Contributing Experts at Medrio: Ian Davison, RTSM Subject Matter Expert; Melissa Newara, VP of Subject Matter Expertise; Rod McGlashing, Data Science Subject Matter Expert at Medrio; Tina Caruana, eClinical Solutions (Digital & Decentralized Trials) Subject Matter Expert at Medrio
One forecast indicates that more than 262.9 to 446.2 million people live with a rare disease worldwide. To develop safe and effective treatments, sponsors and CROs must navigate unique concerns while reducing participant burden, ultimately accelerating ethical, groundbreaking research.
In this article, we answer the questions:
- What makes rare disease research different?
- How can research teams reduce participant burden?
- What are strategies to improve participant engagement?
What Makes Rare Disease Research Different?
The greatest challenge within rare disease research is often the scarcity of available illness information. With limited care options available for many rare diseases, the clinical trials designed to study and treat them are often unique.
Common challenges within rare disease research include:
- Scarcity of available illness information
- Few existing published clinical trials
- Smaller pool of potential participants for recruitment
- Global recruitment may be required for all phases
- Incomplete understanding of natural history
- Variable presentation and progression of disease
- Additional ethical concerns
- Requirement for more sensitive outcome measures to quantify disease
- Limited access to resources and funding
- Lack of established endpoints
Clinical trials for rare diseases tend to take longer because of extended recruitment periods. Study teams may also keep participants in the trial for as long as possible to collect more data points.
Rare diseases, by nature, involve a small patient population. As a result, it can be challenging to recruit participants for these trials. There may also be far fewer participants compared to trials conducted in other therapeutic areas.
Higher recruitment costs
Because of the small potential participant pool, recruitment is critical and the cost-per-participant is extra high. Recruitment can be costly and time-consuming, as it requires extensive efforts to identify, reach out to, and enroll individuals who may be eligible and interested in taking part in a study.
Rare disease trials are more likely to involve multiple sites, potentially spreading across continents, even in early phases. The broad reach often translates into the need for multilingual support for participants.
Less restrictive exclusion criteria
Rare diseases have low prevalence, so finding a sufficient number of participants can be challenging. In early phase rare disease clinical trials, there may be less restrictive exclusion criteria. This adjustment allows for maximum recruitment amongst a limited patient population.
Easing Participant Burden in Rare Disease Clinical Trials
Rare disease clinical trials do not exist in a vacuum. Loved ones and patients with rare diseases are often already managing a heavy load.
Living with a rare disease can dominate life—juggling symptoms, medical visits, and care coordination can lead to chaos and overwhelm. Therefore, researchers need to design their trials with the intention of not increasing the burden.
Ways to reduce burden include:
- Reducing travel or providing meaningful assistance
- Embracing decentralized trial elements
- Responding to patient preferences
Recognize travel burden
The travel associated with clinical trial participation is a big barrier to entry. Travel can be expensive and have negative effects on people’s finances, health, and emotions.
For instance, travel may require participants or loved ones to take time off work or school, resulting in a loss of income. Additionally, it may involve being away from family, sleeping away from home, and impacts on overall quality of life.
Researchers can make travel easier in two ways:
- Reducing the necessity of travel whenever possible
- Offering fair compensation, transportation, concierge services, or other meaningful types of travel assistance
Embrace decentralized elements
Moving to a decentralized clinical trial (DCT) or hybrid model can reduce participant burden by minimizing travel and site visits. For example, remote consent allows patients to join the trial and connect with the research team without any initial travel whatsoever.
Other decentralized trial elements, such as the use of mobile home health nurses, telehealth visits, or home delivery of investigational products, can also lessen participant burden.
Furthermore, technologies that enable remote trial data capture drastically reduce the need to commute to research sites.
Commonly used technologies include:
- Electronic consent (eConsent)
- Electronic clinical outcome assessments (eCOA) including clinician-, patient-, and observer-reported outcomes (ClinRO, ePRO, and ObsRO)
- Online participant portals
- Mobile devices
- Wearable devices
Respond to patient preferences
Researchers should be careful not to prescribe patient-facing technologies to participants without first getting their feedback. Researchers should also be careful not to isolate patients unintentionally in their sincere efforts to alleviate other burdens.
Some participants prefer in-person visits for social support, while others prefer the ability to be remote.
When possible, it is important to provide participants with options so that they can align their participation with their preferences. The goal is to build as much flexibility as possible into any trial.
The most effective approaches to retain participants are to:
- Acknowledge participants’ agency.
- See participants as experts on their personal health experience.
- Give participants a voice in their treatment and care.
- Gather valuable feedback from participants when designing and implementing rare disease trials.
How to Support Patient-Centric Rare Disease Trials
When conducting rare disease research, it’s crucial to adopt a holistic approach that considers the network of individuals surrounding the participant. This includes offering essential social and emotional support throughout the trial to all those involved.
To further facilitate engagement, it is essential to compensate participants and their loved ones appropriately. Make it easier for participants and their loved ones by paying hotel costs, arranging transportation, or supplying meals and restaurant vouchers.
Moreover, recognizing different preferences and offering choices can improve participant engagement and overall experience. The incorporation of decentralized elements, such as eConsent, telehealth visits, or home delivery, may enhance the participation process.
These methods can help clinical trials for rare diseases become more patient-focused and inclusive, ultimately benefiting all involved.
The information in this document is comprised of Medrio’s views and do not constitute for legal or other professional guidance. Please refer to proper industry documentation and consult with your own advisor or known notified bodies for legal or professional advice.