The report for 2014 was released as part of efforts by the EC to make public information around the companies that have benefited from incentives under the regulation, as well as companies that have failed to comply.
In 2014, the Pediatric Committee (PDCO), which is an EMA (European Medicines Agency) committee responsible for assessing the content of pediatric investigation plans (PIP),adopted the highest number of positive opinions on final compliance checks so far - almost double compared to 2013 – which the EC says “will eventually lead to more marketing authorisations and more information on pediatric medicines.”
The proportion of pediatric trials approved (defined as any trial that includes at least one participant younger than 18 years of age) has increased to over 12% of all trials in 2014, whereas there is a general decrease in overall numbers of clinical trials, especially when compared to the years of 2006-2010, according to the European Clinical Trials Database.
The regulation also seems to be working in terms of increasing the number of pediatric trial participants. Since the implementation of the regulation, the number of pediatric study participants in trials has significantly increased to more than 150,000 in 2014, without a significant increase in the number of trials.
However, the EC notes these increases with the caveat that “it is difficult to describe any further trends as the data are heavily influenced by a limited number of trials that include very high numbers of children (e.g. for vaccines); the initiation of these trials in a given year may significantly skew the data, as shown by the wide fluctuation in patient numbers.”
In addition, the commission notes that in recent years the PDCO “increasingly implemented modelling and simulation as well as extrapolation strategies in PIPs, in order to not subject children to unnecessary trials.”
Under the regulation, sponsors can also seek scientific advice on pediatric development, and the number of scientific advice procedures including paediatric questions has increased steadily from the start of the implementation of the regulation. In 2014, 551 requests for scientific advice were submitted, of which 97 included paediatric development questions – which is a greater than 10% increase over 2013.
In addition, fewer applications for PIPs and waivers were submitted late compared to 2013, and the positive trend observed in the previous years continued in 2014.
Annual reports on deferrals indicated that many PIPs progressed without major difficulties, and in 2014, the number of applicants not submitting annual reports on deferred measures has significantly decreased.
Meanwhile, for the first time since the Pediatric regulation came into force, two orphan medicines have benefited from a two year extension of their market exclusivity in 2014, including Shire’s Xagrid and Novartis’ Tobi Podhaler.