Yesterday, the European Commission published an evaluation and fitness check roadmap for its joint evaluation of the legislation on medicines for children and rare diseases (medicines for special populations), i.e., the Orphan Medicines Regulation (EC) No 141/2000 and the Paediatric Medicines Regulation (EC) No 1901/2006. The evaluation will start in Q1 2018 – with a public consultation scheduled for Q3 2018 – and is expected to end in Q3 2019. Stakeholders are invited to provide their feedback by 8 January 2018.

The roadmap explains that the purpose of the evaluation is twofold and seeks to (i) provide an assessment about the strengths and weaknesses of the two legal instruments separately and combined, thereby focusing on the outputs in products catering for a real unmet medical need; and (ii) give insight on how the various incentives that are related to the legislation have been used, and the financial consequences this has resulted in. The evaluation will focus on the results of and the synergy between the two regulations as well as the cost-effectiveness and practical use of the incentives and rewards incorporated in the legislation.

The EU Orphan Regulation and the Paediatric Medicines Regulation are intertwined and partly complementary as they both intend to redirect investment in previously neglected therapeutic areas through a scheme of incentives, rewards and obligations, which go beyond the standard incentives available to all medicines development (such as RDP and SPCs). Given that a considerable number of paediatric diseases qualify as rare diseases, those two areas partly overlap. As already announced in the Commission Report on the Paediatric Regulation, the reason why major therapeutic advances in diseases that are rare and/or unique to children often fail to materialise requires further scrutiny and therefore needs to take into account the orphan legislation as well (see our update: Proposals for reform of EU Paediatric Regulation postponed – EC to take another look at orphan incentives for paediatric medicines).

The public consultation to support this joint evaluation will consist of (i) a 12 week public consultation planned for Q3 2018; and (ii) a targeted consultation involving Member States and specific interest groups to allow different aspects to be explored in more depth. The Commission indicates that a balance of representation among the different stakeholders will be ensured, including the Member States (national medicines regulators, payers, HTA bodies), the European Medicines Agency, patients, hospitals, doctors and academia, the pharmaceutical and biotech industry and NGOs.

The joint evaluation will complement ongoing work, especially the 2017 Commission Report on the Paediatric Regulation and the ongoing Commission study on pharmaceutical incentives, which includes an analysis of impact of pharmaceutical incentives (such as RDP, market exclusivity for orphans and paediatric rewards) on innovation, availability and accessibility of medicines. An additional study will be launched in 2018 with remaining research questions especially on orphan medicinal products.