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Rare Disease & Orphan Solutions

Every Study Unique, Every Patient Matters

We Go the Distance - Helping You Create a Healthier World

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 trials 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.

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Therapeutic Expertise You Can Depend On

When you choose a team with the right therapeutic area experience, you set your study up for success. For more than 20 years, we have specialized in rare and orphan disease research supporting over 120 clinical trials including 16 product approvals, across a variety of novel therapy classes and precision medicine including gene therapies, GMO’s, stem cell therapies, and mRNA and antisense therapies.

Rare-Disease-experience Wheel-x2@2x

 

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 Disease and Orphan Drug Development Expertise

Specialized Staff, Deep Expertise

For a rare disease study to succeed, you need well-qualified people at the helm. At Pharm-Olam, we maintain specialized staff with deep expertise in rare disease research as well as experience working in regulations from the US, EU, and beyond. We have provided CRO services for 16 drug approvals, and over 90% of our PMs and 90% of CRAs have rare disease trial experience.

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 range from 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, every lesson valuable.

Patient Recruitment & Retention

Pharm-Olam utilises multiple pathways to maximise patient identification and recruitment including KOL referrals, patient advocacy groups, site/clinical databases, patient registries, and investigator experience and patient relationships. Once identified it is important to bring the research to the patients and our patient centric approach looks to remove logistical barriers through home nursing visits and patient concierge services, support caregivers, and leverages new supportive technologies through established vendor relationships. Together we go the distance for our patients and sites.

Natural History Studies

Natural history studies help sponsors understand their targeted rare disease and patient population. Pharm-Olam 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, right patients, and collecting clean data for analysis.

Rare Disease By The Numbers

Pharm-Olam is a midsized CRO committed to ease of collaboration and delivery. Agile, innovative, and flexible full-service solutions make us the chosen partner for small to mid-size biopharma rare disease clinical research with over 70% of our experience with small & emerging biotech and 36% of our study experience in the last 5 years in rare diseases and orphan drug designations.

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Need a partner for your next rare disease clinical trial?

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