Published the texts of the Regulation and the Directive as deriving from the trilogues negotiations

Mar 23, 2026

The texts of the forthcoming European Pharmaceutical Package, including a Directive and a Regulation[1], as agreed during the interinstitutional negotiations concluded in December 2025, have been made publicly available on 6 March.

These texts also received positive opinion from the Committee of Public Health of the European Parliament on March 18th. This represents the first step of the formal approvals that will lead to the release of the final official texts. Some of the anticipated changes that will impact on medicines for rare diseases and children, have been confirmed. Among them, the restructuring of the European Medicines Agency (EMA), that will reduce its scientific committees for human medicines from five to two: the Committee for Human Medicinal Products (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC). The expertise of other committees, such as the Committee for Orphan Medicinal Products (COMP), the Paediatric Committee (PDCO), the Committee for Advanced Therapies (CAT) and the Committee for Herbal Medicinal Products (HMPC), will be included through working groups, advisory groups, and a pool of experts, to be established as necessary. For the first time, two patients’ representatives and two healthcare professionals’ representatives (plus two alternates for each category) will be CHMP members, but they will be jointly entitled to one vote.

In the field of paediatric medicines, a more flexible, stepwise approach to paediatric development has been introduced with the initial Paediatric Investigation Plan (PIP), including only the details and the timing of the studies, to be progressively completed as additional scientific evidence becomes available. The stepwise approach could be applied when the active substance is not yet authorised in at EU level and is intended for a novel paediatric condition or for an existing paediatric condition via a new mechanism of action. Additionally, it might also be acceptable on the basis of scientifically justified reasons proposed by developers.

Moreover, the so called “mechanism of action” principle has been confirmed. If a medicine developed for a specific disease in adults has a mechanism of action that may also be relevant to a paediatric disease, it should be studied in children for that paediatric condition as well. This requirement applies only within the same therapeutic area.

A dedicated section on medicines authorised for the use in paediatrics has been foreseen within the EMA database of approved medicines.

In the field of orphan medicines, the introduction of the concept of “breakthrough orphan medicinal products” has been confirmed. This is referred to orphan medicinal products addressing a disease with no authorised treatment and whose use would result into a clinically relevant reduction to disease morbidity or mortality. For these medicines, the duration of market exclusivity is extended from 10 to 11 years.

Concerning orphan designation, an incidence-based criteria will be applicable for conditions which have a short duration and high mortality. In this case, it will be acceptable to justify the rarity of the disease based on the number of people that acquired the disease during a specific time period, instead of prevalence. The orphan designation criterion on the basis of return on investment has been abolished, since it has never been used. The orphan designation will be valid for seven years. That may be extended in case the sponsor can provide evidence that studies on the rare conditions are ongoing and promising.

To maximise the potential benefit of clinical research, continued exploration of new indications for orphan medicinal products should be encouraged. For this reason, it will be rewarded with an additional period of one year of market exclusivity for a new therapeutic indication (with a maximum of two indications).

In parallel, other provisions have been confirmed in the final texts, such as the strengthened support to developing new antimicrobials, including the adoption of the World Health Organization prioritisation model, and medicines targeting neglected tropical diseases. These medicines will be both automatically entitled to access to the PRIME scheme.

The forthcoming legislation will also introduce a range of new regulatory instruments and provisions — such as “regulatory sandboxes”, “transferable data exclusivity vouchers”, “platform marketing authorisations”, and “temporary emergency marketing authorisations” — aimed at significantly improving the development and evaluation of medicines.

Other relevant points for medicinal products research and development, as outlined in the legislation, are as follows:

Particular attention is given to the design and composition of clinical trials to ensure robust and comprehensive data, including, where appropriate, gender-balanced representation.

The legislation also explicitly recognises the role of data generated through in silico methods — such as computational modelling and simulation, molecular and mechanistic modelling, digital twins, and artificial intelligence — in supporting regulatory decision-making.

Competent authorities will be empowered to assess additional evidence that may influence the benefit–risk balance of medicinal products beyond that submitted by marketing authorisation holders. Where justified, they may recommend variations to the terms of a marketing authorisation.
A series of provisions, intended to address the shortage of medicinal products at Eu level, have been also introduced. These measures will be complemented by another legislative document, the Critical Medicines Act, that it is now undergoing interinstitutional negotiations and is expected to be finalised by mid-2026.

Given the advanced stage of the legislative process, substantive changes to the text of the proposal for Regulation and Directive are now considered unlikely. Once the formal adoption from the Council of European Union and the European Parliament will be granted (expected by mid-2026), official texts, including translations, will be formally published.

External sources:

[1] Proposal for a Regulation laying down Union procedures for the authorisation and supervision of medicinal products for human use and establishing rules governing the European Medicines Agency; Proposal for a Directive on the Union code relating to medicinal products for human use

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Published the texts of the Regulation and the Directive as deriving from the trilogues negotiations

Updates on the new EU pharmaceutical legislation

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