Rare & Genetic Diseases
In rare and genetic disease research, every patient, every data point, and every decision carries extraordinary weight. At UNIQ, we understand that these studies are often defined by small populations, complex biology, and an urgent unmet medical need.
Rare & Genetic Diseases
In rare and genetic disease research, every patient, every data point, and every decision carries extraordinary weight. At UNIQ, we understand that these studies are often defined by small populations, complex biology, and an urgent unmet medical need.
Compassion Through Science
Advancing complex science with dignity, clarity, and care.
Each rare-disease study is a balance of science and empathy. unIQ Trials collaborates with advocacy networks, registries, and patient communities to ensure studies are both feasible and human-centered.
Our experience spans rare oncology, metabolic syndromes, neuromuscular disorders, and congenital enzyme deficiencies — always with a commitment to precision and dignity.
Rare disease studies require more than scientific expertise — they demand empathy, creativity, and operational agility. At unIQ Trials, we support sponsors through the unique challenges of small populations, complex biology, and globally dispersed patients.
We design our rare disease programs to minimize burden, maximize retention, and capture high-quality data, even when every single datapoint matters.
Adaptive Trial Models
We use small-population adaptive designs that allow every data point to count. Our decentralized platform enables at-home participation, reducing travel burden and dropout.
Orphan designations
Retention
Inspection readiness
Enabling Smoother, Smarter Rare-Disease Execution
- Reduced variability through centralized laboratory partnerships
- Seamless coordination of genetic and biomarker samples
- Long-term retention supported by remote follow-up strategies
- Reliable data continuity across extended study timelines
- Strong compliance with global genetic-data governance standards
Precision Logistics & Long-Term Data Continuity
Where precision meets compassion.
Rare and genetic disorder trials often require specialized assessments, long-term follow-up, and strict handling of biological samples. unIQ Trials provides an operational framework designed to maintain integrity, consistency, and participant continuity across every stage of the study lifecycle.
We coordinate complex logistics from central lab alignment and biomarker processing to genetic sample shipping and long-term safety monitoring – ensuring that every step is traceable, compliant, and streamlined across countries and vendors.
Our teams support extended follow-up phases with remote visit options, simplified scheduling, digital reminders, and direct-to-patient coordination, helping sponsors maintain clean longitudinal datasets even when studies extend over several years.
