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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mHealth & remote clinical trial strategies

mHealth and remote monitoring allow patients the convenience of staying close to home, while still providing clean, concise data. Rare disease trials often combine these and other techniques, enabling much to be accomplished remotely.


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.

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 242 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

Featured Resources



Six Issues to Consider for Rare Disease Pre-IND Meetings

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

Premier Thought Leadership

Angi Robinson

Angi Robinson has been conducting pediatric and rare disease studies at Premier Research for more than 14 years. She has provided oversight and full management support for over 30 rare disease studies, including multiple U.S. and global programs. She has also supported FDA pre-IND meetings, IND submissions, and NDA/BLA project directorship.

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