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Monday, November 07, 2022 12:38:20 PM
Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases
"Drug sponsors submit a request for designation to the OOPD by presenting evidence that they are developing the drug for a rare disease and demonstrating the scientific rationale of the drug (clinical or preclinical evidence that establishes the medically plausible basis for the use of the drug in that rare disease) [19]...
....There were 5099 orphan drug applications designated between 1983 and 2019. As of December 31, 2019, 724 (14%) of these designations had at least one associated approval, and there were 878 total approvals. For first approvals, 35% occurred within two years of designation, 69% occurred within five years, and 92% occurred within ten years...
Of these orphan products, 59% (3010) were small molecules and 41% (2089) were biologics. This proportion stayed relatively constant when evaluated by decade. Treatments accounted for 92% (4678) of the designations, preventives 7% (332), and diagnostics 2% (89).......
....In just ten years since the last quantitative analysis performed by OOPD, designations and approvals for rare disease drugs have nearly tripled [21]. These recent increases in rare disease drug development have also been observed in the European Union (EU) and Japanese orphan drug programs, indicating that these are international trends [22,23,24]....
This increase in designations also appears to have translated into an increase in approvals, with a similar trend appearing to occur in the EU [4, 24]. However, it is important to note that this may occur on a substantial lag due to the timeline of development: orphan designations may be granted at any stage of the development process, from the preclinical phase up to before a marketing application is submitted [25, 26]. This limits the interpretability of the time from designation to approvals, as well as when designations may translate into approvals in the future. Regardless, the next decade may see a large increase in rare disease drug approvals, if this complementary relationship between designations and approvals persists..... "
https://ojrd.biomedcentral.com/articles/10.1186/s13023-021-01901-6#:~:text=There%20were%205099%20orphan%20drug,there%20were%20878%20total%20approvals.
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