There’s no question that developing treatments for rare diseases is complex. 300 million people globally are living with a rare disease, and many of them are children1. These conditions often involve limited natural history research, complex biomarkers, variations in symptoms and disease progression, and financial challenges. Yet, one of the greatest barriers to success is not scientific or financial—it’s people.
More than 80% of clinical trials do not complete enrollment on schedule, often forcing companies to extend studies or add new trial sites to avoid becoming part of the dreaded 90% of drug candidates that never reach the market2. This hurdle is even more prominent when we’re talking about rare diseases and patient populations that are extremely small and often geographically dispersed. The good news? There are ways to mitigate enrollment challenges by bridging the gap between developers and the patient communities they serve.
The Challenge: Lack of trust in drug developers
For patients and caregivers, the concept of enrolling in a clinical trial often feels daunting. The fear of being treated as “guinea pigs” combined with distrust of the pharmaceutical industry often deters participation. This leads many families to turn away from clinical trials before even considering if they might qualify.
The Tools: Transparency and partnerships with advocacy organizations
Advocacy organizations serve as critical liaisons between drug developers and patient communities. These groups are often founded and led by patients and caregivers, which gives them a uniquely rich perspective on how pharmaceutical companies can best communicate with families. When developers invest in these partnerships, they ensure families receive clear, accurate information surrounding study designs, trial eligibility, and eventually, clinical data.
Partnerships with these groups can also provide developers with valuable insights into the unmet needs, treatment priorities, and concerns of each patient community. Maintaining open communication facilitates building essential trust between both parties.
The Challenge: Dispersed or disorganized patient populations
Rare disease patient communities have historically met challenges related to both geographic and emotional isolation. Patients and families may need to travel long distances to meet with specialists in their condition, which can hinder trial awareness and enrollment. A rare disease diagnosis often comes with emotional isolation—patients and families might go months or even years before meeting someone else with the same condition. The literal and metaphorical distance between patients, physicians, and researchers can create significant hurdles on the path to trial enrollment completion.
The Tool: Technology
Technology provides numerous solutions for rare disease communities by facilitating better communication and opening up opportunities that might not have existed previously. Trial-focused microsites with features like physician locator maps and “Am I a candidate?” questionnaires make it easier for patients to learn about trial opportunities and make informed decisions on what path to take next. Targeted earned and paid social media campaigns can also help build awareness among difficult-to-reach communities.
Industry-sponsored social media communities create the opportunity to reduce feelings of isolation by connecting patients and caregivers facing similar situations while providing accurate information related to the disease and clinical research process. Drug developers can use these platforms to share videos and other forms of educational content from healthcare providers, patients, caregivers, and advocates, which helps strengthen their reputations as trusted partners in the rare disease community.
The Challenge: Misconceptions surrounding the trial process
There are many myths about clinical research that can discourage patients from enrolling in trials. Some believe that clinical trials are only for patients who have no other treatment options and that they are a last resort. Other myths include misconceptions surrounding the role of placebos, the availability of trial sites outside major cities, expenses related to trial participation and the safety of drug candidates. Any one of these misunderstandings can have significant impacts on trial enrollment.
The Tool: Patient and caregiver perspectives
Personal patient and caregiver stories provide strong antidotes to misinformation. Advocacy groups can help identify and encourage patients and caregivers who are willing to share their experiences about participating in a trial, making the intimidating concept of enrolling more relatable while also reducing unknowns. Their stories can translate complex information into understandable language and reframe trial enrollment as an empowering and proactive step forward for individual patients, as well as the greater rare disease community. When developers highlight these experiences through media channels and campaigns, they increase public awareness of the condition and, in turn, encourage trial inquiries, and ultimately, enrollments.
The obstacles to rare disease trial enrollment will continue to evolve, but it is possible to adapt and address them. The path forward is clear: active, two-way communication to help open doors, foster trust through transparency, and broaden perspectives that help empower patients, their families and rare disease communities. By approaching these challenges with the patient and caregivers’ voices at the center, companies can accelerate trial enrollment and bring urgently needed therapeutics to the people who need them most.
1 The landscape for rare diseases in 2024. (2024). The Lancet Global Health, 12(3), e341. https://doi.org/10.1016/S2214-109X(24)00056-1
2 Sponsored Insight. (2021, March 26). Nearly 90 percent of drug candidates fail to reach the market. Can organ-chips help? STAT. Retrieved from STAT Brand Studio
