EMA Publish Draft Guidance on Development of Medicines for Paediatric Use

EMA Publish Draft Guidance on Development of Medicines for Paediatric Use

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Children can neither be regarded as small adults nor as a homogeneous group in themselves. As a consequence, paediatric medicines should be appropriately designed for the target age group(s).
In January 2007 Regulation EC No 1901/2006 (the “Paediatric Regulation”) entered into force. As a result of this Regulation, the number of paediatric formulations that the pharmaceutical industry will have to develop to support their clinical trials will increase. It is expected that the number of medicines applying for a marketing authorisation for paediatric use will increase as a result. Therefore, the existing regulatory documents need to be supported by specific regulatory guidance on the pharmaceutical development of medicines for use in children between birth and 18 years of age.

The physical, metabolic and psychological processes peculiar to growth from birth into adulthood reveal that children can not be regarded as small adults nor can they be regarded as a homogeneous group in themselves. As a consequence, clinical studies in adults are not necessarily predictive for children. Thus, clinical trials may be needed in children of different ages in order to demonstrate that a medicine is safe and effective in all of the indicated target age group(s).

In addition, the treatment of children with medicines poses specific pharmaceutical problems which have not been seen to the same extent in adults and which occurrence may be age dependent. For example, young children are simply unable to swallow conventionally-sized tablets whereas tablets are a favourable dosage form for elder children and adults. Especially neonates pose specific characteristics and needs. They may for example require very small volumes of a parenteral medicine in order to avoid a volume overload. Therefore, children should be treated with medicinal products of which the pharmaceutical design is tailored for use in the target age group i.e. age appropriate medicines.

Knowledge on the critical to quality aspects of paediatric medicines is still limited, especially when considering these aspects in a multidimensional approach to the best attainable and affordable paediatric medicinal products. As a consequence, the usefulness (practicality) of some of the currently paediatric medicines might be questionable / based on minimum standards and could consequently be subject to further optimisation in the interest of parents, other caregivers and children.

On the 26th of January 2007, the “Paediatric Regulation” entered into force (Regulation EC No 1901/2006 of The European Parliament and of the Council, amending regulation EEC No 1768/92, Directive 2001/20/EC, Directive 2001/83/EC and Regulation EC No 726/2004). This regulation aims to “facilitate the development and accessibility of medicinal products for use in the paediatric population, to ensure that medicinal products used to treat the paediatric population are subject to research of high quality and are appropriately authorised for use in the paediatric population, and to improve the information available on the use of medicinal products in the various paediatric populations”. As a result of this Regulation, both the number of paediatric formulations that should be developed by the pharmaceutical industry and the knowledge on the critical to quality aspects of paediatric medicines is expected to increase rapidly.

Bearing the aforementioned in mind, the current regulatory documents need to be supported with guidance on the pharmaceutical development of paediatric medicines. Therefore, this guideline aims to provide additional tools for the rationale pharmaceutical development of medicines for children between birth and 18 years of age to those already described in the current CHMP and ICH guidelines. The guideline intends to balance between predictable and consistent regulatory assessments of paediatric medicines (either generic, innovative, existing or new), the speed of development, industrial feasibility and the need to develop medicines that are better tailored for use in children than the currently authorised, but “questionable” paediatric medicines or the currently applied off-label or pharmacy compounded medicines. The outcome of this balanced approach should not necessarily result in a “gold standard” paediatric medicine.



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Damien Bové is THE Drug Development and Regulatory Consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.

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Damien Bové is the drug development and regulatory consultant (pharmaceutical or biotechnology), I work with my clients to define a drug development target, define a drug development strategy, define a regulatory strategy or define a commercial strategy. Our clients are generally raising funds or looking to license out their technology and we help them achieve it. If you want to know more don’t hesitate to get in touch.


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Fill Out the Short Form Below…




Sign up for the most value add free newsource you can get for free. We spend a huge amount of time and effort monitoring the main drug / device regulators websites for changes in the regulatory environment, and capture between 20 and 40 new regulations, rules and initiatives each month, and summaries them in a fantastic FREE monthly Regulatory and Market Round Up. You can Un-Subscribe at any time and we don not share your details with anybody. You can’t afford to miss out on this service. Just fill in the form below.

“Please note that the pages on this website are designed to provide you with general information only. We make no warranties, representations or undertakings about any of its content. This includes the completeness, accuracy and fitness for any particular purpose, or the content of any third party site referred to or accessed through it. You are personally responsible for ensuring that the information is correct and we will not be held liable or accountable for any mistakes that occur.”