Rare Diseases and Orphan Drugs

Research in rare disease & orphan drugs requires incisive thinking, experience, & a gentle touch.

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We share your passion for finding treatments for intractable disease states.

Understanding the exceptional challenges of research for rare disease and orphan drugs is essential in bringing treatments to the patients who need them. Fifty percent of rare diseases affect children, placing great burdens on families, while the intricacies of genetic research require highly specialized knowledge. Patient-centric trial designs are paramount.

Rare diseases can impact a single system of the body or several, making monitoring progression a challenge. Our Integrated Full-Service Rare Disease Solution leverages insights gathered through the conduct of 120+ rare disease trials to facilitate decision making and improve study design and execution.

From natural history studies to pivotal trials, our expertise includes complex clinical trial considerations such as pediatric patient populations, new biomarkers, surrogate endpoints, and the incorporation of patient perspectives and experiences, across a wide range of innovative medicines and advanced therapeutics in rare and ultra-rare diseases.


Therapeutic Experience You Can Depend On

For more than 20 years, Allucent has specialized in rare and orphan disease research. Our team has supported more than 120 clinical trials, including 16 product approvals, across a variety of novel therapy classes and precision medicine including gene therapies, GMOs, stem cell therapies, and mRNA and antisense therapies.

Rare Diseases and Orphan Drugs
  • Acromegaly (All Subsets)
  • Epilepsies (pediatric, adult; progressive, genetic, refractory…)
  • Autoimmune hemolytic anemia (CAD, PNH, WAD)
  • Blood clotting disorders (ITP, aHUS, TTP…)
  • Spasticity and Sialorrhea disorders (CP, MS, Stroke, Trauma…)
  • Movement disorders (Duchenne, CP, Ataxia, Huntington’s …)
  • Lysosomal storage disorders (different subtypes, details available upon request)
  • Gastrointestinal disorders (Short bowel…)
  • Pulmonary disorders (CF, PAH, Bronchiectasis..)
  • Cardiac disorders (ATTR-CM amyloidosis…)
  • Skin disorders (RDEB, DDEB)
  • Rare CNS disorders
  • Metabolic storage diseases
  • Infectious diseases
  • Cardiovascular diseases
  • Immunologic disorders

Rare Disease and Orphan Drug Development Expertise

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Specialized Staff, Deep Expertise

At Allucent, we maintain specialized staff with deep expertise in rare disease research as well as experience working with regulators in the U.S., EU, and beyond. We have provided CRO services for 16 drug approvals, and over 90% of our project managers and 90% of CRAs have rare disease trial experience.

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Rare Knowledge Base

Our Rare Knowledge Base of clinical trials includes insights and solutions for the most complex rare disease clinical trial and orphan drug development. These insights and solutions cover protocol design and clinical development planning, development, and execution of regulatory strategies for new clinical trial designs, innovative therapies and treatments for ultra-rare diseases, mapping the patient landscape to ensure timely and efficient study initiation, and ultimately accelerating your First-Patient-In goal. Every study is unique, and every lesson valuable.

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Patient Recruitment and Retention

Allucent utilizes multiple pathways to maximize patient identification and recruitment including KOL referrals, patient advocacy groups, site/clinical databases, patient registries, and investigator experience and patient relationships. It is important to bring the research to the patient. Our patient-centric approach aims to remove logistical barriers through home nursing visits and patient concierge services, support caregivers, and supportive new technologies through established vendor relationships.

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Natural History Studies

Natural history studies help sponsors understand their targeted rare disease and patient population. Allucent has extensive experience running both retrospective and prospective natural history studies in rare and ultra-rare diseases to support sponsor data collection and program development. We are experienced in finding the right sites and the right patients and collecting quality data for analysis.

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