Updates on the new EU pharmaceutical legislation

Feb 6, 2026

On 11 December 2025, a landmark political agreement was reached by the European Commission (EC), the European Parliament and the Council of the European Union on the comprehensive reform of the EU pharmaceutical legislation.

The reform was first proposed by the European Commission in April 2023, with the European Parliament position on legislative documents adopted one year later, in April 2024, and the Council of the European Union in June 2025.

The reform includes two legislative proposals – a new Directive and a new Regulation – which together will form the EU regulatory framework for all medicines, including those for rare diseases and for children. The proposed legislation aims to update and streamline the research, approval and availability of medicines to patients across the European Union. Ultimately, it is intended to simplify the structures and procedures introduced by various pieces of legislation over the years. These include Regulation (EC) 726/2004, Directive 2001/83/EC and Regulations on paediatric and orphan medicines ((EC) 1901/2006 and (EC) 141/2000, respectively), which will be repealed, and the Advanced Therapy Medicinal Products Regulation (EC) 1394/2007, which will be amended.

The new Directive and Regulation will be complemented by the Critical Medicines Act, aimed at securing the supply of essential medicines by strengthening EU manufacturing, reducing external dependencies, and improving prevention and management of drug shortages.

Fondazione Gianni Benzi has actively followed the debate, the amendments to the legislative documents and the process leading to approval of the European pharmaceutical reform and will continue to closely monitor its further developments.

Despite the reform introduced substantial changes, whose full details and specific impact on rare diseases is still unknowns, several aspects of the reform, like the simplification of the European Medicines Agency (EMA) Structure and the increased flexibility of some regulatory procedures could have direct implications on medicines for rare diseases and children:

  • Simplified structure of the European Medicines Agency. EMA is expected to simplify its committee structure from five to two scientific committees for human medicines: the Committee for Human Medicinal Products (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC). The new, leaner structure is expected to be complemented by stronger support for the two committees, relying on the best available expertise.
  • Simplified and more flexible regulatory procedures. For example, the opinion on a Marketing Authorisation application is expected to be released in 180 days instead of 210. In addition, the reform may allow the establishment of case-by-case “regulatory sandboxes”, intended as controlled live testing environment to test new and innovative therapies. Within this framework, a substantial modification is expected to be introduced to the Paediatric Investigation Plan (PIP) to provide a more flexible, stepwise approach to paediatric development. It consists with an initial, partial PIP to be agreed and progressively completed as additional scientific evidence becomes available. This procedural flexibility is particularly relevant for orphan medicines, where paediatric data are often limited at early stages of development. Furthermore, academic institutions may be enabled to submit non-clinical or clinical evidence directly to regulators to support new indications for already approved medicines (repurposing).
  • The duration of market exclusivity is expected to be granular according to the type of medicine or the development. For example, it is expected be extended for orphan medicinal products addressing a high unmet medical need.
  • The concept of breakthrough orphan medicinal products may be introduced. This is referred to orphan medicinal products addressing a disease with no treatment authorised and whose use would result a clinically relevant reduction in disease morbidity or mortality.

With reference to the Critical Medicines Act, trilogue negotiations were launched in February, with co-legislators stressing the urgency of concluding the process and reaching an agreement. This first meeting primarily served as an exchange of institutional mandates to identify key technical areas for further work, i.e. scope of strategic projects, stockpiling, joint procurement, financing, and EU-based production of critical medicines. Substantial discussions on the Act’s key provisions will take place at the next political trilogue planned on 16 March 2026.

A more reliable impact will be revealed, as soon as final texts of the forthcoming legislation will be made available, after the formal approval of the Council of the European Union and the European Parliament, expected by the end of 2026.

A new EMA webpage dedicated to the implementation of this new legislation is offering detailed insights into the implementation process and specific technical and procedural aspects, including a timeline. This shows that the new pharmaceutical legislation will become applicable in 2028, following a transition period for the update of national laws at Member States level, the adoption of further acts from the European Commission, as well as implementation guidance, procedures and IT systems.

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