SubscribeAdequate medicinal therapy on the basis of medicinal products for the use in children is a challenge – not only for paediatrics.
There are only a few authorised medicinal products for paediatric use available. Off-label use of drugs is common practise. The lack of medicinal products specifically designed and developed for the treatment of children is a problem throughout the EU. More than 50% of medicines currently prescribed to children have not been studied and authorised for use in children. The few medicinal products authorised for use in children apply to children of primary school age, only.
Conclusion: In general, either no medication or medication not clinically approved for children is applied. Off-label use is currently unavoidable.
Is it ethically justifiable to medicate children on the basis of medicinal products that are not clinically proven for the use in children - or to withhold medicines from children, because of the lack of clinical trials? And on the other hand side: is it justifiable to perform clinical trials in children?
For years not only experts have been discussing these questions.
The European commission made a political decision publishing Regulation 1901/2006 (EC):
According to articles 45 & 46 of this Regulation by 26 January 2008, any paediatric studies already completed, and concerning products authorised in the EU, shall be submitted by the marketing authorisation holder to the EMEA. On the basis of this information the EMEA is going to build up a data base with respect to further paediatric investigational planning to avoid unnecessary clinical trials in children.
According to articles 7 & 8 an application for marketing authorisation for a new chemical entity (NCE) and for line extensions the results of paediatric clinical trials on the basis of an authorised paediatric investigation plan (PIP) are mandatory from 26.07.2008 on– yet, there are certain exemptions: The agency may grant a product specific waiver or a deferral according to an adequate application.
For already authorised medicinal products an independent additional authorisation for a paediatric indication (paediatric use marketing authorisation, PUMA) may be applied for, provided a paediatric clinical trial according to an authorised PIP is carried out.
The introduction of the paediatric investigation plan (PIP) seeks to promote the development of medicinal products for paediatric use by enforcing research, development and authorisations for this type of medicinal product. The submission of the PIP has to be performed at last by the end of pharmacokinetic trial in adults. According to article 20, at the same time as the PIP is submitted a request may be made for deferral of the initiation or completion of some or all of the measures set out in that plan.
Is the specific medicinal product likely to be ineffective or unsafe in parts or all of the paediatric population, or does the disease or the condition for which the product is intended only occur in adult population, or does the product not represent a significant therapeutic benefit for paediatric population, a waiver according to article 11 may be issued.
As a reward for the conduct of paediatric clinical trials the protection period will be extended in cases of new drug applications and line extensions to 6 respective 12 months and in the case of orphan drugs to 2 years. These incentives are granted for the conduction of the studies according to the agreed investigation plan, irrespective of the outcome.
What PharmaLex can do for you?
- We submit your studies according to article 45 & 46.
- We sort your current documentation and investigate the existing studies for your intended indication.
- We evaluate the results of the studies and write expert reports.
- We evaluate the potential for deferral or waiver.
- We support your strategic planning.
- We compile your PIPs.
- We compile your PUMAs.
