RB – 12/2020

On the 25th November 2020, the European Commission published its impact assessment on the revision of the regulations covering children’s and rare disease medicines. This revision of the regulations is one of several measures in the European pharmaceutical strategy that was published in parallel (News 12/2020).

The impact assessment takes up the findings of the regulation evaluations. These are to be addressed in the revision of the regulations. In 2022, the European Commission plans to present a revised proposal regarding legislation covering children and rare disease medicines.

Evaluation of the regulations

Essentially, the results of the evaluation of both regulations show that they support research and development (R&D) into medicinal products for the treatment of children and rare diseases. Despite the positive developments in new marketing authorisations, 95% of rare diseases remain untreated.

Of the 142 rare medicinal products approved, only 28 percent represent new treatments for which no alternative treatments exist to-date.

In addition to the unsatisfactory development of new treatments for children and rare diseases, there is a gap when it comes to accessing innovation in Europe: from Member States with a high GNP (Gross National Product) to those with a low GNP and from Member States with huge populations to those with small populations as well as other attributing factors such as nationally divergent pricing and financing decisions. There are other restrictions when it comes to accessing medicines, as the Rare Medicinal Products regulation does not impose an obligation for a market launch of the medicines in all of the Member States.

On the one hand, the research agenda in the future must be geared towards the actual, as yet unmet, need for medicines and on the other hand, legislation must be amended to reflect the state-of-the-art. Existing financial incentives that have been misused with regard to expanding indications must be eliminated and inefficient processes within the European Medicines Agency (EMA) must be slimmed down.

Impact assessment of the revision of the regulations

The revision of the Regulations aims to ensure that R&D medicinal products for children’s and rare diseases, especially in the unmet needs area, should be focused on the benefits to patients. The availability of medicines and the access to them will be improved in the future. The legislation should be updated to state-of-the-art legislation based on the weaknesses identified in the evaluation. Finally, the revision should help to implement effective and efficient processes in evaluating and authorising medicines for children and rare diseases.

Interaction with the action plan for the protection of intellectual property

The revision of the incentive structures in the regulation covering children's medicines includes provisions covering SPCs (Supplementary Protection Certificates) that will extend patent protection for innovative children’s medicines over time. The published action plan on Intellectual Property Rights (IPC) and the published evaluation on the SPC Regulation point to the currently fragmented implementation of SPCs at Member State level. This is the most serious shortcoming of the system. The resulting non-transparency and inefficiency is to be replaced by a uniform process. A uniform set of regulations should offer innovators more security and manufacturers of generic drugs more planning options.

In order to improve access to innovation, the European Commission is examining which incentives can be optimised to protect intellectual property. The aim is to promote innovation for unmet needs, without overburdening the budgets of national systems.

The following options are mentioned:

  • obtaining an SPC that extends patent protection over time, should be conditional on the rapid completion of a PIP (Paediatric Investigation Plan) and its market launch in all Member States.

  • In addition to the SPCs, so-called "vouchers" could also be issued for innovations, for treatment methods against paediatric diseases that are still lacking, which will accelerate the testing and approval processes for future medicines from the manufacturers.

  • Waiving an SPC. SPCs could be replaced by "vouchers", which would provide focused R&D support for selected indications.

A public consultation is scheduled for the first quarter of 2021. The revised proposals for the regulations are to follow in the first quarter of 2022.