As we continue our exploration into how Europe represents a strategic opportunity for U.S. biotech startups developing therapies for rare diseases, it’s time to examine two highly impactful growth levers: clinical trial expansion in the EU and Early Access Programs (EAPs). Entering the European landscape can support startups’ development timelines and reduce costs while also opening up opportunities for earlier accessibility of novel treatments into national healthcare systems.
Why clinical trial expansion into the EU could boost your development
For biotech companies targeting rare diseases, patient recruitment is notably one of the biggest challenges in the drug development process. The fragmented and often slow recruitment makes it difficult to gather sufficient data in a timely manner. Designing rare disease clinical trials that include the EU can help address this issue on multiple levels.
- Faster recruitment, richer data
Given its large overall population, Europe offers access to a more heterogeneous patient pool. This population variety can contribute to accelerating and improving the recruitment phase for rare disease clinical trials, which can be especially tricky for low-prevalence diseases. Moreover, the inclusion of ethnically diverse communities would result in more robust and generalizable data, which can strengthen the overall scientific and regulatory case for both EMA and FDA submissions.
- Boost regulatory credibility
Clinical data collected in the EU under rigorous, harmonized standards does not only meet EMA expectations but also bolsters credibility with U.S. regulators. In many cases, comprehensive and well-structured data could reduce the need for additional FDA-requested studies, expediting the approval process in the U.S.
- Greater cost-efficiency
Conducting clinical trials for rare diseases in Europe can also be more cost-effective. With EMA-driven regulatory harmonization and cross-border infrastructure, studies can be executed at a lower cost compared to the U.S. (especially in Eastern Europe countries), all while maintaining high-quality standards. This operational efficiency is a significant advantage for early-stage biotech companies managing constrained budgets.
Early Access Programs: unlocking value before market authorization
In addition to clinical trials, EAPs represent another strategic pathway for U.S. biotech startups to accelerate market entry and address high unmet needs in Europe before full marketing authorization is even granted.
- Accelerated entry and early revenues
EAPs allow companies to provide access to innovative treatments for patients with serious conditions who have no other therapeutic options. Through these programs, orphan therapies can be introduced into national healthcare systems earlier than would otherwise be possible, thus significantly improving the patients’ quality of life by reducing delays. Additionally, some national EAPs also provide for the generation of early revenues that can further fund pivotal clinical studies or regulatory submissions.
- Generate real-world evidence (RWE)
Crucially, EAPs generate valuable real-world data on efficacy, safety and patient response. Given the intrinsic challenges with presenting gold-standard clinical data for rare disease studies, RWE can be a helpful tool to complement rare disease clinical trial results and overcome their limitations. In the European rare disease landscape, RWE is becoming increasingly more acknowledged for regulatory decisions, and it could significantly help an orphan drug’s regulatory status. Lately, the EMA and various national regulatory bodies have been working on implementing the use of RWE in their evaluations for the approval of new therapies, especially for those whose Pricing & Reimbursement dossiers require extra attention, as is often the case for rare disease medicines.
- Reduce commercialization risks and boost investor confidence
For investors and strategic partners, EAPs signal both demand and execution capability. Early revenue generation and clinical traction strengthen the commercial strategy. Moreover, applying for EAPs means gaining early access to a diversified market, which reduces the risks associated with a single-market strategy. Together, these factors contribute to making the company more attractive for funding, partnerships, or acquisition.
Looking ahead: from tactical tools to strategic advantages
For U.S. biotech startups in the rare disease space, expanding into Europe is not just about geographic diversification, it’s a strategic imperative. Clinical trials in the EU can speed up development and enhance regulatory data credibility, while EAPs generate real-world evidence and revenue that accelerate the path to market and strengthen reimbursement strategies. Together, they shorten time-to-impact, reduce capital loss risk and create a more compelling narrative for investors.
In today’s global biotech landscape, exploring these opportunities can make a difference to produce valuable impact for rare diseases.