The European Ombudsman has called on the European Medicines Agency (EMA) to increase the transparency of its procedures for ensuring that children can benefit from new medicines.
In October 2009, AstraZeneca and Takeda Global R&D Centre made a complaint to the Ombudsman, P Nikiforos Diamandourous, after the EMA required them to test how their heart-failure drug Atacand (candesartan) could be used to treat children. The firms claimed that this constituted discrimination against them by the Agency, because it had exempted two similar drugs made by other manufacturers - Novartis' Diovan (valsartan) and Merck & Co's Cozaar (losartan) - from the obligation to conduct such tests.
The complaint relates to the Paediatric Regulation, adopted by the European Union (EU) in 2006, before which more than 50% of medicines administered to children had not been authorised for paediatric use or undergone appropriate trials. Among the problems associated with this situation were an increased risk of adverse reactions and ineffective treatment through under-dosage.
The Paediatric Regulation requires drugmakers to carry out studies in accordance with an agreed Paediatric Investigation Plan (PIP). However, it also seeks to avoid unnecessary trials in children and, therefore, to ensure that research is only conducted to meet the therapeutic needs of child patients, it provides for the EMA, in certain cases, to waive the obligation for studies in children.
In the complaint submitted to the Ombudsman, AstraZeneca and Takeda argued that the EMA was wrong to reject their application to have the requirement for studies waived for Atacand, as it had already granted such a waiver to Diovan and Cozaar. All three drugs are angiotensin II type 1 receptor blockers (ARBs), and its decision relating to Atacand infringed the principle of equal treatment, it was not based on an objective and fair assessment and it was not reasoned, the firms argued.
Following his examination of the case, the Ombudsman considered that the EMA's decision to deny a waiver to Atacand was in fact one that it was entitled to reach, in substance. The Agency's Paediatric Committee had concluded that the limited size of the relevant paediatric population justified testing only one of the two products which could be the subject of a PIP for the indication of heart failure - Diovan and Atacand - and that, of the two, Atacand was the most appropriate product.
However, the Ombudsman also concluded that the EMA had failed to ensure adequate transparency of the process through which it reached its decisions and that, as a result, it had failed provide adequate reasons for those decisions.
Mr Diamandouros has told the Agency that it needs to make systemic changes "to avoid similar maladministration" in the future, making full documentation and disclosure of its assessments, and introducing relevant guidelines in this respect, and he has asked the EMA to respond to his recommendation by September 30.