Successfully navigate development challenges to your rare disease therapy

Therapeutics Proof Point

Your work brings hope to the families of over 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.

Winning your fight against rare disease requires the exceptional approach and resources of IQVIA, The Human Science Data Company. With our real-world data analysis, rare-disease experience, and deep clinical knowledge, you can fulfill your promise of hope for millions around the globe.

  • Speed recruitment by quickly and confidently identifying the right early-stage patients with our proprietary algorithms and real-world data on more than half a billion patients.
  • Help reduce costs through our Rare Disease Magnet Sites and relationships with appropriate medical centers worldwide.
  • Benefit from rare disease experts with unmatched experience and insights. Our Rare Disease Center of Excellence helps you design and oversee complex studies and protocols, provide experienced regulatory counsel, and advance your treatment toward rapid approval and reimbursement.
  • Monitor consumers and better support patients through our agile patient engagement team and their relationships with advocacy groups. Utilize tools such as telemedicine, predictive analytics, home health visits, and working across country borders to optimize recruitment and retention.
  • Collaborate with clinical experts who’ve been there and delivered. Our specialists help you better understand disease pathophysiology, underlying mechanisms, and disease sub-types. And our trial specialists can support new investigators, help navigate referral pathways, and develop site-specific recruitment approaches.

Accelerate your fight against rare disease with unparalleled data; insightful and actionable analytics; advanced technology; and unrivaled institutional knowledge.

Let us help you develop and deliver your rare disease treatment successfully.

Orphan Designations
350 million people worldwide are living with a rare disease. More than the entire population of the United States. But only 5% of those known rare diseases have an approved treatment.