In rare disease studies, enrollment is not simply a logistical hurdle — it’s a human endeavor. Behind every data point is a patient who has often spent years searching for answers, navigating misdiagnoses, or managing life with limited treatment options.

In this context, successful recruitment and retention requires more than protocol alignment — it demands empathy, community partnership, and a commitment to meeting patients where they are. One of the most powerful ways to achieve this is through collaboration with Patient Organizations, including Patient Advocacy Groups. As sponsors and CROs continue to face long enrollment timelines and high dropout risks, the patient organizations as a key resource remains consistently underutilized.

At Confidence Pharmaceutical Research, we’ve seen firsthand how partnerships with patient advocacy groups, registries, and disease-specific research networks can transform the trajectory of a rare disease study. These organizations do more than amplify trial awareness—they help build the kind of trust, relevance, and long-term support that enrollment metrics alone can’t capture.

In this second installment of our series, we focus on the crucial interplay between clinical trial sites and patient networks, and how bringing them together early in the study process can enable faster, more sustainable recruitment and retention.

Clinical Sites Provide the Infrastructure—But Patient Networks Provide the Insight

Sites are usually the operational hubs of a study. They manage informed consent, deliver interventions, and collect outcomes data. But in rare disease trials, sites are also asked to identify and motivate patients within populations that are often fragmented, spread over a large area, hesitant, or historically underserved.

That’s where patient organizations come in.

These groups—ranging from small family-led foundations to national nonprofit coalitions—bring deep, lived experience of the disease landscape. Their contributions can include:

• Helping define outreach messaging that resonates with patients’ lived experiences
• Educating providers and caregivers about new diagnostic pathways
• Facilitating referrals through trusted community channels
• Supporting patients through the burden of participation, including emotional, logistical, and financial dimensions

According to Global Genes, nearly 70% of rare disease patients actively turn to patient communities for treatment information, highlighting the central role these groups play in navigating care and evaluating clinical trial participation [1]. Research shows that patients in rare disease communities are more likely to consider trial participation when they hear about it from a trusted patient group or peer network [2].

Building Trust Through Community Alignment

Trust is often the most significant barrier to enrollment in rare disease trials—especially in communities that have faced misdiagnosis, stigma, or unmet clinical needs for years. In many cases, sponsors are new to these communities, while patient organizations have been supporting them for decades.

Bringing Patient Advocacy Groups into the study planning process—early and authentically—can unlock several advantages:

• Improved protocol design: incorporating insights from patients and caregivers about what’s feasible in real life
• Enhanced recruitment materials: tailored, culturally competent materials co-developed with advocacy input
• Higher enrollment conversion: as messaging comes through trusted channels like support groups, social media communities, or patient newsletters
• Better retention: with Patient Organizations helping set expectations and providing ongoing engagement resources throughout the study

The National Organization for Rare Disorders (NORD) emphasizes that early engagement with advocacy groups leads to more inclusive trial designs, increased awareness, and better alignment with the real-world challenges rare disease families face [3]. The U.S. FDA has emphasized the importance of collecting comprehensive and representative patient input to inform clinical trial design, particularly in underserved and rare disease populations [4].

Case Study: How a Patient Advocacy Group Drove Enrollment in an Ultra-Rare Condition

Confidence was tasked with enrolling 100 patients in a Phase 2 U.S. trial for an ultra-rare hereditary dermatologic condition. With fewer than 500 adults diagnosed nationwide, enrollment required reaching more than half of the entire eligible population—a challenge that could not be solved by sites alone.

Seven academic centers were initially activated, each with access to only 7–15 patients. The rest of the eligible population was scattered across the country, many living far from study centers and facing the burden of complex travel.

To close this gap, Confidence partnered with a national Patient Advocacy Group that had long-standing trust within the community. Because the disease was hereditary, many families were already active in this organization. The group helped:

• Point patients to their nearest study site,
  
• Share trial information through trusted community channels, and
  
• Support families as they considered participation.

More than 70% of all randomized patients were ultimately referred by this advocacy group. To further improve access, Confidence worked with the sponsor to open three additional community-based commercial sites in regions with no academic coverage. Supported by travel coordination and mobile nursing visits, these new sites often enrolled and retained patients more effectively than the original academic centers.

The outcome was a study that reflected true patient partnership: the advocacy group brought reach and trust, Confidence removed logistical barriers, and sites provided clinical oversight. Together, this collaboration made it possible to enroll a population that otherwise would have been out of reach for traditional trial models.

Confidence’s Framework for Engaging Patient Organizations

At Confidence, our rare disease operations team incorporates Patient Advocacy Groups engagement into every phase of the clinical development lifecycle. We view these partnerships not as optional outreach, but as core components of a viable trial strategy.

Our typical approach includes:

• Pre-activation partnership mapping: identifying and initiating contact with key patient organizations prior to site selection
• Protocol feasibility discussions with patient advisors: incorporating caregiver and patient feedback into study design before submission
• Joint development of recruitment materials and patient FAQs
• Ongoing communication throughout the trial: including co-hosted webinars, progress updates, and feedback loops for continuous improvement

We’ve seen this model shorten enrollment timelines significantly in certain indications and reduce mid-study dropout rates by as much as half—particularly in trials with heavy caregiver burden or complex eligibility requirements.

What’s Next in the Series?

In our next post, we’ll explore another essential lever for rare disease enrollment: the strategic use of advertising campaigns and external patient recruitment vendors. While these tools are often associated with large-scale trials, they can be highly effective in rare indications—when used thoughtfully and in alignment with site capabilities and patient needs.

Until then, remember: in rare disease research, every connection counts—and no one connects patients to trials more meaningfully than the communities that support them.

References

1. Global Genes. (2021). Engaging with Patients in Rare Disease Clinical Trials. Retrieved from https://globalgenes.org/toolkits/engaging-patients-clinical-trials/
2. EURORDIS. (2018). Rare Barometer Survey: Clinical Trials and Rare Diseases. Retrieved from https://www.eurordis.org/publication/rare-barometer-survey-clinical-trials-and-rare-diseases/
3. National Organization for Rare Disorders (NORD). (2020). Patient Engagement in Rare Disease Clinical Trials. Retrieved from https://rarediseases.org
4. U.S. FDA. (2023). Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. Retrieved from https://www.fda.gov/media/139088/download