Orphan medicinal products

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On 11 December, after overnight interinstitutional negotiations between the European Parliament and the Council of the European Union (“Council”) and the European Commission, the institutions reached a provisional political agreement on the reform of the European Union (“EU”) pharmaceutical legislation.

This agreement concludes months of trilogue discussions and follows a much longer legislative process that began with the European Commission’s  proposal adopted in April 2023, the European Parliament’s position adopted on 10 April 2024, and the Council’s position adopted on 4 June 2025 (see our detailed advisory on the Commission’s proposal and our BioSlice blog posts on the Parliament’s and Council’s positions here and here).

The provisional agreement must now be formally adopted by both the Parliament and the Council.Continue Reading European institutions agree on the reform to the EU Regulatory Framework for Medicinal Products

A new policy paper released on 2 November 2025 sets out the MHRA’s proposals on overhauling and transforming the UK regulation of rare therapies.

The intention is for a “bold new rulebook for rare therapies” to be published in 2026. Rare therapies is defined as medicinal products intended to treat rare diseases, specifically, conditions with a prevalence of no more than 5 in 10,000. The Medicines and Healthcare products Regulatory Agency (MHRA)’s press release on the matter provides the concerning statistic that, while ≈3.5 million people in UK are affected by rare diseases (equivalent to one child in every classroom), and many more if carers are taken into account, only 5% of rare diseases have approved treatment. An intention of the reform is therefore to shorten the time for development of therapies for such conditions from initial discovery through to the stage of delivery to patients.

The MHRA aims to position the UK as a global leader in developing, regulating, and integrating rare therapies into healthcare. This policy paper outlines a new regulatory framework designed to accelerate access to innovative treatments for rare diseases while maintaining safety and evidence standards.

The initiative is supported by the newly formed Rare Disease Consortium, a collaborative effort made up of various different stakeholders including the MHRA, NICE, DHSC, NHS England, as well as patients and their representatives, academia and research institutions and industry.

A draft of the framework is anticipated to be available by Spring 2026, with external review in the first half of the year. A public consultation will also be conducted in 2026.Continue Reading Rewriting the Rulebook: MHRA’s Vision for Rare Disease Therapies

Introduction

On 4 June, EU Member States, meeting in the Council of the European Union (‘Council’), have agreed on a position on the pharmaceutical reform package.

This agreement was reached despite significant divergences between EU Member States in the preceding weeks. It marks a key milestone in the process for adoption of the EU revision of EU’s general pharmaceutical legislation, as it sets out the Council’s position for the trilogue negotiations to find a text agreeable to the Parliament and the Council, which can now begin.

The innovative pharmaceutical industry will welcome aspects of the Council’s adopted negotiating mandate as it provides greater certainty with regards to regulatory data protection and it lightens some of the obligations introduced in the European Commission proposal for reform of the EU pharmaceutical legislation adopted in April 2023 and the European Parliament’s position adopted on 10 April 2024 (see our detailed advisory). At the same time, the Council has adopted positions on some aspects of the proposals that could be seen as less favourable to industry.

We discuss some of the key provisions and changes in the Council’s adopted position below. This is, however, not covering all elements of the reform of the EU pharmaceutical legislation and the final outcome of the legislative process remains uncertain.Continue Reading Council of the European Union backs reforms to the EU Regulatory Framework for Medicinal Products