The impact of the orphan and paediatric regulations

The European Commission (EC) has published results from an evaluation of the EU orphan and paediatric regulations.

The outcomes of this evaluation will be used to guide future legislative changes and shape the EU’s pharmaceutical strategy. The evaluation report reviews the positive and negative impacts of the regulations, based on external studies and consultations with stakeholders.

Overall, the evaluation found that both regulations improved the development and availability of medicines for patients with rare diseases and children. However neither regulation 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.”

The report credits the orphan regulation with increasing the speed at which patients could access these medicines by 9 months on average. Furthermore, the orphan regulation is estimated to have added 210,000-440,000 quality-adjusted life years for patients in the EU. Orphan medicines received an additional 3.4 years of market exclusivity on average.

The paediatric regulation increased the amount of clinical trials involving children by almost 50% and led to more than 1000 paediatric investigation plans (PIPs). However, the EC feels that the greatest therapeutics needs of children were not completely addressed.

The report acknowledged that both regulations have led to increased costs for healthcare systems, however, the benefits to patients outweigh the costs.

Click here to access the full evaluation report.