Concerns

It has been suggested by independent observers that the impact of the Paediatric Regulation should be closely monitored to assess whether it meets the needs it originally set out to address (1).

Authors in the US have expressed concern about the effect of the US Pediatric Rule because the paediatric studies reported to FDA did not necessarily address the most urgent needs in paediatric drug development (6). In the US, some of the major areas that received attention in terms of paediatric drug development were antidepressants and mood stabilisers, lipid-lowering preparations, HIV antivirals, and nonsteroidal antiinflammatory and anti-rheumatic drugs. These areas were identified as having more in common with the largest commercial markets for adult medicines rather the needs of the paediatric population. In particular, the authors reported that among the drugs granted paediatric exclusivity, there were 5 out of the "Top 10" prescription drugs with the highest sales figures in North America in 2005, generating combined sales of $24.1 billion (6).

In its July report, EMA commented that several companies had failed to comply with the Paediatric Regulation (3). In 2011, the number of delayed PIP applications was 44, which was an improvement over the 65 noted in 2010. However, the length of delay was greater in 2011 than it had been the previous year. Out of the 44 delayed PIP applications, only 4 were considered to have a valid justification for the delay. Twelve applications did not include any justification at all. While noting these delays, it is unclear from the report what action the EMA intends to take now against the offending parties. Apart from repeating the requirements that a PIP must be submitted in a timely manner, no potential punishments are actually detailed. The regulators only hint that the Commission Regulation on financial penalties (Commission Regulation (EU) No 488/2012) may be a future deterrent.

Tackling the issues

European regulators are actively working to make progress on practical issues with respect to paediatric clinical trials. The setting up of the European Network of Paediatric Research at EMA (Enpr–EMA) in May 2010 was a key development (3, 7). Although it does not perform clinical trials, fund studies or research, or decide on areas for paediatric research, Enpr–EMA contributes to greater scientific understanding in the field and improves administrative competence at a European level. Enpr-EMA has members both inside and outside of the EU to represent various therapeutic areas, age groups and specific activities in paediatric medicine, such as pharmacovigilance. Enpr–EMA also holds annual workshops to discuss progress, with the last one being held in March 2012. At is 2012 meeting, Enpr–EMA discussions were still focused on how members could best collaborate and what approaches could be used to ensure representation from different stakeholders. Membership of the network is based on self assessment. According to the EMA's latest figures, 34 networks have submitted their self-assessment reports. At present, there are two networks that potentially fulfil all minimum criteria, but clarification over some issues is required before they can become members. Fourteen others still to meet the minimum qualifying criteria (3). The original deadline for networks to complete the self-assessment procedure was July 2010, suggesting that progress at Enpr–EMA is occurring slowly and it is too early to document an impact on paediatric development.