Global regulatory standards

For pharmaceutical companies preparing for global expansion, launching in both the US and EU, the UK market often seems like the natural next step. Yet success in one region does not guarantee success in the other. While regulators may share high-level principles, the US, EU and UK operate under different systems, with distinct expectations for clinical evidence, timelines and commercial readiness.

Clinical programmes must do more than meet regulatory standards – they must generate the ‘right data for the right market at the right time’. Regulatory harmonisation remains a goal, but region-specific strategies are essential for successful product launch.

The US remains the world’s largest and most commercially attractive market for new medicines. Its regulatory framework, led by the Food and Drug Administration (FDA), offers streamlined and well-defined pathways such as the New Drug Application (NDA) and Biologics License Application (BLA).

The FDA places strong emphasis on clinical efficacy and safety, and offers fast-track mechanisms that can bring therapies to market at pace – particularly for areas of high unmet need. This flexibility extends beyond approval: medicines can often begin generating revenue while further data is collected through real-world evidence (RWE) or post-marketing studies.

From a clinical trial perspective, sponsors aiming for the US typically focus on:

• Early FDA engagement to align on endpoints and study design
• Robust efficacy and safety data from pivotal trials
• RWE strategies to support broader adoption post-launch
• Digital health data and outcomes that strengthen payer conversations.

In contrast, launching in the EU presents a more complex path. While the European Medicines Agency (EMA) provides a centralised approval route, access to patients is ultimately decided country by country through individual health technology assessment (HTA) processes, pricing negotiations and local reimbursement decisions.

‘Each market demands its own strategy, beginning at the clinical development stage’

The new EU HTA Regulation (EU 2021/2282), which came into full effect on 12 January 2025, marks a major shift in how health technologies – particularly medicines – are evaluated across Europe. It introduces a joint clinical assessment (JCA) process designed to streamline and harmonise evaluations across member states. As of January 2025, JCAs apply to new cancer medicines and advanced therapy medicinal products (ATMPs).

Each member state evaluates medicines against its own criteria. For example:

• Germany’s Gemeinsamer Bundesausschuss (G-BA) may require comparative effectiveness data and economic modelling
• France’s Haute Autorité de Santé (HAS) values real-world outcomes and quality-of-life measures
• Spain’s regional authorities expect budget impact analyses and often prefer established distribution networks.

• Comparator selection that reflects local standard of care
• Patient-reported outcomes relevant to national value assessments
• Subgroup analyses that support regional reimbursement arguments
• Language- and country-specific adaptations for later value dossiers.

Since leaving the EU, the UK has emerged as a standalone regulatory environment. The Medicines and Healthcare products Regulatory Agency (MHRA) now operates independently from the EMA but remains scientifically aligned with both the EMA and FDA.

For sponsors, this presents both challenges and opportunities. The UK offers:

• Expedited regulatory timelines that are often more agile than the EU
• A highly integrated NHS, enabling efficient recruitment and long-term follow-up
• Access to real-world data through electronic health records and linked data sets
• A favourable environment for early-phase and adaptive trial designs.

• NICE-specific evidence needs, including health economic models and quality-adjusted life years (QALYs)
• Alignment with the MHRA through scientific advice, particularly for novel therapies
• Utilising the NHS infrastructure for pragmatic, real-world-relevant designs
• Global relevance of UK data, which is still widely accepted by both the EMA and FDA.

Understanding the commercial landscape informs how trials should be designed and sequenced.

In the US:

• Launch nationally, build prescriber and payer engagement
• RWE strengthens market position post-approval
• Speed to market can provide first-mover advantage.

• Secure reimbursement before widespread uptake
• HTA and pricing approvals vary by country
• Tailored evidence and local engagement are essential.

• Independently but globally aligned
• Efficient regulatory pathways and integrated healthcare system
• Real-world data generation and cost-effectiveness evidence matter.

‘Region-specific strategies are essential for successful product launch’

Each market demands its own strategy, beginning at the clinical development stage. Trial design, endpoint selection and site strategy must reflect these diverging regulatory and commercial priorities.

To avoid costly delays and duplication of effort, sponsors should:

• Design trials with global goals: incorporate endpoints relevant to FDA, EMA, MHRA and HTA expectations. Include health outcomes, economic data and utility measures from the outset
• Run regulatory pathways in parallel: plan for concurrent interactions with the FDA, EMA and MHRA. Harmonise core data packages while accounting for regional variations in reporting and documentation
• Build market access into study protocols: clinical programmes should support pricing and reimbursement as well as approval. Select comparators carefully, include resource use data, and ensure follow-up captures real-world effectiveness
• Think nationally, act locally: localise evidence and engagement strategies. Develop value dossiers tailored to national payer priorities. Engage country-specific key opinion leaders (KOLs), advocacy groups and clinical leaders early.

The earlier you align your clinical strategy with your launch goals, the stronger your position will be across the US, EU and UK.

In today’s fragmented but interconnected markets, medicines don’t just need to be safe and effective, they need to be strategically positioned. And that begins at the trial stage.

Richmond Pharmacology is a UK CRO specialising in delivering trials that support global ambitions while accounting for regional complexities. From protocol development to data delivery, our focus is on building flexible, rigorous programmes that accelerate both regulatory approval and market access.

Lisa Campbell is Director of Regulatory Strategy at Richmond Pharmacology