This article is part of our series covering the proposed reforms to the EU pharmaceutical legislation. To view the entire series, click here.

In July 2023 we outlined the European Commission’s proposed reforms to the regulation of paediatric medicinal products (“PMPs”), which formed part of a broader legislative reform. The Commission proposed a new Directive[1] and Regulation[2]. Stakeholders were rewarded with an update on 10 April 2024, following the Parliament’s first reading. Several changes were proposed, some of which affect PMPs. This article discusses the revised proposed Directive[3] and Regulation[4], insofar as it affects paediatric medicines. 

As we discussed previously, the EU Commission’s 2020 evaluation of medicines for rare diseases and children identified several shortcomings, including the need for development in therapeutic areas that are less profitable. The Commission’s aim, among other things, is to reduce patient access time and commercialising at least three PMPs every year. The reward for originators developing new PMPs, the Commission claims, is EUR103 million in gross profits annually.

Paediatric Investigation Plans and working groups

Of the two new legislative provisions, the Proposed Regulation (now revised) outlines the procedures governing Paediatric Investigation Plans (“PIPs”). The previous iteration of the draft set out the various procedural requirements for PIPs including deferral and waiver schemes. The amended wording currently before the Council permits the deferral of measures contained in a PIP “based on scientific, ethical and technical grounds or considerations related to public health”.[5] This could be seen as a positive change by originators, requiring a deferral to be justified by the Agency. 

With respect to draft Article 84 (“Modification of a paediatric investigation plan”), the Agency can request changes to the PIP if the agreed plan is no longer appropriate, but these requests must now be based on “detailed scientific grounds”.[6] This should provide some, albeit limited, comfort to stakeholders because Agency requests for changes to a PIP must be justified. 

Article 88 of the Proposed Regulation states that where a PIP is discontinued, the applicant must notify the Agency (and provide reasons) within six months. This has now been extended to six months “…or as soon as possible”.[7] This wording isn’t necessarily very clear but could provide some leeway for originator applicants if it isn’t possible to make a notification within six months. 

Article 146 of the Proposed Regulation outlines the general provisions concerning scientific committees. The Commission now proposes to include a “paediatric representative” in the list of parties that the scientific committees should consult on an advisory basis.[8] The list also includes patient and consumer organisations and healthcare professionals.

Parliament also proposed the establishment of an “ad hoc working group on paediatric medicinal products”[9] but at the time of writing, it is unclear exactly what that would look like. This could potentially introduce further red tape in an already complex (and expensive) approval process. 

Rewards and failure to complete a PIP

As we discussed previously, the rewards for completing a PIP are contained in the Proposed Directive (as revised), alongside provisions on checking PIP compliance. 

On the whole, very few amendments were proposed. However, Parliament emphasised that “effort should be made” to address problems such as the failure to complete paediatric clinical studies in time, or obtaining the data required for marketing authorisation.[10] The Revised Directive does not, however, clarify what “every effort” means. 

In order to support small patient populations (such as for paediatric or rare diseases), Parliament proposes the use of “alternative paths” to making PMPs available.[11] Member States are encouraged to utilise the National Competent Authorities on Pricing and Reimbursement and Public Healthcare Payers (“NCAPR”) to exchange best practice regarding implementation of cross border access agreements and negotiations. 

Article 114 (“urgent Union procedure”) provides various procedures that could be triggered if concerns are raised about a particular product. Parliament suggests that in the absence of a PIP, justification must be provided and where relevant, evidence shall be obtained from post-marketing long-term studies.[12]

Lastly, the marketing authorisation must contain a statement indicating compliance with the PIP. The Revised Proposed Directive now also proposes to make public the conclusions of the assessment regarding PIP compliance.[13]

What happens now?

The Parliament’s revised position will be scrutinised by the European Council, which could either accept or reject the new wording. Depending on the Council’s review, the Parliament may have to make further changes. To read more about the legislative process and for links to other articles in this series (which covers other topics, such as antimicrobial resistance, parallel imports and regulatory data and market protection rules), see this article.

This article is part of a series covering updates to the proposed reforms to EU pharmaceutical legislation. To view the whole series, please see below:

• EU Parliament Adopts Position on Pharmaceutical Reform 
• EU Parliament Adopts Position on Pharmaceutical Reform: Focus on Combating Antimicrobial Resistance
• EU Pharmaceutical Law Revision: Re-Boxing Set to Become the New Normal for Parallel Imports?
• EU Parliament Adopts Position on Pharmaceutical Reform: Focus on Regulatory Data and Market Protection Rules
• New EU Pharmaceutical Reform: Key Changes and Impacts on Orphan Medicinal Products
• EU Parliament Adopts Position on Pharmaceutical Reform: implications for the Environmental Risk Assessment

[1] Proposal for a Directive of the European Parliament and of the Council on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC (“Proposed Directive”).

[2] Proposal for a Regulation of the European Parliament and of the Council laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006 (“Proposed Regulation”).

[3] European Parliament legislative resolution of 10 April 2024 on the proposal for a directive of the European Parliament and of the Council on the Union code relating to medicinal products for human use, and repealing Directive 2001/83/EC and Directive 2009/35/EC (COM(2023)0192 – C9-0143/2023 – 2023/0132(COD)) (“Revised Proposed Directive”).

[4] European Parliament legislative resolution of 10 April 2024 on the proposal for a regulation of the European Parliament and of the Council laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency, amending Regulation (EC) No 1394/2007 and Regulation (EU) No 536/2014 and repealing Regulation (EC) No 726/2004, Regulation (EC) No 141/2000 and Regulation (EC) No 1901/2006 (COM(2023)0193 – C9-0144/2023 – 2023/0131(COD)) (“Revised Proposed Regulation”).

[5] Amendment 68 (Regulation), Recital 112.

[6] Amendment 218 (Regulation), Article 84(2)(1).

[7] Amendment 221 (Regulation), Article 88(1).

[8] Amendment 321 (Regulation), Article 146(8)(1).

[9] Amendment 332 (Regulation), Article 150(5)(a) (new). 

[10] Amendment 10 (Directive), Recital 9. 

[11] Amendment 43 (Directive), Recital 58 (new). 

[12] Amendment 114 (Directive), Article 6(5)(2)(a) (new). 

[13] Amendment 160 (Directive), Article 49(2).