Small sized trials with big needs. 

In rare disease research, unmet patient needs are spurring new scientific discoveries. But with small, hard-to-find patient populations, each study requires an experienced team who knows how to navigate the nuances. And tackle the unknowns. So you can bring new treatments to patients and their families.

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Bringing new therapies, new hope to rare disease patients

Rise to the challenge

Your work brings hope to the families of more than 350 million people living with one of 7,000 rare diseases. With so few approved treatments, you can make a real difference.

But you face immense challenges in finding dispersed patients, identifying meaningful endpoints, creating novel study designs, and executing everything flawlessly.

Backed by the exceptional approach and resources of IQVIA, the leading Human Data Science Company™, our unified team of experts is ready to help.

IQVIA integrates the patient perspective in rare disease research
IQVIA integrates the patient perspective in rare disease research

Decentralized Trials tailored to rare diseases

IQVIA's Experience

336 studies

in rare disease in 71 countries worldwide since 2016

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

IQVIA puts unparalleled data, actionable insights, advanced technology, and deep therapeutic expertise to work in solutions that will accelerate results for rare diseases. By tapping into these integrated capabilities, you can

  • Identify the right sites: Identify sites treating patients for your protocol with IQVIA's proprietary algorithms and real world data analysed by IQVIA's rare disease experts
  • Global and expansive reach: Tap into IQVIA's extensive global site and patient advocacy group relationships in rare disease
  • Collaborate with experts: Benefit from rare disease experts who've been there, and delivered; our experts can provide counsel on needs from complex study and protocol designs, to regulatory approval and reimbursement market access
  • Improve commercial decisions: IQVIA's advanced analytics, including machine learning and predictive analytics capabilities, help you generate new insights from your data
  • See the impact of new innovations and treatments: Be ahead of the curve in planning for and executing on regenerative medicines and cell and gene therapies
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Cell & Gene Therapy Center

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Real World Data Sets

Richer real world data insights can drive smarter decisions.

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