit the use of drugs. 

 

The new medicine lead the orphan drug market 

 

BMS, novartis, roche and Johnson &johnson, followed 

 

According to the report, by 2022, the new base medicine (its) is expected to transcend all competitors on orphan drug sales, thanks to its leukaemia drug sold by drug lenalidomide (Revlimid). The drug's sales are expected to be increased from nearly $7 billion in 2016 to more than $2022 in 13.5 billion. As early as in 2005, the drug has been approved indications for orphans, was used for the treatment of myelodysplastic syndrome. Since then, it has successively been approved for the treatment of non-hodgkin's lymphoma and multiple myeloma indications such as orphans. 

 

 

 

In addition, bristol-myers Squibb (Bristol Myers Squibb), Novartis Novartis, Roche (Roche) and Johnson & Johnson (Johnson & Johnson) will be ordered in this list after new medicine. EvaluatePharma forecast in the report, bristol-myers squibb Opdivo checkpoint inhibitor drugs in Europe will be 2022 best-selling orphan drug on the market. 

 

Last year, the orphan drug for each patient, on average, paid $140443, rather than the orphan drug data for $27576. By using the median price, EvaluatePharma analysts found that in 2016, each patient on orphan drug expense is 5.5 times higher than the orphan drug. 

 

Despite its strict definition, but still orphan drug is one of the most hot areas of drug development. From public policy considerations, in view of their legal framework is widely considered a huge success. However, the risk is that in the future, orphan drug may be in the near future will face the orphan drug common, the same pricing pressure. 

 

, "the publication of the report comes as U.S. senator Charles Grassley (Charles Grassley) promised to study of American orphan drug laws. The orphan drug act issued in 1983, for those who are willing to participate in rare diseases treatment pharmaceutical company provides development momentum and the sole protection, however, grassley said "unintended use" of the law may allow taxpayers paid a big price.