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
- Cystic Fibrosis
- Leading research efforts with 11 studies across multiple phases
- Gaucher Disease (Type I & III)
- Advancing treatments with specialized trial designs
- Behçet’s Disease
- Investigating potential therapies to manage symptoms
- MPS I H (Hurler Syndrome)
- Exploring treatment strategies for this rare metabolic disorder
- Osteonecrosis
- Conducting studies to evaluate novel therapeutic options
- Pompe Disease (including registry studies)
- Addressing complex disease management with innovative research
- Molybdenum Cofactor Deficiency Type A (MoCD Type A)
- Developing breakthrough therapies for ultra-rare conditions
Our Impact in Numbers
- Agile & Adaptive Trial Design
- We tailor study frameworks to meet the evolving demands of research.
- Global Scale, Local Expertise
- Our international footprint enables streamlined multi-country studies with localized precision.
- Patient-Centric Focus
- We prioritize patient engagement, retention, and experience to ensure high-quality data integrity.
- Regulatory Mastery
- Deep expertise in global compliance to navigate complex regulatory landscapes.
