Uncommon Cures, a clinical trial program launched in March 2023, is pioneering a new approach to rare disease clinical trials, addressing the critical need for more efficient and patient-centric research models. Recognizing the limitations of traditional contract research organizations (CROs), Uncommon Cures blends regulatory expertise with a "concierge-based" facility, aiming to streamline trial processes and improve outcomes.
Addressing the Challenges of Rare Disease Trials
According to CEO Marshall Summar, M.D., Uncommon Cures has seen significant demand, exceeding initial expectations. The company has eight signed contracts, is negotiating five more, and is actively engaged with over 50 potential clients. Summar, a veteran in the rare disease field, notes that the traditional CRO model often leads to dissatisfaction due to its cumbersome nature and disconnect from patient realities. Three-fourths of orphan clinical trials fail, often because they are out of touch with the realities of patients and too cumbersome to conduct and participate in.
Uncommon Cures' model is designed to be a scaled-up, regulatory-compliant version of investigator-led trials, with in-house experts guiding study sponsors from Investigational New Drug (IND) application to Phase 4 post-marketing studies. The company's primary facility in Washington, D.C., features a sophisticated medication dispensary and controlled-environment rooms to ensure patient safety. Expansion plans include a second site in Utah and international programs in Chile, Switzerland, and the United Arab Emirates.
Key Innovations in Trial Design and Execution
Global reach is crucial for identifying and engaging rare disease patients, offering them opportunities to participate in investigational therapies. Uncommon Cures focuses on several key areas to improve trial efficiency and success:
- Adaptive Trial Designs: Uncommon Cures emphasizes adaptive clinical trials and crossover studies, which require fewer patients compared to traditional double-blind, placebo-controlled trials.
- Patient-Centric Approach: The company minimizes the burden on patients by reducing the frequency of in-person visits and utilizing decentralized trial components, such as home-based lab draws and patient monitoring.
- Inclusive Enrollment Criteria: Uncommon Cures avoids overly restrictive exclusion criteria that can hinder patient recruitment, recognizing the complexity of rare disease populations.
Impact and Future Directions
Uncommon Cures collaborates closely with patient advocacy organizations, patient registries, and clinicians to facilitate patient screening and build key opinion leader groups. The company's team includes physicians, nurses, clinical research associates, laboratory personnel, and financial experts, all cross-trained to handle diverse responsibilities.
Looking ahead, Uncommon Cures anticipates actively managing 15 clinical trial projects within the next year, serving a range of clients from large pharmaceutical companies to patient-driven organizations. The company's data suggests substantial cost savings, exceeding 40%, compared to current market trends for similar studies. More importantly, study sponsors value the time savings achieved through Uncommon Cures' streamlined approach.
Summar emphasizes that the rare disease field as a whole can improve clinical trial practices. The company's success demonstrates the potential for innovative models to accelerate therapy development and improve outcomes for patients with rare diseases.
