European Commission Issues Draft Pediatric Regulation Report
The European Commission is requesting public comment on its draft report to the European Parliament on the five years of experience since the Pediatric Regulation entered into force in January 2007. The Regulation was adopted in response to a lack of suitable, authorized medicinal products for children in the EU. Studies had shown that over 50% of the medicines used for children might not have been tested in pediatric populations, leaving providers not choice but to use off-label and unapproved products with the associated risks of inefficacy or adverse reactions.
To address this problem the Regulation established a system of requirements, rewards and incentives to ensure the development of drugs to meet the therapeutic needs of children.
To this end, the Regulation provides for:
- An expert pediatric committee within the European Medicines Agency;
- Submission of data on the use of the product in children in accordance with an agreed pediatric investigation plan when applying for marketing authorization;
- A system of waivers from the requirement for drugs unlikely to benefit children and a system of deferrals to ensure that medicines are only tested in children when it is safe to do so and that approval of medicines for adults is not delayed;
- A reward for compliance with the requirement in the form of a six-month extension to the supplementary protection certificate;
- For orphan medicines, an additional two years of market exclusivity added to the existing ten years awarded under the EU's Orphan Regulation, as a reward for compliance;
- Anew type of marketing authorization, the PUMA, which allows ten years of data protection for new studies on off-patent products;
- Measures to strengthen pharmacovigilance and maximize the impact of existing studies on medicines for children;
- An EU inventory of the therapeutic needs of children to focus the research, development and authorization of medicines;
- An EU network of investigators and trial centers to carry out the required research and development;
- A system of free scientific advice for the industry, provided by the EMA;
- A public database of pediatric studies;
- A provision on EU funding for research leading to the development and authorization of off-patent medicines for children.
According to the Commission's draft report, the Regulation, "Has paved the way for pediatric product development", with nearly 500 pediatric investigation plans approved by the EMA.
That development, however, remains secondary to the development of products for adult indications. The new Pediatric Use Marketing Authorization (PUMA) has not caught on, with only one PUMA submitted to date. The "hidden gem" in the law has been the requirement (Articles 45 and 46) that companies holding data on the safety or efficacy of authorized products in the paediatric population submit those studies to European regulatory authorities, which has led to the submission of about 18,000 studies conducted on 2,200 products.
The deadline for comments on the draft report is 28 November 2012.
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