Global by default

For many CGT companies, the U.S. has long been the default starting point for new ventures. Biotech and pharma companies have traditionally built commercial strategies around the primacy of the American market, prioritizing the FDA pathway and U.S. funding streams, and treating other regions as secondary opportunities once a product had clinical and commercial momentum.

In recent years, that model appears to be weakening, as more companies look beyond the U.S. earlier in their development. It’s a shift that has major implications for patients and sponsors, who will increasingly need to embed a global perspective into their access planning and commercialization strategies from the outset.

A broader global footprint

But firstly, how significant is the movement away from the U.S.? Latest biopharma data provide some insight into how early-stage activity has developed — and although these figures are not CGT-specific, they do show some important changes to market patterns.

For example, a study by Georgetown researchers found that the US share of global early-stage drug development programs fell from about 48% in 2015 to just over 37% in 2024, while China’s share rose from 8% to just over 32% over the same period.¹

Australia has also become an important focal point. More than 18,000 registered trials recruited participants there between 2006 and 2020, with 40% of these trials taking place between 2016 and 2020, according to an analysis published in the Medical Journal of Australia.²

Similarly, Brazil has been strengthening its clinical research and advanced therapy environment through regulatory reforms and incentives intended to support clinical development. And these trends extend to the Middle East too, where analysts predict the clinical trials market will grow from about $638 million in 2024 to $1.17 billion by 2033³ — with Saudi Arabia recently reporting a 53.4% year-on-year increase in clinical trials during 2025 alone.⁴

Of course, these figures need to be put in context, as the U.S. continues to account for just over half of global clinical trials and remains a dominant presence in the market. Even so, the direction is clear: The geography of CGT development is slowly broadening out and shifting beyond the prevailing U.S.-first model.

Why sponsors are looking beyond the U.S.

So why is this happening?

The reasons are both practical and commercial. Early-stage CGT research is highly sensitive to speed, cost and patient access, and different markets offer different advantages, including faster trial setup, lower development costs and investment in local infrastructure. For sponsors, these factors can shorten timelines, reduce risk and help test whether a therapy can be developed and delivered beyond its first approval market.

At the same time, other hard-edged commercial imperatives are also forcing their hand. Stung by early failures in CGT, investors are no longer satisfied with a strong scientific case. Before they commit to early research, they increasingly want to understand where the most commercially relevant patient populations are, how they will be identified and referred for treatment, what evidence will support reimbursement, and how the product will be manufactured and delivered across markets.

This commercial scrutiny is fundamentally changing how sponsors think about their geography, since early development decisions strongly influence how effectively the product can be marketed globally. Evidence requirements are a case in point. If the development stage is heavily U.S.-centric, companies may generate strong FDA-aligned data with limited relevance for EU health technology assessment bodies or other global regulators. Even in markets offering accelerated routes that take account of FDA or EMA approvals, including Saudi Arabia’s verified pathways, those routes still require local regulatory, pricing and stakeholder engagement and do not remove the need to plan for evidence that supports access after approval.

The risks and benefits

A more globally representative approach to early-stage development may bring some important benefits. It could potentially broaden access by bringing research closer to the populations most affected. For rare and genetic conditions concentrated in specific regions, running trials locally can create a more representative evidence base and ensure affected populations are directly included.

It may also help to strengthen site capabilities, referral pathways and diagnostic readiness across other parts of the world. This would overcome a key barrier in CGT trials — limited site feasibility outside the most established healthcare markets — potentially allowing enrollment to extend across small, dispersed patient populations.

Another potential advantage is that it requires companies to validate their operational model earlier, particularly around manufacturing, logistics and cross-border supply. In practice, this reduces the need to retrofit supply chains or commercial strategies post-approval, a common source of delay and inefficiency when scaling up.

Yet these shifts also carry additional risks and complexities. Operating across multiple territories can be particularly challenging in CGT because advanced therapies depend on time-sensitive logistics. Companies entering new environments may also have to navigate fragmented GMP, GDP and import/export rules, and must also assess whether local treatment centers have the trained staff, infrastructure, quality systems and patient-management processes needed to deliver highly specialized CGTs safely.

Pricing and reimbursement planning also need to move earlier in the process. CGTs often carry high upfront prices and many health systems require clear evidence of value, durability and budget impact before access can be secured. For sponsors, this means the economics of the product cannot be left until launch — they will need to understand early on how price, evidence, affordability and patient access will fit together in each geography to present a strong case.

Manufacturing adds a further pinch point, especially where models rely on centralized production, as capacity issues can emerge quickly once demand begins to increase. Many companies have to build fully integrated systems that provide real-time visibility across the patient journey, product movement and process milestones across different markets.

Finally, the speed of CGT development raises the stakes. Some therapies have reached approval on relatively compact phase 1/2 or early pivotal datasets, leaving little time to build the access and commercial model once the clinical case is established. If this work is left too late, companies may secure approval without being ready to supply patients, creating clinical risk, reputational damage and lost commercial opportunity.

Global intent

In our view, these developments point to a future in which the separation between clinical strategy, commercial planning and operational delivery narrows rapidly. The traditional CGT commercialization model has often assumed that sponsors could build around a first major approval, usually in the U.S., and expand out commercially from there.

This approach is now becoming less tenable. As investors, regulators and health systems place greater pressure on sponsors to prove therapies can be financed, approved and delivered at scale, the path to successful CGT commercialization will increasingly depend on generating evidence, securing reimbursement, manufacturing reliably and reaching patients wherever the need exists.

We believe the future will therefore belong to those companies that embed a truly global perspective into their clinical, commercial and operational planning from the outset. 

References

1. Teber, K. (2026, March). Over the Past Decade, Early-Stage Drug Innovation Shifted Toward China, Georgetown Researchers Find. Georgetown University, School of Health.
2. Seidler, A.L. et. al. (2023, Aug). The changing landscape of clinical trials in Australia. Med J. 219(5).
3. Healthcare IT Research Team. (2025, Sept.) Middle East Clinical Trials Market Size, Share & Trends Report. Grand View Research.
4. Saudi NIH. (2025). Health Research Performance in Saudi Arabia.