Fig 1.
Boxplots with medians of the number of patients studied prior to approval (non-orphan medicines, n = 322 versus orphan medicines, n = 176). Boxplots present the 50th percentile, i.e., the median value is given, with the interquartile range (25th and 75th percentiles) indicated by the box, the 2nd and 98th percentiles indicated by the horizontal bars of the whiskers, and outliers indicated by individual circles. The total number of patients studied (y-axis) is plotted on a logarithmic scale.
Fig 2.
Boxplots with medians of the number of patients studied prior to approval according to the type of Marketing authorisation: standard (n = 392), conditional (n = 78) or approval under exceptional circumstances (n = 28). Boxplots present the 50th percentile, i.e., the median value is given, with the interquartile range (25th and 75th percentiles) indicated by the box, the 2nd and 98th percentiles indicated by the horizontal bars of the whiskers, and outliers indicated by individual circles. The total number of patients studied (y-axis) is plotted on a logarithmic scale.
Fig 3.
Boxplots with medians of the number of patients studied prior to approval.
Results for non-orphan medicines (n = 322) are here presented by intended length of use of the products (short-term, n = 82, intermediate, n = 86, or chronic, n = 154). Boxplots present the 50th percentile, i.e., the median value is given, with the interquartile range (25th and 75th percentiles) indicated by the box, the 2nd and 98th percentiles indicated by the horizontal bars of the whiskers, and outliers indicated by individual circles. The total number of patients studied (y-axis) is plotted on a logarithmic scale.
Fig 4.
Boxplots with medians of the number of patients studied prior to approval.
Results for the orphan medicines (n = 176) are here presented by intended length of use of the products (short, n = 25, intermediate, n = 50, and chronic-term, n = 101). Boxplots present the 50th percentile, i.e., the median value is given, with the interquartile range (25th and 75th percentiles) indicated by the box, the 2nd and 98th percentiles indicated by the horizontal bars of the whiskers, and outliers indicated by individual circles. The total number of patients studied (y-axis) is plotted on a logarithmic scale.
Table 1.
Number (percent) of non-orphan and orphan medicines categorised according to total number of individuals studied prior to marketing authorisation.
Fig 5.
Medians of the total number of patients studied prior to approval for the period 2011-2023.
Table 2.
Number (percent) of non-orphan medicines categorised according to total number of individuals studied for 6 and 12 months (long term) prior to marketing authorisation.
Fig 6.
Scatterplot displaying the total number of patients studied prior to approval plotted against the number of patients studied long term (for 6 and 12 months) for chronic medication (for non-orphan medicines).
Reference lines are added to indicate the minimum criteria from the ICH E1 guideline: 1,000 patients in total and 300 and 100 patients studied for 6 and 12 months, respectively.
Table 3.
Number (percent) of orphan medicines categorised according to total number of individuals studied for 6 and 12 months (long term) prior to marketing authorisation.
Fig 7.
Scatterplot displaying the total number of patients studied prior to approval plotted against the number of patients studied long term (for 6 and 12 months) for chronic medication (for orphan medicines).
Reference lines are added to indicate the minimum criteria from the ICH E1 guideline: 1,000 patients in total and 300 and 100 patients studied for 6 and 12 months, respectively.