The main objective of this study is to support the Impact Assessment for the revision of the EU legislation on medicines for rare diseases (‘Orphan Regulation’) and children (‘Paediatric Regulation’). This will be done by evaluating the policy options considered by the European Commission against the reference of the baseline of ‘no EU action’. The study team will also address the following objectives, namely to:
- Foster research and development of medicines for rare diseases and children, especially in areas of unmet need and in better alignment with patient needs;
- Ensure an increase in availability and more timely access of patients to orphan and paediatric medicines;
- Ensure that the legislation is fit to embrace technological and scientific advances;
- Provide effective and efficient procedures for the assessment and authorisation of orphan and paediatric medicinal products.
The study team will collect evidence about the 27 EU Member States and highlight potential differences in the situation of specific countries or regions. The methodological approach to this study is based on recognised qualitative and quantitative data collection and analysis methods, including desk research, CBA, econometric analysis, and multi-criteria analysis, among others. The stakeholder views will be explored through a variety of field research activities, including online public consultation, targeted surveys, and interviews as well as focus groups. Towards the end of the study, we will perform the assessment of policy options to analyse the data gathered through the analysis, as well as develop the monitoring and evaluation arrangements as well as propose conclusions and recommendations.