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Orphan & Rare Diseases

Orphan and Rare diseases present unique challenges, and our mission is to drive research that leads to life-changing therapies. At Confidence Pharmaceutical Research, we are committed to advancing clinical trials for orphan and rare diseases, providing hope to patients with limited treatment options.

Our global studies leverage innovative methodologies to accelerate the development of novel therapies for these complex conditions. In addition to traditional clinical studies, we also manage Compassionate Use Programs (CUP) / Named Patient Programs (NPP), ensuring that patients can access life-saving medications even before regulatory approval—whether globally or in their specific country. These programs, approved by the Ministry of Health and Ethics Committees, offer a critical lifeline when no other treatment options exist.

Our studies span both pediatric and adult populations, including ultra-rare conditions where patient recruitment and trial design require highly customized approaches.

Focus Areas

  • Leading research efforts with 11 studies across multiple phases
  • Advancing treatments with specialized trial designs
  • Investigating potential therapies to manage symptoms
  • Exploring treatment strategies for this rare metabolic disorder
  • Conducting studies to evaluate novel therapeutic options
  • Addressing complex disease management with innovative research
  • Developing breakthrough therapies for ultra-rare conditions

Our Impact in Numbers

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Clinical trials conducted
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Patients enrolled
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Clinical sites
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Countries

Transforming
Digestive Health

We’re not just a CRO—we’re a catalyst for change.
Our expertise spans across the full spectrum of gastroenterology, from inflammatory conditions to 
motility disorders, ensuring that every trial we conduct 
is an opportunity to push science forward.

  • Identifying novel therapies that improve patient outcomes.
  • Using real-world evidence to refine clinical strategies.
  • Conducting global trials that represent real-world patient demographics.
  • Driving efficiency without compromising on quality.

Why Work With Us?

  • We tailor study frameworks to meet the evolving demands of  research.
  • Our international footprint enables streamlined multi-country studies with localized precision.
  • We prioritize patient engagement, retention, and experience to ensure high-quality data integrity.
  • Deep expertise in global compliance to navigate complex regulatory landscapes.

Ready to discuss your Clinical Trial needs?

Schedule a Consultation with Confidence