Europe – European Commission reviews impact of orphan, pediatric regulations

The European Commission on Tuesday published the results of a yearslong evaluation of the EU’s orphan and pediatric medicines regulations it says will be used to guide future legislative changes and shape the EU pharmaceutical strategy.
 
The more than 100-page evaluation reviews the positive impacts and shortcomings of the orphan regulation, Regulation (EC) No 141/2000, and the pediatric regulation, Regulation (EC) No 1901/2006, based on external studies and various consultations with stakeholders.
 
“The evaluation found that both regulations have fostered the development and availability of medicines for patients with rare diseases and for children,” the Commission writes. “Nevertheless, both regulations have not adequately managed to support the development in areas where the need for medicines is greatest. Products tend to be developed in certain more profitable therapeutic areas for which the number of available treatments is increasing.”

According to the report, 142 orphan medicines have been authorized in the EU since 2000, 131 of which are still on the market. While the Commission notes that the orphan regulation alone did not spur the development of most of those products, it estimates that 18-24 orphan medicines were developed as a result of the regulation and credits it with speeding access to patients by nine months on average…