Rare Disease – A Case Study in Reaching Recruitment Goals

Recruiting participants for clinical trials is a crucial aspect of advancing medical research, but when it comes to rare diseases, the challenge becomes exponentially more difficult. Rare diseases, by definition, affect a small percentage of the population, making it difficult to find eligible participants. As a result, reaching recruitment goals for clinical trials in these areas requires creative solutions, effective strategies, and often, a deep understanding of the disease landscape.

On a day-to-day basis, our Clinical Trials Optimization team here at Inizio, works with clients to overcome these challenges. Our flagship initiative, Site iMPACT, encompasses a wide range of techniques that use intelligence-based optimization to boost site and patient engagement and accelerate trial recruitment.

In this article Anthony Haywood, Vice President, Clinical Trials Optimization, explores a recent case study that illustrates how our innovative recruitment approaches can successfully meet enrollment goals for a rare disease clinical trial.

The Challenge

Consider a clinical trial focused on a rare autoimmune disease that impacts fewer than 1 in 100,000 people worldwide. For pharmaceutical companies or research institutions, finding eligible participants for such a study presents a significant challenge. There are not only limited numbers of patients who meet the criteria, but those who are eligible may be scattered across various regions, making recruitment efforts even more complex.

Additionally, because rare diseases are less understood by the general public, there’s often a lack of awareness and education about both the disease and the clinical trial opportunities available. As a result, patients may be unaware that they qualify or may hesitate to participate due to concerns about the trial’s safety, complexity, or travel requirements.

The Solution

In this case, our team employed a multifaceted approach to overcome these challenges. The first step was to establish a strong, patient-centered outreach program. We collaborated with patient advocacy groups, which are often well-connected within the rare disease community. These groups helped to identify potential participants who had not been reached by traditional recruitment methods.

Simultaneously, we launched a digital awareness campaign, using social media platforms, disease-specific forums, and websites frequented by patients and caregivers. Digital ads targeted specific search terms, ensuring that individuals with the rare disease would be exposed to recruitment messages. This allowed for better geographic reach and engagement with those who may have been unaware of the trial.

One of the critical factors in recruiting for rare disease trials is building trust with the patient community. Patients with rare diseases are often skeptical about participating in clinical trials due to past experiences, limited treatment options, or concerns about the unknown. Our team understood that clear, empathetic communication was essential.

We hosted workshops and online webinars, led by both researchers and patient advocates, allowing prospective participants to ask questions and learn more about the trial’s goals, process, and benefits. These sessions helped alleviate concerns about safety and clarified the potential benefits of participating in a clinical trial, not only for the individual but for the broader rare disease community.

Given the geographical dispersion of eligible participants, our strategy called for virtual consultations and remote monitoring. We coordinated with our client to amend the protocol and update their processes. This alleviated the need for patients to travel long distances for in-person visits, making participation more accessible for those who lived far from the trial site. In addition, through our Referral Networks initiative, local healthcare providers were enlisted to be referring sites. This allowed local health centers to refer potential patients to the recruiting site to boost recruitment and improve patients’ access to clinical trials.

The Outcome

By using a combination of targeted digital marketing, partnerships with patient advocacy groups, trust-building communication, and telemedicine solutions, the trial successfully met its recruitment goals. Not only did the team reach the required number of participants, but they also ensured that the process was as convenient and reassuring as possible for patients.

This case study highlights the importance of a tailored, patient-first approach when recruiting for clinical trials in rare diseases. The lessons learned in this process can be applied to future trials, ensuring that rare disease research continues to progress and that patients have the opportunity to contribute to meaningful advancements in healthcare.

If you would like to hear more about our Site iMPACT initiative, reach out for a chat!