EMA Hopes Early Dialogue With Drugmakers Will Spur Pediatric Drug Development

Regulatory NewsRegulatory News | 16 June 2015 |  By 

The European Medicines Agency (EMA) is piloting a new initiative to offer early meetings with companies to foster discussions about their pediatric development strategies for new medicines.


Companies are often reluctant to conduct clinical studies in children due to ethical concerns and fear of adverse events during trials. However, conducting clinical research on children is critical to determining whether drugs are safe and effective in pediatric populations.

To ensure adequate research is conducted in pediatric populations, companies developing medicines in the EU are required to complete a paediatric investigation plan (PIP).

The requirements for a PIP are found in Regulation (EC) No 1901/2006 and Regulation (EC) No 1902/2006, known collectively as the Paediatric Regulation. However, while companies are generally required to conduct studies in pediatric populations, waivers can be granted for specific products and product classes:

  1. "Production of the information referred to in point (a) of Article 7(1) shall be waived for specific medicinal products or for classes of medicinal products, if there is evidence showing any of the following:
    1. that the specific medicinal product or class of medicinal products is likely to be ineffective or unsafe in part or all of the paediatric population;
    2. that the disease or condition for which the specific medicinal product or class is intended occurs only in adult populations;
    3. that the specific medicinal product does not represent a significant therapeutic benefit over existing treatments for paediatric patients.
  2. The waiver provided for in paragraph 1 may be issued with reference either to one or more specified subsets of the paediatric population, or to one or more specified therapeutic indications, or to a combination of both."

Additionally, deferrals can be granted if, "it is appropriate to conduct studies in adults prior to initiating studies in the paediatric population or when studies in the paediatic population will take longer to conduct than studies in adults."

Early Dialogue

In the years since the Paediatric Regulations went into effect, EMA says it has "identified a number of issues which often delay the paediatric development" of new products. In turn, these delays either result in slower access to medicines for children, or authorized medicines that lack safety and dosage information for pediatric populations.

EMA is looking to address these delays by offering companies "free-of-charge early paediatric interaction meetings." Through these meetings, EMA hopes to iron out potential issues before a company submits a PIP.

EMA says it will pilot the initiative until June 2016 and will use the experience gained in the pilot to either continue or modify the initiative. The agency will be limited in how many early pediatric meetings it can facilitate through the pilot, so "medicines for rare diseases or high paediatric need" will be prioritized."


EMA Press Release, Paediatric investigation plans: questions and answers: "Is it possible to have a meeting before the application for PIP or waiver is actually submitted?


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