As more APAC biotech and pharma companies look to Europe for regulatory approval and commercialisation, navigating complex EU and UK requirements early is becoming increasingly critical. BioPharma APAC spoke with Catherine Moncad, Director of Commercial Services and VP of Regulatory Services at TMC, to understand the most common regulatory and market access challenges APAC companies face when engaging with the European Medicines Agency (EMA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA). Catherine shares practical insights on aligning clinical development with European expectations, integrating patient-centric approaches and preparing for regulatory approval and market access across Europe.

For APAC biotech and pharma companies developing novel therapies, what are the most common early-stage decisions that later become barriers when seeking EMA or MHRA approval?

Not undertaking a Paediatric Investigation Plan (PIP) procedure is probably the most common mistake we see for APAC companies; in fact, it’s probably the most common mistake overall. It is mandatory in both the EU and UK that an approved PIP is in place before a marketing authorisation application (MAA) can be validated, and this process can take up to a year. As a result, we frequently see companies that are otherwise ready to submit their MAA facing an entirely avoidable delay simply because the PIP has not been addressed early enough.  

This requirement also applies to conditions that are not thought to affect children. In those cases, companies still need to apply formally for a waiver and demonstrate why paediatric studies are not appropriate, unless the product falls into a category that is automatically waived. 

Another common misconception is that a company might feel they don’t need an approved PIP because they are focused on paediatrics and have already begun paediatric trials. If paediatric trials have taken place but are not aligned with the approved PIP, regulators are likely to request additional data. In the worst-case scenario, this can mean repeating a paediatric study, with significant implications for timelines, cost and resource allocation.

It should also be determined at an early stage whether the pharmacodynamics and pharmacokinetics of the drug in development differ between racial groups, particularly Asian and Caucasian populations. If there is evidence or a reasonable expectation that differences may exist, non-Asian patients should also be included in clinical trials. European regulators will expect to see data demonstrating that the safety and efficacy profile is applicable to a diverse patient population, and in some cases, this may require bridging studies to confirm comparability across ethnic groups.

How should APAC-based clinical development programmes be structured from the outset to avoid costly redesigns when European regulators assess data integrity, endpoints and population relevance?

It is wise to seek scientific advice from the EMA before starting Phase II development, and in some cases even earlier. If there are clear and well-established European scientific guidelines for a particular indication and these are being closely followed, formal advice may not be essential. However, we see a lot of novel therapies, particularly in rare diseases or complex oncology, coming from APAC, and such guidance either does not exist or leaves room for interpretation.

In these situations, early interaction with European regulators allows protocol design, endpoints and study populations to be assessed before substantial investment is made. We often see companies assume that alignment with US Food and Drug Administration (FDA) expectations will automatically translate to Europe, but this is not always the case, particularly when it comes to primary endpoints or acceptable clinical outcome measures. Engaging with the EMA early helps ensure that studies are designed to generate data that can support a positive benefit-risk assessment in Europe, even though approval can never be guaranteed.

It is also increasingly important to consider market access requirements at this stage. Designing trials that capture not only safety and efficacy data but also outcomes relevant to health technology assessment bodies can significantly reduce downstream challenges.

Europe places increasing emphasis on patient-centric evidence and advocacy. How can APAC companies meaningfully integrate patient engagement strategies early, rather than treating them as a late-stage compliance exercise?

APAC companies should engage with European patient organisations and advocacy groups as early as possible and involve them in protocol design. Patient groups can provide invaluable insight into what is realistic and acceptable for patients and caregivers in terms of visit frequency, procedures and study burden.

We often see protocols that are scientifically sound but practically unworkable from a patient perspective. For example, frequent hospital visits may be unrealistic for patients with limited mobility or those requiring full-time care. Early patient input can highlight these issues and encourage alternative approaches, such as home nursing visits or decentralised trial elements, which ultimately support better recruitment and retention.

Patient organisations can also act as a gateway to key opinion leaders (KOLs) and experienced investigators. These relationships strengthen trial design and can prove extremely valuable during regulatory interactions, where clinical and patient perspectives play an increasingly important role.

For small to mid-sized APAC companies without a European footprint, what does a realistic and effective regulatory engagement roadmap with EMA, MHRA and national authorities actually look like in practice?

For companies without an established presence in Europe, meaningful engagement with EU and UK regulators typically requires partnering with an organisation that is locally based and has proven regulatory expertise. Engaging an experienced European regulatory partner helps ensure that critical procedures, such as PIPs or scientific advice, are initiated at the right time and that costly missteps are avoided.

In practice, this engagement should begin once a company has established proof of concept and believes the product has genuine potential for international development. Ideally, this is around Phase 1B, when dosing, safety and early efficacy are being explored, allowing European considerations to be addressed in parallel with APAC development activities.

Early engagement also allows companies to optimise regulatory strategies, manage fees and ask the right questions of authorities at the right time. The earlier this alignment begins, the greater the opportunity to de-risk later-stage development and approval.

Market access across Europe remains highly fragmented. How should APAC innovators prioritise countries and reimbursement pathways without overextending resources or delaying launch momentum?

Europe should not be viewed as a single, homogeneous market. Reimbursement pathways, standards of care and even trial feasibility can vary significantly between countries, which may influence both clinical development and launch sequencing decisions. Prioritisation should, therefore, be strategic, focusing on markets where clinical value can be clearly demonstrated and access pathways are realistic.

Market access planning in Europe must now also start much earlier than many APAC companies expect. For all new oncology medicines and advanced therapy medicinal products (ATMP), a Joint Clinical Assessment (JCA) must be prepared and submitted at the time of MAA submission. For orphan medicinal products approved from 2028 onwards, this requirement will also apply, and by 2030, it will extend to all other new centrally authorised medicines.

This means that evidence required by payers and health technology assessment bodies must be considered well before pivotal trials begin. We support clients in preparing JCA dossiers and, critically, in identifying what data payers will expect to see. This often includes demonstrating not only clinical benefit but also how a therapy may reduce long-term healthcare costs, such as by preventing hospitalisation or avoiding chronic disease management.

Beyond regulatory approval, what aspects of post-trial readiness — such as real-world evidence generation, supply planning and stakeholder communication — are most often underestimated by APAC companies entering Europe for the first time?

APAC companies are often highly innovative, particularly in rare diseases and oncology, but many underestimate what is required after approval. For therapies targeting small patient populations, it is not always possible to generate the volume of data needed for a full marketing authorisation. As a result, approvals are frequently granted under conditional or exceptional circumstances, with obligations to generate further data post-approval.

These obligations may include additional safety monitoring, new clinical or non-clinical studies, or further chemistry, manufacturing and controls (CMC) work. The outcomes of these activities must then be integrated into regulatory documentation and patient-facing materials, such as the Summary of Product Characteristics and Patient Information Leaflet. Real-world evidence generation is particularly valuable here, supporting both regulatory commitments and a company’s own understanding of long-term safety and effectiveness.

Supply chain readiness is another common blind spot. In the EU and UK, a company must hold a Wholesale Dealer’s Licence (WDL) to buy and sell its own product. This licence can take up to a year to obtain, and without it, commercial launch may be delayed even after regulatory approval is granted. Companies must also ensure that quality agreements and distribution pathways are fully defined and compliant.

Finally, engagement with reimbursement bodies must begin before approval. Without early dialogue, there is a real risk that a product may be authorised but unable to reach patients due to delays in pricing and reimbursement decisions. Together, these factors highlight why post-trial readiness should be treated as an integral part of the European entry strategy, not an afterthought.