European Commission publishes guidelines for PIPs
The European Commission has published guidelines for drawing up a paediatric investigation plan (PIP) under the new paediatric medicines regulation. The 19-page document, which is submitted for public consultation until March 30, also details the conditions under which companies can defer or even be exempted from submitting a PIP.
The European Commission has published guidelines for drawing up a paediatric investigation plan (PIP) under the new paediatric medicines regulation. The 19-page document, which is submitted for public consultation until March 30, also details the conditions under which companies can defer or even be exempted from submitting a PIP.
The European Union adopted the paediatric medicines regulation on 26 January to encourage the development of medicines adapted for children. Its flagship measure involves a six-month extension of intellectual property rights for companies that develop paediatric medicines.
Companies filing for authorisation for a medicine after 26 July, 2008 will have to submit a PIP to a new expert committee, which the EMEA will set up within the next six months.
The industry has welcomed the regulation, but until now many questions remained about the content of the PIPs. The guidelines published by the Commission's enterprise directorate general on 31 January have begun to provide answers to these questions.
The recommendations say that companies must submit a paediatric development strategy outlining clinical, non-clinical and quality issues. The PIP must cover all sub-categories of age within the paediatric population - including neonates - that are not covered by a waiver. A company filing for marketing authorisation must discuss the possible extrapolation of adult data to paediatric patients and between different paediatric age groups.
The company must also provide a review of existing information about the product in the paediatric population, referring both to literature and to any information on off-label use and known class effects.
As a paediatric product may need to be produced in a new form that is better suited to children - for example, syrup rather than tablets - the suitability of existing forms of a product must always be discussed in the PIP. The company must also show that children to be included in a paediatric trial are representative of the population in which the product will be used, addressing issues found in pharmacodynamic, pharmacokinetic, efficacy and safety studies.
The PIP must discuss the measures the company intends to take to protect the paediatric population during the development of the product, such as using less invasive methods.
The regulation makes it possible for a company to obtain a deferral or even a waiver in certain cases. The Commission says that any request for a deferral must specify the age group concerned and should be justified on scientific or technical grounds or for public health reasons. Such justifications could include the fact that it is appropriate to develop the product for adults first, or that paediatric studies will take longer to conduct than studies in adults.
Requests for a waiver must specify which paediatric population subsets and/or indications are concerned. The European Medicines Agency (EMEA) will publish a list of waivers for certain therapeutic classes.
The Commission's document says that the competent authorities will conduct compliance checks on PIPs submitted with marketing authorisation applications. The guidelines also discuss assessment of the significance of studies started before and completed after the regulation came into effect.