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November 27, 2018updated 30 Nov 2018 5:10pm

Orphan drugs in 2017: an analysis of clinical trials

In 2017, the FDA approved 80 drugs with orphan drug designation. GlobalData’s Pharma Intelligence Center database covers the clinical trials for those 80 drugs, including a key analysis of the top 10 drugs with the most clinical trials, by therapy area, trial status, and trial phase.

By GlobalData Healthcare

In 2017, the FDA approved 80 drugs with orphan drug designation. GlobalData’s Pharma Intelligence Center database covers the clinical trials for those 80 drugs, including a key analysis of the top 10 drugs with the most clinical trials, by therapy area, trial status, and trial phase.

Orphan drugs in 2017

Clinical trials for the top 10 drugs occupy only three therapy areas, with majority of them in Infectious Disease (70.4%), followed by Oncology (23.8%) and Hormonal Disorders (5.8%) (Figure 1). Also, over 57% of these clinical trials have been completed, with 30.2% ongoing, 7.3% planned, and 5.5% terminated (Figure 2). At least 20% of trials are in each phase between Phase I and Phase IV, while only 0.8% are in Phase 0 (Figure 3).

Figure 1:Top 10 orphan drug designation drugs with most clinical trials, by therapy area

Source: GlobalData, Pharma Intelligence Center

Figure 2: Top 10 orphan drug designation drugs with most clinical trials, by trial status

Source: GlobalData, Pharma Intelligence Center

Figure 3: Top 10 orphan drug designation drugs with most clinical trials, by trial phase

Source: GlobalData, Pharma Intelligence Center

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