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Rare Disease CRO Services & Orphan CRO Services

Every Study Unique, Every Patient Matters

We Go the Distance - Helping You Create a Healthier World

Rare disease can impact a single system of the body or several, making monitoring progression a challenge. Every study is unique and so is every single patient. Our rare disease CRO therapeutic team has the knowledge and expertise to connect the dots.

We go the distance - to help you create a healthier world.

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Rare Disease By The Numbers

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Case Study: aTTP Rare Disease Phase III

Acquired Thrombotic Thrombocytopenic Purpura (aTTP) is a rare disease that, when left untreated leads to a 95% morbidity rate. We executed the Phase III study which lead to the marketed drug approval.

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Case Study: aTTP Rare Disease Phase III

Acquired Thrombotic Thrombocytopenic Purpura (aTTP) is a rare disease that, when left untreated leads to a 95% morbidity rate. We executed the Phase III study which lead to the marketed drug approval.

Read The Case Study

Rare Disease and Orphan Expertise

Vast Rare CRO 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 as well as experience working in regulations from the US, EU, and beyond. We have provided CRO services for 14 drug approvals, and roughly 88% of our PMs and 92% of CRAs have Rare Disease experience.

Rare Knowledge Base

As new drugs are developed and treatments discovered, we can understand the underlying mechanisms of rare diseases better. To this end, our study execution repository for Rare Disease is continually evolving. We capture lessons learned from our clinical studies to leverage insights and develop tools to improve rare disease study execution and decision making.  Every study is unique, every lesson valuable.

Supportive Vendor Relationships

Rare disease clinical trials often require numerous supportive vendors.  In rare diseases it is important to bring the research to the patients where they are.  Pharm-Olam has built several relationships over the years with vendors that can support this effort, vendors like, home nursing, patient concierge services, etc.  Together we go the distance for our patients and sites

Natural History Studies

Natural history studies help sponsors understand their targeted rare disease. Pharm-Olam has extensive experience running 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.

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Therapeutic Expertise You Can Depend On

When you choose a team with the right kind of therapeutic experience, you set your study up for success. For more than 20 years, we have specialized in rare and orphan disease clinical trials supporting global research across a wide variety of indications:

Acromegaly
Autoimmune hemolytic anemia (CAD, PNH, WAD)
Blood clotting disorders (ITP, aHUS, TTP…)
Cerebral palsy
Duchenne MD
Essential thrombocythemia
Graft versus host disease
Hereditary angioedema
IgA nephropathy
Lupus (SLE, Nephritis…)
Lysosomal storage disorders
Mesothelioma
Multiple myeloma
Multiple sclerosis
Pulmonary hypertension (IPAH, FPAH, CTEPH)
Sialorrhea from neurological disorders
Sickle cell anemia
Thrombotic thrombocytopenic purpura

Need a partner for your next rare disease clinical trial?

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