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Rare Disease & Orphan CRO

Clinical Research in Rare Disease is a Journey and We Go the Distance - Helping You Create a Healthier World

We understand that no matter your size, resources are limited, drug development is costly and time to market is precious. We share your goals of bringing new treatments to market and recognize that milestones in the performance of your trials are critical, potentially enabling you to secure your next round of funding and continue your development program.


What is a Center of Excellence?

Pharm-Olam created three Centers of Excellence (Rare Disease CRO, Oncology-Hematology CRO, and Infectious Disease and Vaccines CRO). Our "centers" are purpose-built to increase efficiency and delivery to timeline and budget. Each Center of Excellence brings together an ecosystem of cross-functional, therapeutically aligned teams that specialize in optimizing investigators, vendors and partners around the world to deliver your trial effectively and efficiently. Pharm-Olam has specifically invested in these three centers to drive true tangible value for our Sponsors and their studies.

This scalable delivery ecosystem/model (one of our pillars in the pattern of excellence) creates synergies and efficiencies that optimize how Pharm-Olam, investigators and vendors collaborate to provide enhanced solutions for expediting and completing your studies on time and on budget.

Working in rare disease requires a CRO partner that is nimble, proactive, able to scale and reach out geographically to where the patients are, and simply must be patient and site focused. Every patient matters in rare and orphan disease research.


Rare Expertise You Can Trust, Rare Expertise That Delivers

Number of Studies
Number of patients
Number of sites
Number of countries

Rare Disease CRO & Orphan CRO Expertise

When You Need Rare Knowledge & Strategy, Our Team Delivers

While it’s true that clinical trials for rare and orphan diseases present a unique set of obstacles, they aren’t impossible to overcome when you have the right expertise and experience. Because of our focus as a Rare Disease CRO, we have invested in the creation of therapeutically aligned project teams who are filled with research professionals with the right experience and learnings that come from conducting numerous rare and orphan disease clinical trials. They put that expertise to work for you.

Rare and orphan disease studies are all unique in what they require of the sponsor, CROs and supporting vendors involved. Being dedicated to the patients and sites means we (as a team) are willing to go the extra mile to support their participation in clinical trials. These rare diseases are often debilitating or life-threatening. Supporting the sites, patients and caregivers is instrumental to creating a successful study ecosystem where every patient matters.


Deep Roots in Rare Disease

Number of Approvals
% project managers with rare and orphan disease experience
% CRAs with rare and orphan disease experience

Orphan Sponsor Testimonial

"When sites identified by other CROs struggled to identify qualified patients, we brought Pharm-Olam into the study to supplement enrollment and limit the damage of slipping timelines.  Now that the studies are complete, we are very impressed with the metrics that Pharm-Olam achieved in terms of study start-up, enrollment and database lock.  I would strongly recommend Pharm-Olam for future projects and look forward to working with the team again!"

Biotech - VP, Project Management

Pharm-Olam Pharm-Olam

Patient and Site-Centric Solutions

Supportive Solutions That Deliver Successful Trials

While all studies benefit from patient and site-centric solutions, the success of your orphan or rare disease clinical trial hinges upon them. Every patient matters in rare disease clinical research. Working around the daily challenges of patients and caregivers while balancing the operational requirements of sites is especially critical for these types of trials.

We partner with sites, KOLs, advocacy groups (national or regional) and patient organizations to ensure we find the patients, make them aware of the research, and support them in participation. These important resources and relationships help our teams understand the patient perspectives and challenges unique to each condition. We then prepare appropriate and study specific strategies unique to your patient population, and work with vendors of all types (travel, reimbursement, home nursing, etc.) to help reduce the burden as much as possible for the patient and their caregiver.

In rare and orphan studies, we often work with research naive investigators. Our strategies to support their engagement in your clinical trial must also be thoughtful of their unique needs and how our ability to support benefits them and the patients they see. Creating an engaged and supportive study ecosystem is critical to your studies success and our shared goal to create a healthier world.

Pharm-Olam Pharm-Olam

Read about our Rare Disease Insights.


The Use of Gene Therapies in Rare Disease Research

At Pharm-Olam, March is Rare Disease Month. Over the past three weeks, we have been exploring the...

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The Use of Oligonucleotide Therapies in Rare Disease Research

Rare disease research is such a complicated area because it involves every therapeutic area...

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Pharm-Olam Pharm-Olam

Natural History Studies

Setting Up For Success Through Conducting Natural History Studies

As a Rare Disease CRO and Orphan CRO, we fully understand the benefits that natural history studies offer sponsors seeking to begin their clinical stage development activities. We have run numerous natural history studies with several being in ultra rare indications. We have seen the benefits first-hand of how this has supported our previous and current rare disease sponsors and their development programs.

Natural history studies are an avenue for Sponsors to understand a disease and how it is managed by clinicians.  They also help Sponsors understand the unmet needs that exist within the patient population.  Our team of orphan specialists leverage our global reach to access patient populations wherever they maybe and assist with setting a minimum amount of required data, which is the inclusion of data directly reported by patients combined with data reported by health professionals. Furthermore, we are experienced in the collection of data on both living and deceased patients. These efforts combined help to ensure more focus on your specific condition area rather than therapeutic interventions.  

By using our team’s expertise and implementing strategies that reach even the most hard-to-find patients, we conduct studies that yield information on critical biomarkers and other correlates of clinical outcomes. We can help you to understand the etiology, range of manifestations and progression of rare diseases.  

Pharm-Olam Pharm-Olam
Pharm-Olam Pharm-Olam

Culture of Accountability

Expecting and Delivering Excellence in Orphan and Rare Disease Clinical Trials

As a part of our goal to help you create a healthier world, we work to provide solutions that improve every aspect of rare and orphan disease clinical trials for patients, sites and sponsors. From start to final study report, we make your study our personal responsibility. This is our Culture of Accountability which is part of the Pharm-Olam Pattern of Excellence. It ensures that when you work with our teams, we provide:

  • Ownership:  We are accountable for the entirety of the study. This includes our work, affiliate vendors, sites, patients and teams.
  • Partnership:  We are one team sharing your goals and your mission.
  • Transparency and collaboration:  We leverage communication, technology and teamwork for your benefit.

Learn more about how we deliver successfully for your projects by visiting our Project Management services page.

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Pharm-Olam Pharm-Olam

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:

Autoimmune hemolytic anemia (CAD, PNH, WAD)
Cerebral palsy
Duchenne MD
Essential thrombocythemia
Graft versus host disease
Hereditary angioedema
IgA nephropathy
Lysosomal storage disorders
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?

Talk with our team today.  

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