A Paediatric Investigation Plan Case Study
- PDF / 169,687 Bytes
- 9 Pages / 612.28 x 793.7 pts Page_size
- 56 Downloads / 224 Views
CURRENT OPINION
Adis ª 2012 Springer International Publishing AG. All rights reserved.
A Paediatric Investigation Plan Case Study Klaus Rose klausrose Consulting, Riehen, Switzerland
Abstract
The aim of paediatric legislation in both the US and EU is to ensure that children participate in and benefit from pharmaceutical progress more than this is achieved by the market mechanisms that drive drug development in adults. The EU paediatric regulation, in force since 2007, has established the paediatric investigation plan (PIP) as a new key document in the general drug development process. PIP submission and successful negotiation are now a prerequisite to register new drugs in the EU. Without an agreed PIP, the European Medicines Agency (EMA) will not validate a marketing authorization application (MAA), so market access of any new medication is blocked until a PIP is negotiated. The PIP should be submitted at the end of human pharmacokinetic studies in adults and before proof of concept, i.e. before clinical efficacy of the new compound has been shown. The PIP is an elaborate document of typically 50–100 pages in length. Apart from company (e.g. name and address, correspondence e-mail) and regulatory information (e.g. previous consultation with regulatory authorities), the PIP discusses the mode of action of the compound, the targeted disease in adults, expected therapeutic benefits and the assumed clinical use of the new compound in children of all age groups from preterm newborns to adolescents. The PIP must list the ‘proposed’ development measures for paediatric use including juvenile animal studies, development of age-appropriate formulation(s) and clinical studies. The PIP will also request a ‘partial waiver’ for those age groups where, in the company’s view, no specific paediatric development is required. Key parameters of the proposed measures, including a start and end date, must be outlined. Most clinical studies will not physically begin before proof of concept in adults. Most paediatric clinical trials will be deferred, i.e. will start and end after the PIP has been approved. In the PIP preparation, the building up of a paediatric development strategy is the most demanding part. Usually, the company developing the new compound has solid expertise in the targeted disease in adults but not in children; therefore consultation with experienced paediatric specialists is essential. The key questions are: does the disease exist in children; are there areas of high medical need in paediatric medicine beyond the adult indication that might be treated with the new compound; and which development steps are required? Once these questions are answered, the writing of the PIP is rather straightforward. The PIP submission is followed by an elaborate negotiation process with the EMA and its paediatric committee (PDCO) that can be concluded within 9 months if everything runs smoothly. The EMA/PDCO expect a programme that, after completion, results in a registration in children, provided the drug is effective and has an
Data Loading...
