EU Pharma Package: Key Takeaways for Clinical Evidence & JCA Strategy

Following the political agreement of 11 December 2025 on the EU Pharmaceutical Package, the summary below highlights the elements most relevant to clinical evidence generation and Joint Clinical Assessment (JCA) planning. This reform represents the most comprehensive update to EU pharmaceutical legislation in more than two decades and marks a structural shift in how innovation, market access, and evidence generation will be evaluated across Europe.

The agreement reflects a broader strategic objective to strengthen the EU’s competitiveness as a location for pharmaceutical research, development, and investment while addressing affordability, access, and supply resilience. For biopharmaceutical companies, this is more than a regulatory update. It reshapes how value is created, protected, and sustained in the EU market, with direct implications for investor confidence, asset valuation, and capital allocation decisions.

The overall direction is clear: greater emphasis on early, high-quality clinical development as the key to securing value and exclusivity. Market protection and differentiation will rely less on post-approval positioning and far more on trial design and evidence strategy developed early in the pipeline.

Why the EU Is Rewriting the Rules

Over the past several decades, pharmaceutical investment and R&D activity have increasingly shifted toward other regions, including the United States and parts of Asia. EU policymakers have acknowledged that regulatory complexity, uncertainty, and fragmented incentives have contributed to this trend, weakening the EU’s competitiveness as a location for pharmaceutical development.

The EU Pharma Package is intended to respond directly to these challenges. Rather than focusing solely on regulatory simplification, it seeks to recalibrate the balance between innovation, access, and supply resilience by streamlining certain processes while linking incentives more closely to public health priorities and demonstrable clinical value. The resulting framework reflects a new operating environment shaped by heightened global competition, in which early development decisions will play a greater role in determining regulatory protection, speed to market, and long-term commercial durability across Europe.

1. Accelerated Regulatory Approval - Confirmed 

The agreement streamlines the EMA evaluation process, enabling faster market entry.

• Assessment he rationale behind early protocol modifications

• Implications: Sponsors may benefit from faster approvals, but this will require stronger, more complete dossiers at submission to minimize clock stops and avoid downstream delays.

2. New Regulatory Data Protection Periods

The standard market exclusivity duration has been shortened, altering baseline assumptions for lifecycle planning

• Previous standard: 10 years (8 years data protection + 2 years market protection)

• New standard: 9 years (8 years data protection + 1 year market protection)

3. Recovering the Lost Year: The New "+1" Extension

Protection can still reach 10 years, but the extension is now conditional rather than automatic.

A company may gain +1 year of market protection if the product meets two of three criteria, likely including:

• Addressing a High Unmet Medical Need (HUMN)

• Conducting comparative clinical trials

• Containing a new active substance, meeting development-related conditions, and obtaining marketing authorization outside the EU within 90 days of submission inside the EU

Additional details:

• A further +1-year regulatory protection extension is possible if, during the data protection period, the MAH secures authorization for a new therapeutic indication that provides significant clinical benefit versus existing options.

• The maximum cumulative protection is 11 years.

Implication: Clinical Development Plans will need to be more strategically designed. Obtaining approval alone will no longer secure optimal exclusivity. Trials must be powered to demonstrate significant benefit or clearly meet unmet-need criteria, which are also central to payer and HTA assessments.

4. "Launch in 27 Member States" Incentive Removed

• The proposed +2-year exclusivity incentive for launching in all EU Member States has been removed.

• The Instead, Article 56a introduces the ability for Member States to require supply of medicines to address or prevent shortages, shifting emphasis toward supply reliability and public health needs.

5. Early Generic/Biosimilar Entry: Expanded Bolar Exemption

• The exemption has been broadened to allow generics and biosimilars to conduct studies, run trials, and file pricing and reimbursement applications or tender bids before the originator’s IP expires.

• Effect: Competitors can be positioned to launch immediately on the day of patent expiry, increasing pressure on originator products following loss of exclusivity.

6. Orphan Medicinal Products

Designation Criteria
A product qualifies if:

a) The condition affects no more than 5 in 10,000 people in the EU at the time of applicaiton, and

b) No satisfactory diagnosis, prevention, or treatment method exists in the EU.

Breakthrough Orphan Medicinal Products
These must:

• Have no authorized treatment in the EU, and

• Deliver a clinically relevant reduction in morbidity or morality.

Exclusivity Duration

• • 9 years for orphan medicines not covered by specific enhanced categories

  • 11 years for products addressing a high unmet medical need

  • 4 years for products authorized under Article 13 of the revised Directive 2001/83/EC

7.Regulatory Sandboxes for Innovative Therapies

The Commission may establish regulatory sandboxes on a case-by-case basis when:

a) Full compliance with standard medicinal product requirements is scientifically or technically infeasible, and

b) The characteristics of the product are reasonably expected to positively and distinctively improve quality, safety, efficacy, or significantly enhance patient access to prevention, diagnosis, treatment, or care.

These sandboxes are intended to enable early regulatory engagement for highly innovative therapies while maintaining appropriate safeguards for patient safety and evidence quality.

Overall Implications

The updated EU pharmaceutical framework strengthens the link between clinical evidence strategy, regulatory pathways, HTA outcomes, and long-term commercial value. Early decisions (including comparator selection, endpoint strategy, and the ability to substantiate unmet need) will now directly influence:

• Approval success

• Exclusivity duration

• HTA evaluation outcomes

• Competitive positioning across the EU

What This Means for Veristat Clients

Regulatory success in the EU will increasingly be determined by how well development programs are positioned early, scientifically, and operationally.

Conditional exclusivity, faster reviews, and earlier competitive entry mean that clinical development, regulatory strategy, and evidence planning can no longer be treated as sequential steps. They must be integrated from the outset.

Veristat’s experience across EU regulatory pathways supports sponsors in aligning clinical development, regulatory strategy, and evidence generation with evolving EMA expectations. This includes, between other regulatory activities, expertise across centralized procedures, EMA scientific advice, orphan and pediatric frameworks, and alignment with evolving HTA and Joint Clinical Assessment requirements.

For Veristat clients, this translates into:

• Clearer insight into how early trial design decisions affect EU protection and competitiveness

• Regulatory strategies built around conditional incentives rather than fixed assumptions

• Submissions prepared to meet shorter review timelines with fewer avoidable delays

• Integrated EU and global planning that minimizes divergence across regions

Preparing for What Comes Next

Once formally adopted, most elements of the Pharma Package are expected to apply within approximately two years. Additional legislation, including the Critical Medicines Act and the Biotech Act, will further shape the regulatory environment. Given the length of R&D and manufacturing cycles, this shortens the effective window for sponsors to adapt development and commercialization strategies.

Despite the transition period, development, manufacturing, and clinical trial design decisions made today will determine how well programs align with the future EU framework.

The EU remains one of the world’s largest and most influential pharmaceutical markets. Sponsors that understand how to operate effectively within this evolving regulatory landscape will be better positioned to sustain value, attract investment, and deliver therapies to patients across Europe.

Veristat continues to monitor developments closely and support clients with EU-focused regulatory intelligence, strategic planning, and execution as this reform moves from policy to practice.

Learn more about Veristat's global regulatory affairs, operations, and consulting expertise here.

Source: ‘Pharma package’: Council and Parliament reach a deal on new rules for a fairer and more competitive EU pharmaceutical sector

Additional information:

• European Parliament press release on reform of the EU pharmaceutical legislation

• European Parliament background note on elements of the provisional agreement

• Council press release on GPL deal