Aaron Berger (UBC) and Paul Kelly (THREAD) presented an innovative case study at the DIA 2023 annual conference in Boston last month to a standing-room-only audience. The presentation focused on combining patient-centric, modernized study design with intelligent technology integration to enable effective real-world evidence (RWE) generation.
This case study shows how modern study design elements can be incorporated into a long-term observational study in patients with a rare disease in a real-world setting. The rare disease under investigation affects up to 25,000 individuals in the U.S. The goals of the study are two-fold:
- Evaluate the long-term safety of the marketed therapeutic in patients with the rare disease
- Generate real-world evidence on the long-term effectiveness of the marketed therapeutic in patients with this rare disease
The challenge was to design a trial that is:
- More inclusive
- Accessible and adaptive for patients and their caregivers
On the surface, this challenge may seem overwhelming. This is a rare disease, so patient numbers are low. Mobility issues may make travel to study sites prohibitively burdensome. Some patients live in remote locations and many do not receive routine care at one of the healthcare settings participating in the traditional brick-and-mortar site-based study.
UBC and THREAD worked with the sponsor to incorporate a central virtual site model, a virtual patient coordinating center, and a DCT Technology Platform (Figure 1) in addition to the ongoing traditional brick-and-mortar study design.
This brought flexibility and convenience to patients to enroll, eConsent, and complete activities onsite or fully remotely, streamlining the patient and caregiver experience throughout the seven-year study. Configuration of the central virtual site expanded geographic access and provided options and flexibility for patients. In addition to the ~100 patients enrolled at 25 active brick-and-mortar study sites in the first 18 months, the central site brought an additional ~30 patients in just its first three months.
This hybrid study design is enabling real-world evidence generation studies in post-marketing research settings. This case study demonstrates the future of inclusive and diverse observational studies, especially in rare disease. Similar study designs can be implemented across other therapeutic areas. To learn more about UBC’s unique position in the post-marketing research space, click here. With a focus on generating powerful real-world evidence for regulators, payers, and providers, we bring experience in implementing modernized study designs geared toward your unique research question and patient population. You can get in touch with our team directly here.
About the Presenters
Aaron Berger is the Head of Evidence Development Solutions for UBC. Mr. Berger joined the UBC team in 2003 and has served in a range of diverse roles leveraging global capabilities in the execution of evidence development programs. UBC’s Evidence Development Solutions team’s mission is to design and execute modernized study designs that incorporate the latest advances in data acquisition technology to generate vital evidence on the safety and value of treatments.
Paul Kelly is the Executive Director of Business Development at THREAD. His career includes 25+ years in the clinical trial industry spanning various roles in clinical operations through technology implementation. THREAD’s purpose continues to be advancing research for everyone, everywhere modernizing clinical trials to be patient-centered, inclusive, and efficient.