Small-Sized Trials, Big Impact

In rare disease research, addressing unmet patient needs is driving breakthrough scientific discoveries and treatments. Each study demands a compassionate and expert team to navigate the nuances and tackle the unknowns. With experience that covers the full spectrum of rare diseases, our expertise includes Muscular Dystrophy, genetic syndromes, hematology, metabolic disorders and more.

We rise to the challenge with you

Global reach and impact: IQVIA's rare disease solutions span thousands of clinical sites worldwide, ensuring broad access and efficient trials. Your work brings hope to over 350 million people affected by more than 7,000 rare diseases. Clinical delivery expertise: Our team includes highly experienced Clinical Research Associates (CRAs) and Patient Leads (PLs) with specialized expertise in rare diseases across all major regions. Passion and commitment: Our dedicated team is committed to improving the lives of rare disease patients, ensuring compassionate, patient-centric care. Global Site Network: IQVIA’s global network accelerates study start-up times, enhances patient recruitment, and leverages key relationships with KOLs and geneticists.

Key Study Enablers

Home study visits IQVIA’s home visit solution utilizes directly employed research nurses and phlebotomists, where regulations allow, to deliver study visits in the home setting. Comprehensive support for patient recruitment and retention IQVIA leverages advanced health technologies and decentralized clinical trials (DCTs) to alleviate patient and caregiver burdens, ensuring better retention rates. Concierge support and enabling decentralization Our concierge support services facilitate local setup and provide personalized assistance, enhancing the patient and caregiver experience throughout the trial process. Clinical Trial Educators (CTEs) Dedicated CTEs are deployed to relieve site burden and enhance patient engagement, significantly boosting recruitment efforts. Engagement with patient advocacy groups Collaboration with patient advocacy groups to leverage the best strategies for engaging and retaining patients in clinical trials.

Access the latest in human science and data science to accelerate results

Real World Evidence & Registries: Leverage real world evidence to support natural history studies and registries, addressing challenges of low patient numbers and difficult endpoints. Feeder registries identify patients and connect them to trial sites. Regulatory & Genomic Expertise: Regulatory support from pre-clinical to clinical stage, the IQVIA Laboratories and genetic testing enhance patient identification, crucial for ultra-rare diseases. Innovative Study Designs: Implement novel, adaptive, basket, and seamless study designs to improve trial efficiency and outcomes. Accelerate Results with Integrated Capabilities and IQVIA's Connected Intelligence: Utilize IQVIA's data, actionable insights, advanced technology, and therapeutic expertise to identify the right sites, collaborate with rare disease experts, and make informed commercial decisions. Access extensive global site networks and patient advocacy relationships to enhance trial success.