The IIA is based on the evaluation published this summer, which assessed outcomes and weaknesses of the two legislative frameworks. While highlighting that both Regulations increased availability of medicines for patients with rare diseases and for children, the review also observed that access to these products remain problematic. 

In the IIA the Commission recognises the need to hone the Regulations to better target unmet need in rare diseases and rare paediatric diseases and address access and availability issues. Four policy options are presented on redefining the criteria for orphan designation, revising the existing incentives, particularly market exclusivity, and introducing additional novel incentives. 

ESIP replied to the Inception Impact Assessment, building upon recommendations from a recent position paper and highlighting to the need to:

• better define criteria for unmet medical need, considering patient and societal needs and aligning existing definitions across the EU legislative framework;
• revise definition of “significant benefit”, based on data showing improvements in patient-relevant outcomes;
• revise the criteria for orphan designation, combining the prevalence of all therapeutic indications to re-assess the prevalence threshold and maintaining the criterion of insufficient return on reimbursement;
• include a standard definition of “sufficiently profitable”, considering profit generated by all indications of a product;
• consider a number of alternative incentive, including de-linking research and development from commercialisation costs, with R&D led by publicly funded institutions. 

A public consultation is planned for the first semester of 2021 while the proposal for new orphan and paediatric regulations are expected in the first quarter of 2022.

Read ESIP feedback to the inception impact assessment here and find our position paper on the Orphan Regulation here.