About this initiative
Joint evaluation of Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December 2006 on medicinal products for paediatric use and Regulation (EC) No 141/2000 of the European Parliament and of the Council of 16 December 1999 on orphan medicinal products
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UNITE2CURE - CCI (France)
Cancer is the first cause of death by disease for children over one year of age in Europe. Drug companies have little interest in paediatric cancers, which are perceived as an unattractive commercial market. As a consequence, more that 50% of drugs given to children have never been investigated in this population, but only in adults, and those drugs are administered “off-label”. Both the Paediatric Regulation and the Orphan Regulation have...
Ministry of Health, Welfare and Sport, the Netherlands (Netherlands)
Dutch government feedback on the roadmap for the evaluation of the legislation on medicines for children and rare diseases (medicines for special populations) 8 January 2018 The Ministry of Health, Welfare and Sport of the Netherlands is strongly in favor of a proper and thorough assessment of the regulatory framework to ensure that the EU offers effective incentives that encourage innovation, but also safeguard accessibility, availability and...
TEDDY - European Network of Excellence for Paediatric Clinical Research (Italy)
This initiative is strongly welcome. Some comments are following reported: Context -Evidence shows that the orphan reward is not as attractive as expected by the various legislations. In the same time, it must be acknowledged that the development of a medicine to treat a rare disease in a paediatric setting could be very difficult (e.g. inborn errors of metabolism affecting children from very early life). -With reference to the special funds...
Fondazione per la Ricerca Farmacologica Gianni Benzi onlus (Italy)
A. Context, purpose and scope of the evaluation Context - So far, great efforts have been made by the European Medicines Agency on the availability of drugs for rare diseases. They are still required from the EU, its institutions and member states. In fact, results deriving from a study performed on EuOrphan data (Giannuzzi et al., Orphanet 2017) reveal that, thanks to the incentives issued by the regulations aimed at encouraging the...
Plasma Protein Therapeutics Association (Belgium)
PPTA, the Plasma Protein Therapeutics Association, welcomes the opportunity to comment on the European Commission Roadmap regarding the Evaluation of the legislation on medicines for children and rare diseases which was published on 11 December 2017. The Plasma Protein Therapeutics Association (PPTA) is the standard setting and global advocacy organization with a strong European presence representing private manufacturers of plasma-derived...
European Social Insurance Platform - ESIP (Belgium)
The European Social Insurance Platform, ESIP, strongly welcomes the initiative of the European Commission to evaluate the legal framework concerning paediatric and orphan medicines and the interaction between the two pieces of legislation. In particular, we welcome the intention to carry out a more detailed analysis of the impact of the orphan medicinal products Regulation as regards fulfilling patients’ needs and the financial and societal...