On November 25th 2020, the European Commission published an inception impact assessment (IIA) evaluating proposals to review EU regulations for medicines for rare diseases and for children. It followed the evaluation conducted over the summer that identified shortcomings in the current legislative framework. In this assessment, the European Commission highlighted that both legislative instruments have stimulated R&D of medicines in these areas. However, according to the Commission, shortcomings in terms of addressing unmet medical needs and access for patients cannot be addressed by non-legislative measures, though legislative measures alone will not solve all issues as factors external to the legislation also have an impact (i.e. P&R and companies’ strategic decisions).
Four main objectives are laid out for this revision :1) To foster R&D of medicines for rare diseases and for children, especially in areas of unmet needs and better alignment with patient needs; 2) To contribute to ensuring the availability and timely patients’ access to orphan and paediatric medicines; 3) To ensure that the legislation is fit to embrace technological and scientific advances; 4) To provide effective and efficient procedures, for assessment and authorization of these medicines.
To address these four objectives, the Commission proposes to review the legislation, presenting four options for each of the regulation.
For the paediatric regulation
For the paediatric regulation, there are common elements included in each option, namely criteria to determine unmet needs for children, improved rules linking incentives to the placing on most of EU national markets and a revision of the conditions to allow exemptions to complete a Paediatric Investigation Plan (PIP). Additionally, the four options explored in the IIA all examine changes to the current system of supplementary protection certificates (SPC) for paediatric drugs and look at the possibility of creating novel incentives.
For the orphan regulation
In parallel, the IIA looks at criteria to determine unmet needs for orphan diseases, ensuring that new products provide a real benefit to patients and encouraging faster development through a temporal validity of the orphan designation. The options focus on reviewing the length of the market exclusivity depending on the type of development, as well as modifying the current system of designation criteria.
An impact assessment, which will aim to quantify the costs and benefits of the proposals, will run until the first quarter of 2021. It will be accompanied by a study “used to source additional evidence on the costs and benefits of the different policy options outlined.”