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Transforming Rare Disease Clinical Trials: The Power of Digital Platforms

Clinical Trial Management

A cornerstone of medical progress, clinical trials lead to treatments and innovations that improve the lives of countless people suffering from a wide range of health conditions. However, when it comes to rare diseases, these trials present unique and formidable challenges.

Small patient pools, difficulties in enrollment and retention, and unclear outcome measures make it difficult for traditional clinical trial platforms to conduct impactful medical research in support of rare disease patients and their families

In this article, we consider the obstacles faced in rare disease clinical trials and its most promising solution: full-service digital platforms.

Small Participant Populations

Millions of patients globally suffer from over 7,000 – 10,000 rare diseases, many of which lack established standards of care. Even well-known conditions, like cystic fibrosis, fall under the rare disease umbrella due to their complex genetic variations that determine treatment strategies. Nevertheless, clinical trials often face recruitment challenges because many participants cannot reach clinical trial sites, or participants are misaligned with trial criteria.

Enrollment and Retention Obstacles

High dropout rates are prevalent in rare disease clinical trials, often causing delays and, in some instances, rendering them unviable.

According to the Medical Research Network (MRN), the average distance from clinical sites was 177 miles in the United States and 136 miles in the rest of the world. For rare disease patients and their families, driving hundreds of miles round trip for clinical trial appointments isn’t feasible.

Furthermore, the severe physical limitations in some rare disease patients may require caregiver support, which may not always be accessible. These physical disabilities can, in turn, hinder compliance with complex trial protocols

Unclear Clinical Endpoints

Unlike more common diseases, rare diseases typically lack established treatment pathways and clearly defined clinical endpoints. This absence of prior data makes it challenging to design and implement clinical trials effectively. Patients and caregivers need reassurance that their participation will contribute to meaningful progress in treating the disease.

A New Era in Rare Disease Research Has Begun

To address the challenges in this field, sponsors need a patient-centric, full-service digital solution that adapts to the unique needs of rare disease patients and their families. Here’s how purpose-built digital platforms can revolutionize hybrid and DCTs in rare disease research:

Remote Participation

An array of digital tools makes it easier and simpler for rare disease patients to participate in medical research. Digital platforms seamlessly enable remote participation, removing geographical barriers and empowering participants to contribute to medical research, regardless of their location or physical limitations.

Patient-Centric Approach

By bringing clinical trial visits to the participants’ homes or local clinics, we reduce the physical and emotional burden on rare disease patients and their caregivers. This patient-centric approach not only makes participation more accessible but also fosters a sense of trust and commitment.


Full-service digital platforms can handle multiple tech integrations and facilitate user-friendly televisits, making it easier for participants to consult healthcare providers. This can foster meaningful connections between participants and their trial team.

Global Recruitment

Rare disease population sizes make recruitment extremely difficult. eClinical trial platforms offer a host of features including televisits, eConsent, seamless translations, and eCOAs that enable global recruitment reach.

Enhanced Engagement and Retention

With customized alerts, emails, SMS, phone and video calls available at the touch of a button, participant engagement is optimized. Real-time status of all compliance, data entry, technical, or safety updates ensure that trial teams can streamline data collection, support compliance, and maximize data integrity.


In the past, many rare disease trials were priced out from using eClinical platforms. However, technical innovations enable full-featured, value-based solutions specifically targeted to rare disease trial needs and budgets.

Adaptable for Smaller Trials

Rare disease clinical trials are inherently smaller in scale. Digital platforms are flexible, accommodating any trial size while maintaining the highest standards of collaboration, data integrity, and trial outcomes.

Alethium: Bridging Gaps in Rare Disease Clinical Trials

The challenges of conducting clinical trials for rare diseases are undeniable. But the solution is here: full-service digital platforms that are patient-centric, value-driven and adaptable.

Alethium is thrilled to be at the forefront of rare disease clinical trials, making participation more accessible, reducing dropout rates, and accelerating research progress. By removing barriers and enhancing engagement we can improve trial outcomes and bring hope to those affected by rare diseases.

Learn how Alethium can quickly and reliably support your trial. Schedule a demo today!