EU regulatory activity is driving comprehensive reforms for pharma in 2025

Pharmaceutical regulatory changes taking effect in 2025 and new legislative proposals across the region are touching upon the full lifespan of medicines –  from trials and drug development to health technology assessment and drug shortages.

These developments are poised to have substantial repercussions on biotech and pharmaceutical businesses, trial sponsors and marketing authorisation holders. This will necessitate strategic adjustments to ensure compliance and leverage new opportunities.

EU and cross-border trials

The EU's approach to clinical trials is evolving along with the implementation of the Clinical Trials (CT) Regulation (EU) 536/2014 that came into full effect in late January.

Since 30 January, any clinical trial that has not transitioned to the new Clinical Trials Information System (CTIS) established under the CT Regulation will face corrective measures enforced by local healthcare authorities. Sponsors of these trials must now submit new applications through CTIS, as previous transition procedures are no longer available. The European Medicines Agency (EMA) announced that, throughout 2025, continuous improvements to CTIS performance and user experience are to be expected.

Cross-border clinical trials are also receiving increased attention. The EU-X-CT initiative, co-led by the European Forum for Good Clinical Practice and the European Federation of Pharmaceutical Industries and Associations (EFPIA), is crucial in facilitating access to clinical trials across Europe. This initiative is especially important for patients with rare or life-threatening diseases.

Draft EU-X-CT recommendations have been issued to address barriers to cross-border participation in trials, offering guidance to sponsors, investigators and trial sites to ensure smooth and effective trial management. The public consultation on these draft recommendations has been extended until the end of March, with EFPIA encouraging members to participate actively in the process. The target date for adoption is set for June this year.

At the same time, trials in advanced therapy medicinal products (ATMPs) continue to be at the forefront of EU regulatory developments. The EMA's Committee for Advanced Therapy has recently published guidelines for investigational ATMPs. ATMPs can include gene therapy, somatic cell therapy, tissue-engineered products and ATMPs combined with medical devices. The EMA underscores the importance of well-conducted studies to generate reliable data for these complex products.

EU pharma package updates

The European Commission's 2025 work package has recently reaffirmed the ongoing overhaul of EU pharmaceutical legislation, positioning it as a key element in Europe's new strategy for sustainable prosperity and competitiveness. Reports from the European Parliament's Committee on Environment, Public Health and Food Safety  in  February indicated that the proposals are still "work in progress" at this committee level.

On 13 March, the Official Journal of the European Union published the Parliament's stance adopted at first reading on the proposed laws (C/2025/1328 and C/2025/1329). These freshly released texts solidify the position adopted on 10 April 2024, but they do not include any new amendments. The EU proposals, which affect key pharma regulatory instruments will continue to navigate through the Union's legislative process following the ordinary legislative procedure.

Shortages and critical medicines

Medicine shortages have become a pressing issue within the EU, exacerbated by global events such as the Covid-19 pandemic and geopolitical tensions.

The Commission's proposal for a Critical Medicines Act released this March aims to address these shortages by strengthening the supply chains for critical medicines. The draft act focuses on diversifying manufacturing capacities, incentivising supply chain resilience, and leveraging collaborative procurement to ensure the availability of essential medicines.

The European Shortages Monitoring Platform (ESMP) is another initiative aimed at addressing medicine shortages. The online platform provides real-time data on the availability of medicines, enabling authorities to take proactive measures to prevent and mitigate shortages. It is meant to facilitate the coordination of supply chain activities across Member States, ensuring a more efficient and responsive system.

The use of the ESMP became mandatory in February. Marketing authorisation holders and national competent authorities must rely on the platform to directly report information on supply, demand and availability of nationally and centrally authorised medicines during crises. It should also be utilised to support preparedness actions led by the EMA.

Substances of human origin

Another significant milestone that will be reached this year will be one of the initial regulatory deadlines tied to the Substances of Human Origin (SoHO) Regulation (EU) 2024/1938, ahead of its full application in mid-2027.

The SoHO Regulation, unveiled in June last year, sets forth stringent standards of quality and safety for all SoHO intended for human application, as well as activities related to these substances. It encompasses SoHO intended for human use and those utilised in the production of items governed by other EU legislation, such as medicinal products and ATMPs but also investigational drugs.

The primary goal is to ensure robust protection of human health, particularly for SoHO donors and recipients, by bolstering the continuity of supply for critical substances.

By 7 August, the Commission is tasked with the adoption of implementing legislation that will define the technical specifications for the EU SoHO platform. This will serve as a pivotal digital resource for authorities and stakeholders, streamlining data exchange and administration within the sector. Developed and hosted by the Commission, it will act as a central hub, offering access to information on registrations, authorisations, technical guidelines for professionals, and best practices for competent authorities across Member States.

Simultaneously, on the same day, the SoHO coordination board, established under the regulation, is charged with compiling a list of existing substances, products, or activities that require regulatory opinions under the SoHO regulation to mitigate potential risks.

Health technology assessment uptake

On 12 January, the Health Technology Assessment (HTA) Regulation (EU) 2021/2282 came into effect across the EU, debuting a new era of collaboration among Member States on health technologies, including specific medicinal products and certain medical devices.

This regulation mandates that certain medicines undergo joint clinical assessments by multiple EU countries to ensure their safety and efficacy prior to market entry. This includes new cancer treatments and ATMPs from 12 January this year, orphan drugs from 13 January 2028 and other medicinal products from 13 January 2030. The Commission has released scientific specifications of medicinal products that are subject to these assessments.

Complementing this framework, the Commission's implementing regulation (EU) 2024/1381, which is also effective from 12 January, delineates procedural rules for joint clinical assessments of medicinal products. This regulation aims to streamline the assessment process to enhance predictability and efficiency. It outlines the cooperation and information-sharing protocols with the EMA, the timing and interactions among various groups and experts, and the general rules for stakeholder consultations, including patients and clinical experts. Additionally, it specifies formats and templates for the information and evidence required from pharma and biotech developers, as well as the format for the final assessment reports.

Osborne Clarke comment

The EU's 2025 regulatory overhaul signals a transformative era for the pharmaceutical sector as  significant emphasis is placed on pre-marketing activities and drug shortages. This will have an impact on a broad spectrum of the industry, from marketing authorisation holders and biotech firms to advanced therapy companies, trial sponsors and contract manufacturing organisations.

With the full implementation of the CT Regulation and the transition to the CTIS, trial sponsors were required to act swiftly to ensure conformity and avoid corrective measures. The EMA guidelines for investigational ATMPs underscore the necessity for robust data generation, compelling biotech firms and other stakeholders in these cutting-edge fields to stay informed and adapt their strategies accordingly.

The updates to the EU's pharmaceutical package demand vigilance, throughout the full life-cycle of authorised medicines. The forthcoming Critical Medicines Act and the now-live ESMP mandate marketing authorisation holders to proactively report supply and demand data. Additionally, the upcoming SoHO Regulation and the existing HTA Regulation highlight the importance for businesses to align with new standards and participate in joint clinical assessments.

To successfully navigate these regulatory changes, life sciences businesses must remain vigilant, informed, and agile. By championing compliance and seizing emerging opportunities, they can position themselves advantageously within the dynamic EU regulatory landscape.