Rare Disease Clinical Trial Experience

Recruiting from small populations, deriving valid conclusions from limited data, helping sponsors attain orphan drug status – it’s nothing rare for us.

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Helping move rare disease research forward

Deep connections and personal relationships

Researching rare disease requires comfort with the unknown, something we’ve developed over years of studying some of the rarest conditions. Our work spans all aspects of clinical trials and drug development, encompassing more than 240 rare disease studies across multiple indications in the past five years.

Why choose Premier?
  • Deep connections with advocacy groups help us locate hard-to-find patients and engage them as people, not subjects
  • Rare disease expertise ensures that trials stay on course, even when we’re navigating uncharted territory
  • Continuous investment in our capabilities to analyze data, rapidly evolve protocols, and drive clinical trials toward achieving treatment breakthroughs


Rare Disease

Premier’s deep expertise in rare diseases and rare oncology accelerates clinical development to transform data into life-changing therapies for patients who need them most. Get our brochure.


Coordinating a pediatric gene therapy trial

With their potential for long-term or even curative efficacy, gene therapies are of great interest to researchers, clinicians, patients and caregivers alike. But coordinating and conducting a global, multi-center gene therapy trial is a complex, high-risk undertaking.

Featured Resources



Premier Research and CENTOGENE Launch Strategic Partnership to Accelerate and De-Risk Rare Disease Clinical Development

Premier Insight

Premier Insight

#274: Site Selection, Manufacturing, & Long-Term Follow-Up in a Transition Phase 1/2 Gene Therapy Trial

White Paper

White Paper

Mapping the New Landscape of Orphan Drug Development

Rare Disease Experts

Angi Robinson

Angi Robinson

Senior Vice President, Franchise and Project Management
Adam Bloomfield, M.D.

Adam Bloomfield, M.D.

Executive Director, Medical Affairs, Rare Diseases and Pediatrics

Connect with us

Ready to get started? So are we. Drop us a line to learn more about how we can help.