Health and food safety

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Medicines for children and rare diseases (medicines for special populations)

Summary
Evaluation
Commission Proposal
Legal Act
Implementation

Overall state of Play:

Evaluation finalised: SWD(2020)163, on 11/08/2020
Preparation of Commission proposal(s) on-going, expected to be adopted in 2023

State of play, main conclusions, outlook

The Commission finalised its evaluation of the Paediatric Regulation (EC) No 1901/2006 and of the Orphan Medicinal Products Regulation (EC) No 141/2000.

Building on the evaluation findings, the Commission has started preparatory work in view of proposing a revision of both Regulations. The revised provisions will be integrated in the revision of the general EU pharmaceutical legislation in order to ensure coherence and complementarity between the various provisions applicable to these medicinal products. These are flagships initiatives planned to be adopted in 2023 as part of the Pharmaceutical Strategy for Europe that was launched in 2020 to create a future-proof and crisis-resistant framework ensuring Europe’s supply of safe and affordable medicines. All options considered by the Commission will include elements of simplification and burden reduction.

State of Play:

Finalised, SWD(2020)163, on 11/08/2020

Scope:

Evaluation of the Paediatric Regulation (EC) No 1901/2006 and of the Orphan Medicinal Products Regulation (EC) No 141/2000.

Evaluation findings:

The evaluation found that both Regulations have fostered the development and availability of medicines for patients with rare diseases and for children.

However, the evaluation also showed shortcomings in the functioning of the existing legal framework. This is partly due to the legislation not being able to stimulate development of medicines in areas of unmet needs (e.g. 95% of rare diseases still have no treatment option) and to better ensure that European patients actually get the medicine (access), independently from which country they live in. Moreover, weaknesses relate to the Regulations not being flexible enough to allow scientific developments and certain procedures turned out to be inefficient and burdensome.

The evaluation pointed also to certain procedures being inefficient and burdensome: there may be room for simplification and streamlining of procedures and internal processes including within the European Medicines Agency to avoid the risk of possible inconsistencies and delays. Furthermore, some reporting requirements create administrative burden without fully achieving their objective (e.g. the obligation for sponsors to submit an annual report on the orphan designation to the Agency). Moreover, the extension of the Supplementary Protection Certificate requires individual requests to each national patent office, making the procedure for obtaining this reward complex and time-consuming and resulting often in unreliable outcomes.