Science 37 played a pivotal role in nearly doubling the U.S. enrollment for GSK's Phase 3 trial focused on an investigational medicine for cholestatic pruritus in primary biliary cholangitis (PBC), a rare liver disease. The trial, requiring 230 participants globally, benefited from Science 37's direct-to-participant model, which significantly enhanced patient access and retention.
Overcoming Barriers in Rare Disease Trials
Recognizing the challenges of enrolling patients in rare disease studies, GSK embraced an innovative approach to broaden trial access. Science 37's decentralized trial solution allowed participants to engage from their homes, mitigating symptom-related obstacles such as fatigue that often hinder adherence to traditional site visits. This approach was particularly beneficial for patients in rural areas who would otherwise need to travel long distances to clinical sites.
Brandon Maggio, Global Head of Digital Operations & Process Optimization at GSK, noted, "Science 37’s innovative services meant we could bring the trial to the homes or neighborhoods of patients in rural areas who would typically have to travel hundreds of miles to the nearest clinical site, which may otherwise have been a barrier to participating."
Impact on Patient Engagement and Study Continuity
Science 37's capabilities enabled screening across most U.S. states, including areas lacking traditional brick-and-mortar sites. Furthermore, the platform facilitated seamless transitions for participants at risk of dropping out from conventional sites, ensuring study continuity. This strategy resulted in an impressive 82.3% completion rate for Part A of the trial, a critical milestone for evaluating the investigational drug's initial effects compared to placebo.
Decentralized Approach for Rare Disease Research
Dr. Debra Weinstein, VP of Internal Medicine and Principal Investigator at Science 37, emphasized the value of decentralized trials in rare disease research: "The enhanced access of a decentralized approach becomes invaluable when sponsors are dealing with small patient populations that are widely dispersed across countries or even continents."
The success of this collaboration underscores the growing importance of patient-centric, technology-driven solutions in clinical research, particularly for rare diseases where patient populations are limited and geographically dispersed. The ability to bring the trial directly to patients' homes not only improves enrollment rates but also enhances patient engagement and retention, ultimately leading to more robust and reliable clinical trial outcomes.
