New EU pharmaceutical legislation: Strategic implications for drug developers

This blog is part of The Regulatory Navigator series, where we explore the evolving regulatory landscape with actionable insight from Parexel's experts, sharing their experience to maximize success for clinical development and patient access.

The European Union has recently adopted a sweeping reform of its pharmaceutical legislation, marking the most significant regulatory update in over twenty years. This legislative package, often referred to as the "Pharma Package", was finalized in December 2025 and is designed to modernize the regulatory framework governing medicinal products across the EU.^1,2 The changes aim to accelerate patient access to innovative therapies, strengthen supply chain resilience, and embed sustainability into the lifecycle of medicines. Among the most notable updates are: 

1. Acceleration of the review timelines 
2. Revised exclusivity periods 
3. New obligations for equitable medicines access across all Member States 
4. Mandatory environmental risk assessments (ERAs) 
5. Regulatory sandboxes and structural changes 

These reforms are not isolated; they align with recent announcements from the European Medicines Agency (EMA) emphasizing digitalization, efficiency, and environmental responsibility in regulatory processes. Together, these developments signal a fundamental shift in how pharmaceutical companies operate in Europe. The newly adopted EU pharmaceutical legislation is more than a regulatory update; it is a catalyst for profound change in drug development. While headlines have focused on shortened review timelines and revised exclusivity periods, the consequences reach deep into trial design, operational execution, evidence strategies, and environmental aspects. Where complexity is already high and patient populations are often fragile, e.g., in oncology trials, these changes demand a new level of agility and foresight. 

The newly adopted EU pharmaceutical legislation is more than a regulatory update; it is a catalyst for profound change in drug development.

1. Acceleration of the regulatory review timelines 

One of the most immediate shifts is the acceleration of centralized review timelines from 210 to 180 days. This compression means that pivotal trials must deliver submission-ready data packages earlier than ever before. In oncology, where adaptive designs and biomarker-driven cohorts are increasingly common, sponsors will need to lock databases and finalize analyses on a tighter cadence. Consider a Phase III trial in metastatic non-small cell lung cancer (NSCLC) using an adaptive design to enrich for PD-L1–high patients, mid-study. Under the new timelines, interim analyses and cohort adjustments must be executed flawlessly, with statistical rigor and data integrity maintained throughout. Any delay in cleaning imaging data or reconciling adverse event narratives could jeopardize the ability to meet accelerated submission windows. 

2. Revised exclusivity periods 

The legislation recalibrates incentives, with baseline regulatory data protection set at eight years and market exclusivity reduced to one year, extendable under specific conditions. For sponsors, this underscores the importance of strategic evidence generation. For example, a company developing a novel checkpoint inhibitor for melanoma might consider head-to-head trials against standard-of-care therapies to secure additional exclusivity time. Similarly, programs targeting rare oncologic indications, such as sarcomas, need robust natural history data and long-term follow-up commitments to maintain orphan designation benefits. In other words, a comprehensive and well-thought integrated evidence generation planning (IEGP) is now more important than ever, and will be a key to success.  

3. Equitable medicines access across all Member States 

The legislation introduces obligations to ensure medicine availability across all EU Member States, such as new requirements compelling marketing authorization holders (MAHs) and public health authorities to collaborate in safeguarding the continuous availability of critical medicines within the EU. It strengthens companies’ obligations to ensure uninterrupted supply; mandates advance notification of shortages and withdrawals alongside the implementation of shortage prevention plans for prescription medicines. It also establishes systematic monitoring of both anticipated and actual shortages by the EMA and national competent authorities based on industry notifications. Additionally, it provides for the creation of an EU wide list of critical medicines to support targeted assessments of supply chain vulnerabilities. 

This requirement reshapes trial geography and post-approval planning. For example, sponsors developing CAR-T therapies for hematologic malignancies will need to demonstrate not only efficacy and safety but also the feasibility of delivering these complex treatments in smaller Member States with limited infrastructure. This could influence site selection strategies, pushing sponsors to include centers in countries that historically have not participated in advanced therapy trials. It also raises operational challenges: scaling logistics, qualifying local apheresis units, and ensuring GMP compliance across borders, all while maintaining trial integrity. 

4. Mandatory Environmental Risk Assessments (ERAs) 

Environmental sustainability adds another dimension. There is evidence to suggest that the production, use and improper disposal of pharmaceuticals potentially have negative impacts on the environment.³ The proposed new measures are targeted amendments preserve the objectives of the existing regulations and earlier commitments⁴ to maintain and safeguard a high level of protection of health and the environment. With the new legislation, inadequate assessment of environmental risks or proposals for mitigation of those risks could lead to a refusal of authorization. The assessment will be extended to consider the entire lifecycle. For instance, mandatory ERAs will require sponsors to evaluate the ecological impact of manufacturing processes, packaging, and clinical operations. As an example, for large-scale trials involving thousands of patients and frequent shipments of investigational medicinal products (IMPs), sponsors may need to rethink packaging configurations or consolidate shipment schedules to reduce waste. These considerations must be integrated early into protocol development to avoid last-minute changes that could disrupt timelines. 

Despite the increased regulatory burden, the proposed revisions to the legislation still aim to simplify procedures and foster innovation. Hence, sponsors of clinical trials that concern advanced therapy investigational medicinal products (ATMPs) containing or consisting of genetically modified organisms (GMOs) which present no or negligible risks, may not be required to submit an environmental risk assessment.⁵ Instead, sponsors will likely need to submit a declaration to clarify the categorization of ATMP and the lack of environmental risk as part of the clinical trial application. 

Antimicrobials will be subject to more stringent risk assessments, where careful evaluation of the manufacture, use, and disposal of the product will be required to ascertain the potential for antimicrobial resistance (AMR) selection in the environment. Additional information will be required in the patient information leaflet, along with an awareness card to help reduce the risks of AMR. 

Companies should note that additional details of the studies performed to assess environmental impact will be included in the medicine's European Public Assessment Report (EPAR). Moreover, it should be noted that for legacy products, the new rules will likely introduce obligations for medicinal products authorized prior to October 30, 2005, requiring MAHs to submit ERAs.  

5. Regulatory sandboxes and structural changes 

The revised legislation places the regulatory sandbox at the heart of the EU's strategy to encourage more agile and innovation-friendly oversight of medicines. Rather than relying solely on traditional, prescriptive rules, the sandbox creates a supervised space where developers can work with regulators to try unconventional scientific methods, digital tools, or manufacturing approaches which do not fit easily into the current regulatory framework. This arrangement is meant to help both sides understand what regulatory adaptations might be needed. By embedding this flexibility directly into the regulatory system, the EU aims to shorten the path from scientific innovation to real-world application while still maintaining strong safeguards for patients. 

The restructuring of the EMA's committee system represents one of the most organizationally significant elements of the reform. The new framework consolidates the existing human medicines committees into a smaller, more coherent structure, concentrating expertise and reducing procedural duplication. By streamlining scientific evaluation and simplifying governance, the EU aims to create a regulatory environment that can deliver faster, more consistent assessments while still drawing on a broad base of specialist knowledge. Significantly for ATMPs, the multidisciplinary Committee for Advance Therapies (CAT) has been taken out of the legislation, and its function will be subsumed by various working parties, with details still awaited. 

The reform also reshapes how the EU handles early access to medicines in that it updates and integrates the compassionate-use framework better into the overall framework. However, it still does not impose new obligations on Member States to operate such schemes, which is somewhat of a drawback. 

Lastly, the legislation introduces a new mechanism that allows the EMA to issue recommendations on whether a product should be considered a medicinal product in the first place. This represents a significant shift from the previous framework, under which the Agency had no authority to determine a product's fundamental regulatory status, and the CAT could only classify advanced therapies once they were already accepted as medicines (a national competency). By enabling centralized recommendations on borderline products, and by linking this process to the new legislation on tissues, cells and blood, the reform provides developers with clearer insight into the regulatory pathway they will need to follow. This is expected to reduce uncertainty, avoid divergent national interpretations, and support more coherent planning well before a marketing application is prepared. 

Moving forward – practical actions  

The new EU pharmaceutical legislation is a transformative moment for drug development. It offers a chance to deliver therapies to patients faster, meet sustainability goals, and strengthen competitive positioning. But success requires decisive actions and engaging with partners who understand the nuances of European regulation. The timeline for implementation is aggressive, with key provisions taking effect in 2026 and transitional measures extending into 2027–2028.  

To avoid delays and capitalize on new opportunities, we recommend that sponsors consider: 

• Immediate actions: Audit your portfolio to identify programs exposed to compressed timelines. For late-stage assets, accelerate database lock and prepare key analyses ahead of submission. Prioritize adaptive trial designs, such as basket trials using “Bayesian borrowing” for rare solid tumors. 
• Medium-term priorities: Strengthen supply chain resilience for pan-European availability. Include sites in smaller Member States to generate real-world evidence for HTA submissions. For CAR-T and advanced therapies, validate logistics pathways early. Embed environmental measures into protocols: consolidate shipments, use sustainable packaging, and leverage digital pathology. Document these in risk management plans to meet ERA requirements. 
• Long-term strategy: Design trials that deliver comparative evidence against current standards of care to unlock additional exclusivity periods and strengthen payer negotiations. Incorporate real-world evidence through pragmatic trials or registry-based studies. 

Early adoption helps sponsors navigate compliance requirements and avoid potential penalties and delays. Those that act decisively can leverage incentives through a new voucher scheme and position themselves for a potential guaranteed revenue scheme.^6,7 

If you want to future-proof your programs in the EU and turn legislative change into strategic advantage, connect with Parexel's regulatory and clinical consulting team today. Our differentiated approach, rooted in evidence, experience, and commitment to partnership, positions our clients to thrive under the new framework. Together, we can ensure compliance, accelerate development, and deliver meaningful outcomes for patients across Europe. 

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References 

1. EMA, News: EMA welcomes political agreement on new EU pharmaceutical legislation, December 2025 
2. EMA, Information: Reform of the EU pharmaceutical legislation, status as of February, 2026 
3. European Commission, Frequently Asked Questions: revision of the Pharmaceutical legislation, April 2023  
4. European Commission, European Union Strategic Approach to Pharmaceuticals in the Environment, March 2019 
5. European Commission, European Biotech Act, December 2025 
6. Council of the European Union, press release: “‘Pharma package’: Council and Parliament reach a deal on new rules for a fairer and more competitive EU pharmaceutical sector”, December 2025 
7. European Commission, Key elements of the EU pharmaceutical reform, December 2025 

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