Deliverable 75 - Report on the socioeconomic impact of the development of paediatric drugs

A large proportion of medicines used in children are prescribed outside the terms of the drug license i.e. off-label, which can place children at a direct risk of under- or overdosing and a delayed risk of long-term adverse effects. The European Commission decided to tackle this problem providing a structural solution: The Regulation (EC) no 1901/2006 on medicinal products for paediatric use which intends to ensure that medicinal products used to treat the paediatric population are subject to ethical research of high quality and are appropriately authorised for use in the paediatric population. The Regulation envisages specific requirements, incentives and supporting measures for pharmaceutical industries. After having drafted the European Paediatric Regulation, the European Commission committed RAND Europe for an a-priori impact assessment. The main results of this analysis are that o The effect of the regulation will be felt in new drugs instead of in generic ones; o SMEs could not have adequate infrastructures to participate in the market; o Higher remunerating sectors could be promoted instead of therapeutic needs; o The decrease in the use of off-label drugs is unknown; o The recruitment of expertise by industries could be a critical issue; o The possibility to increase capacity of patients recruitment in EU funded research projects needs to be verified; o The pharmaceutical industries perception of the Paediatric Committee (PDCO) as a totally independent and impartial body has to be verified too. The present workrepresents a pilot study and has involved 16 pharmaceutical industries (2 SMEs and 14 big enterprises) to evaluate the impact of the Regulation AFTER its adoption. Companies have been invited to answer an on- line questionnaire which investigates the following areas: o Awareness/knowledge; o Impact on the organization; o Actions undertaken after the approval of the Regulation; o Reactions of the pharmaceutical industries to the changed scenario and proposals for possible improvements of the European framework. The preliminary analysis has shown a quite good and general understanding of the Regulation in the enterprises areas and the needed training process that has TD-D075-Report_socioeconomic_impact_develop_paediatric_drugs 4 / 41 EUROPEAN UNION TEDDY SIXTH FRAMEWORK PROGRAMME NETWORK OF EXCELLENCE been organized to cope with changes appears in a quite advanced situation even if there still exist some critical areas: Paediatric Investigation Plan and Marketing Authorizations requirements in the first places but also Planning and managing CTs and Ethical Issues are pointed out. The introduction of the Regulation had a remarkable impact on the organization especially for R&D and Regulatory affairs, Post marketing surveillance, and Marketing and Pricing. The attractiveness of the 6 months extension of the Supplementary Data Protection (SPC) appears to be lower than the one for the 10 years extension of the Data Protection and this is in contrast with the expectancy of the RAND report. Considering that the most part of the participants are involved in the authorization of innovative drugs there could be a bias in this result. Other open critical issues are pointed out such as: patient recruitment, innovative study designing, practical and timely application of the Regulation. Participants would like an improvement in the reimbursement policies as a compensation for the additional expenses due to the Regulation requirements. A deeper analysis needs to be done to better evaluate the impact of the Regulation on the Pharmaceutical Industries based on this preliminary results.