The revenue-generating potential of orphan drugs as great as for non-orphan drugs, even though patient populations for rare diseases are significantly smaller. : 6 By 2012, the market for orphan drugs was worth US$637 million, compared with US$638 million for a control group of non-orphan drugs. : 660 For the same decade the compound annual growth rate (CAGR) of the orphan drugs was an "impressive 25.8%, compared to only 20.1% for a matched control group of non-orphan drugs". Īccording to Drug Discovery Today, the years 2001 to 2011 were the "most productive period in the history of orphan drug development, in terms of average annual orphan drug designations and orphan drug approvals". Orphan Drug Act of 1983 (ODA) and similar acts in other regions of the world driven by "high-profile philanthropic funding".
Orphan drug registration#
clinical trials: 350 in the pipeline from research until registration Įffect on investment, sales and profit Īccording to Thomson Reuters in their 2012 publication "The Economic Power of Orphan Drugs", there has been increased investment in orphan drug research and development, partly due to the U.S. Number of orphan drugs in phase 2 trial: 231.Number of orphan drugs in clinical trials: 600.Cancer treatment was the indication in more than 30% of orphan drug trials. dominated development of orphan drugs, with more than 300 in clinical trials, followed by Europe. More than 60% of orphan drugs were biologics. Global statistics Īs of 2014, there were 281 marketed orphan drugs and more than 400 orphan-designated drugs in clinical trials. As of 2017, there was no official integration of the orphan drug programs between the FDA and EMA. EMA also qualifies a drug as orphan if – without incentives – it would be unlikely that marketing the drug in the EU would generate sufficient benefit for the affected people and for the drug manufacturer to justify the investment. 5.5 Transthyretin-related hereditary amyloidosisĪccording to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for the treatment, prevention or diagnosis of a rare disease or condition, which is one that affects less than 200,000 persons in the US" (which equates to approximately 6 cases per 10,000 population) "or meets cost recovery provisions of the act." In the European Union (EU), the European Medicines Agency (EMA) defines a drug as "orphan" if it is intended for the diagnosis, prevention or treatment of a life-threatening or chronically and seriously debilitating condition affecting not more than 5 in 10,000 EU people.5.4 Phospholipase 2G6-associated neurodegeneration.3 Effect on investment, sales and profit.
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