The ESIP & MEDEV amendments aim to address the unintended consequences in terms of market distortions brought about by the existing legal framework, such as concentration of R&D in specific disease areas (of higher profit), artificial segmentation of conditions into smaller subgroups as well as increasingly high price demands. The consolidated chart focuses on rebalancing the incentives systems, steering research and development towards areas of truly rare diseases and unmet needs.

To ensure that the revised legislation is fit for purpose we recommend to:

• Adjust the prevalence criterion for orphan designation in order to focus incentives on the development of therapies for truly rare diseases: lower the threshold for single indications (to 1:10,000), introduce a threshold for all authorised indications combined (of 5:10,000);
• Revise and clarify the definition of ‘significant benefit’ laid down in Regulation (EC) 847/2000: ‘major contribution to patient care’ should be deleted, since it cannot be easily defined;
• Maintain the profitability criterion for the re-evaluation of market exclusivity (ME), by setting a threshold for the volume of sales (to 1 billion EUR per year) for all combined indications;
• Clarify and strengthen criteria for maintaining ME: combining profitability, prevalence, proof of a clinically meaningful benefit, submission of pricing and reimbursement (P&R) applications in all EU Member States within 2 years after marketing authorisation (MA);
• Anticipate the review of ME criteria at the end of the second year after MA and henceforth annually;
• Entrust the EMA with these regular reviews, followed by the termination of ME if the criteria above are no longer met.

Find our sets of ESIP-MEDEV amendments to the OMPs legislation and the accompanying position paper.

This position complements our detailed ESIP-MEDEV amendments to the EU general pharma legislation, published in June 2022 - here the key messages.