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RARE & ORPHAN DISEASE clinical TRIAL SERVICES

AT PHARM-OLAM, THE RAREST STUDIES ARE NOT RARE. WE APPRECIATE THE UNIQUE CHALLENGES OF MANAGING THESE STUDIES, AND HAVE PROVEN SUCCESS IN PATIENT RECRUITMENT AT SPECIALIST SITES.
Comprehensive Phase I-IV Trial Management Solutions

EXPERTS IN RARE & ORPHAN DISEASE

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Number of Studies:
107

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Number of Patients:
10,423

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Number of Sites:
1,923

Pharm-Olam has specialized in rare disease and orphan indication clinical trials for more than twenty years, and offers a full-suite of CRO services supporting Phase I-IV research on a global scale.  Rare disease and orphan studies require unique solutions which are patient-centric —and often site-centric— requiring our staff to understand the daily lives of our patients and their caregivers, while also considering the operational requirements of our investigators

Finding sites with interested patients suffering from rare or orphan diseases is often difficult for sponsors, but Pharm-Olam works to provide supportive solutions and services, so all parties (patient, caregiver and site) can participate and meet our goal of: Helping to Create a Healthier World

Over 75% of our CRAs and >80% of our PM's have rare disease and/or orphan experience in indications including:

  • Acromegaly
  • Brain Cancer
  • Diabetes, Type 1
  • Gastric Cancer
  • Leukemia, Chronic Lymphocytic
  • Lupus Nephritis
  • Mesothelioma
  • Multiple Myeloma
  • Non-Hodgkin’s Lymphoma
  • Sickle Cell Anemia
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Patient-Centric Support Services for Rare Disease & Orphan Trials

We understand that participation in clinical trials is often demanding, and potentially disruptive, to a patient’s normal daily routines. Therefore, we see it as our responsibility to make it easier for them to adjust to this change and have as positive a participation experience as possible. 

Pharm-Olam knows the right specialist sites, advocacy groups and patient/family organizations to recruit qualified patients. Pharm-Olam does this through one-to-one, established relationships that span decades in some cases, and also through innovative methods including industry events, social media, educational groups and industry consortiums.

Our partnerships with global advocacy groups and travel/reimbursement vendors support patients and their caregivers.  This results in higher awareness of our trials and an increased willingness to participate and remain on-study.

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Individual Attention. Vast Resources. The Expertise You Need.

At some CROs, there exist “layers” of communication which impede collaboration. Not at Pharm-Olam. The sponsor can pick up the phone and have a discussion, where ideas for process improvement are heard and implemented more quickly.

In addition, sponsors can leverage the size of Pharm-Olam to maximize their study's potential, while maintaining the person-to-person communication necessary for rare disease and orphan indications. In addition, project teams leverage our 60+ country coverage, where our native staff and Country Managers are able to navigate regulatory nuances. All of this while having the infrastructure and medical experts available at the organizational level to offer insights.

Our on-the-ground PMs, CRAs and medical professionals can tap into patient populations' needs and can more nimbly respond to a trial’s changing needs.

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Natural History & Registry Studies

In addition to supporting Phase I-IV study designs, Pharm-Olam also has experience in running Natural History (NH) studies.  These studies are an important tool for understanding the etiology, range of manifestations, and progression of rare diseases. Well conducted NH studies can yield information on biomarkers and other correlates of clinical outcomes. Especially for extremely rare conditions where little is understood about the disease, a Natural History study plays a crucial role as it will set the baseline for future clinical trials.

With Orphan research, we know about the importance of reaching out to patients in even the most remote locations, and Pharm-Olam is familiar with collecting data on both living and deceased patients. 

Tapping into our global resources and local networks, we can also support Registry Study designs, a valuable tool commonly seen for orphan indications. Their implementation is essential for building trial readiness, and requires the effort and cooperation of patients and organizations on one side and clinical Investigators on the other.

Our Orphan Specialists can set-up registries with the largest possible geographic coverage, and will assist with setting a minimum amount of required data (the inclusion of data directly reported by patients combined with data reported by health professionals). This helps maintain more focus on a certain disease or group of diseases, rather than simply on therapeutic interventions.

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Specializing in Pediatric Trials

Pediatric trials often face a complex set of criteria and considerations, including informed consent, study endpoint definition, patient recruitment and access to patients over a designated amount of time.

Pharm-Olam’s pediatric specialty services are born of over two decades of progressive, complex research study experience, and our physicians and regulatory experts are able to provide advice specific for the conduct of studies in this unique and vulnerable population.

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Federal Funding for Specialty Studies

The FDA announced it has awarded six new research grants for natural history studies in rare diseases. The aim of the research is to inform medical product development by better understanding how specific rare diseases progress over time. One potential application of these studies is the opportunity to eventually use natural history models to augment the need for placebo arms in studies of drugs that target very rare disease, where trial recruitment can be challenging. This is the first time the FDA is providing funding through its Orphan Products Grants Program to conduct rare disease natural history studies.

Pharm-Olam's US Government Federal Research Services Team can help as this expands and other opportunities arise.

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