Partnering with Site Networks

Partnering with site networks offers a strategic solution to overcome the unique challenges of conducting clinical trials in rare diseases. Limited patient populations, complex recruitment pathways, and the geographic dispersion of expertise often hinder the progress of these trials. By leveraging established relationships, specialized knowledge, and streamlined processes, site networks can enhance trial efficiency, accelerate patient enrollment, and ensure high-quality data collection.

Rare disease drug development is one of the most rewarding but also one of the hardest paths in clinical research. The science is advancing rapidly, yet the practical hurdles remain stubborn: patients are few and often scattered across wide geographies, their diagnostic journeys can take years, and many trials never get off the ground simply because not enough patients can be enrolled. In fact, nearly one in three rare disease trials ends for this reason alone.

This is why site networks are gaining so much importance today. Rather than relying on individual sites or investigators, networks bring together multiple centers, patient registries, and specialist expertise under one umbrella. They change the picture entirely: making trials more coordinated, more reliable, and easier for patients to take part in. For sponsors, this shift is more than a tactical choice; it represents a new way of thinking about how rare disease trials should be run.

This article highlights why partnering with site networks is vital for rare disease clinical trials. It outlines key challenges with traditional site selection, the benefits networks provide such as improved patient access and efficiency, and offers guidance on how sponsors can collaborate effectively with them.

2. Why traditional site selection struggles in rare diseases

Traditional site selection usually means choosing a few hospitals or investigators based on experience or existing connections. While this can work in more common conditions, it is rarely effective in rare diseases. The main challenges include:

2.1 Scattered and small patient pools

Although rare diseases together affect an estimated 300 million people worldwide, each condition on its own is extremely uncommon. In the EU, a rare disease is defined as affecting fewer than 1 in 2,000 people; in the U.S., fewer than 200,000 individuals. This means that even when patients exist, they are spread thinly across countries and regions. For trial sponsors, finding enough eligible participants in one location is almost impossible without a broader, more coordinated approach.

2.2 Diagnostic delays and misdiagnosis

Reaching the right diagnosis is often a long journey. Studies show that patients typically see several doctors and may receive multiple incorrect diagnoses before the right one is made. For some families, this search for answers stretches into decades. Such delays not only affect patient care but also limit the number of people identified in time to take part in clinical trials.

2.3 Strict eligibility criteria

Rare disease studies often have very specific inclusion requirements, such as a confirmed genetic mutation or a defined biomarker profile. While necessary for scientific rigor, these criteria further shrink the number of patients who qualify. Even among those diagnosed, only a small fraction may meet all the conditions needed to enroll.

2.4 Geographical burden

Patients with rare diseases are rarely concentrated near specialist centers. Many must travel long distances, often over 100 miles each way, to reach a trial site. For families already managing complex health needs, this travel can be a heavy burden in terms of time, cost, and physical effort.

2.5 High trial burden

In addition to travel, trial designs themselves often demand a lot from participants. Multiple site visits, invasive procedures, and lengthy follow-up commitments can place significant physical and emotional strain on patients and caregivers. In rare disease settings, where many patients are children or have advanced health challenges, this level of burden makes retention particularly difficult.

What this means: While rare diseases are receiving increasing regulatory and commercial attention, illustrated by the fact that 52% of FDA drug approvals in 2024 were for rare diseases, recruitment challenges continue to hinder progress.

These challenges are exactly what site networks are designed to close.

3. Strategic advantages of partnering with site networks

Site networks offer a different model: instead of relying on isolated sites, they bring together groups of specialized centers that share patients, expertise, and infrastructure. This collective approach not only makes recruitment more reliable but also strengthens trial quality and reduces operational risk. In practice, sponsors working with networks gain several major advantages.

3.1 Enhanced patient access and recruitment success

By pooling patients across multiple specialized centers, site networks make it possible to reach populations that would otherwise remain inaccessible. The Rare Diseases Clinical Research Network (RDCRN), for example, has enabled more than 80 clinical trials by connecting hundreds of sites worldwide. Case studies also show that sponsors working with networks can expand site count quickly and improve recruitment trajectories, particularly when combined with advocacy group partnerships and digital outreach.

3.2 Concentrated expertise and standardization

Rare disease research depends heavily on specialist knowledge. Networks bring this expertise together, linking experienced investigators and standardizing approaches across sites. This ensures consistent quality in trial conduct, improves the reliability of data, and reduces the risk of protocol deviations.

3.3 Accelerated timelines and reduced costs

Network-based approaches can significantly shorten recruitment and study timelines. By relying on shared infrastructure and pre-established collaborations, sponsors avoid many of the delays that come with starting each site from scratch. Some analyses show that networks can reduce timelines by several weeks and cut study costs by over $1 million.

3.4 Centralized administration

Networks often handle contracts, ethics approvals, and safety reporting centrally. This reduces duplication, accelerates start-up timelines, and ensures greater consistency across all sites.

3.5 Cost efficiency and flexibility

By relying on high-performing sites within the network, sponsors can reduce overhead, avoid unnecessary duplication, and achieve economies of scale. Networks also provide the flexibility to adjust quickly if protocols or recruitment strategies need to change.

3.6 Expanded geographic reach and diversity

Networks extend access to patients in different regions and underserved communities, improving trial inclusivity and meeting regulatory expectations around diversity and representation.

3.7 Support for decentralized and hybrid approaches

Many networks have the capacity to support home visits, telehealth, or remote monitoring, which reduces travel demands on patients and makes trial participation more feasible.

Together, these advantages explain why site networks are increasingly seen as a critical part of rare disease strategy. The next step is to look at real-world examples that show this impact in practice.

4. Illustrative examples of network impact

The benefits of site networks are most clearly seen in real-world practice. Different rare disease networks show how collaboration changes trial outcomes:

RDCRN (US): Over 20 years, 19 consortia and more than 140 advocacy groups have supported studies in over 200 rare diseases, contributing to FDA approvals and thousands of publications.
TREAT-NMD (Global): A network of more than 100 centers across 54 countries that coordinates registries and improves trial readiness in neuromuscular disorders.
CDCN (Patient-led): Developed natural history registries and a biobank that accelerated diagnostics, enabled biospecimen access, and supported life-saving therapies.
RDCTN (Ireland): Connects patients, researchers, and industry across Ireland with a strong focus on patient involvement and international trial collaboration.

Together, these examples show how networks, whether academic, global, or patient-driven, expand patient reach, improve trial design, and speed development.

5. Structured approach to engaging site networks

Working with a site network works best when approached with a clear plan. Sponsors should think about aligning their goals with the network’s strengths and setting up a structure that supports recruitment, operations, and data quality from the start.

Network assessment and selection (months 1–2): Phase I

Review networks based on disease focus, investigator expertise, and registry capabilities.
Consider infrastructure for virtual trial components and past regulatory track records.

Collaborative protocol development (months 2–4): Phase II

Involve patient groups early to shape patient-centric trial designs.
Align endpoints and outcomes with network expertise.
Establish shared operating procedures across sites.

Execution and continuous optimization (months 4+): Phase III

Use real-time recruitment tracking across sites.
Implement centralized data management systems.
Adapt protocols based on ongoing feedback from network partners.

6. The strategic imperative for partnering with site networks

Partnering with site networks add value at every stage of development, from patient identification to regulatory approval. They enable faster recruitment, stronger data, and lower operational risk. In rare diseases, where timelines are long and patient numbers are limited, these advantages often decide whether a trial succeeds or fails.

The impact of site networks can be seen across multiple dimensions:

Faster recruitment: Site networks enable recruitment up to four times faster than traditional site-by-site models.
Regulatory impact: Established networks have contributed to multiple FDA approvals.
Access to patients and expertise: Networks reach patients and investigators that are otherwise hard to engage, making trials more representative and patient-centric.
Reduced operational risk: Standardized protocols and operational experience minimize delays and trial failures.

As the rare disease market moves toward $426 billion by 2030, sponsors who embed networks into their development strategy today gain the strongest position to capture growth, reduce risk, and deliver life-changing treatments to patients sooner. We also have an article, Clinical Development Strategy in Rare Diseases, which explores key elements of successful rare disease trials, from site and investigator selection to trial design, feasibility, regulatory considerations, and innovative patient engagement approaches. You can check it out here.

7. Conclusion for partnering with site networks in rare disease trials

With the rare disease market projected to exceed $426 billion by 2030, the opportunity is clear, but the challenges remain. Operational hurdles and science shape rare disease development equally. Site networks help bridge this gap by turning scattered patient populations and complex trial designs into workable, reliable programs. For sponsors, the real decision is not if networks should be part of their strategy, but how quickly they can integrate them.

Do you want to understand how to connect with site networks, which networks are most relevant for your disease area, and how early engagement can improve patient access and study planning?

These are questions many sponsors encounter as they design and run rare disease trials. If you are exploring these issues, feel free to reach out. We work with sponsors in rare disease development and can guide you in choosing the right way to engage with networks, so your studies start aligned with the right partners, the right patients, and a clear strategy from the outset.