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Rare Disease

 Your Rare Disease, Orphan Drug & Patient Advocacy Partner

Just because a patient population is small doesn’t mean that those patients require less support. Patients facing rare diseases need integrated, high-touch care to access life-saving medications. UBC supports the entire rare-disease product lifecycle from Sponsors who are developing products to improve the diagnosis and treatment of these conditions to manufacturers who are bringing these breakthrough medications to market. Our teams have supported many rare diseases and orphan indications over the years.

UBC has supported more than 200 rare disease projects, involving more than 15,000 subjects and 1,700 sites across the product development lifecycle – from clinical through commercialization.

Supporting Development of Breakthrough Therapies

UBC offers Global Periapproval Services & Late Stage Clinical Research services tailored to rare diseases, including clinical trial implementation and management, patient recruitment, peri and postapproval studies, registries and value demonstration studies.

Finding the right sites and subjects is a challenge for all studies, but especially for small patient populations. UBC’s site feasibility team utilizes an extensive, proprietary investigator database for site identification to administer feasibility assessments and provide direct outreach to rare disease foundations, patient advocacy groups and treatment centers of excellence.

Foundations & Patient Advocacy Groups

UBC has decades of experience working with a variety of foundations and patient advocacy groups. In rare diseases where access to these patients is critical to better understand the disease process, patients and their caregivers, we work directly with the foundation, or a manufacturer looking to more deeply engage with the foundation, enabling them to participate in a wide range of pre- and post-marketing late stage research. Foundations are particularly important to furthering our understanding of products and populations in unique indications where less is known about the disease process and epidemiology.

UBC helps facilitate connections and important clinical, safety, effectiveness, and commercial discussions between pharmaceutical manufacturers and foundations to create mutual alignment between the foundations’ medical, scientific and advocacy agendas and the manufacturer’s product specific clinical development approach. In return, data sharing agreements are established between the parties or directly with UBC to ensure access to critical patient data in a secure, HIPAA-compliant manner, captured through innovative real world technologies and infrastructure.

The programs we support in rare, orphan and/or specialty products often combine collaboration with the manufacturer and key local or national foundations, underpinned by evidence generation approaches that consider both traditional data collection and other real world data access opportunities. These data points may include healthcare settings or direct-to-patient virtual engagement, providing a comprehensive assessment of the most optimal way to implement a rare disease program.

Finding the right sites and subjects is challenging for all studies, but especially for small patient populations. UBC leverages foundations to directly source data on these patients and better understand their referral patterns throughout the healthcare journey from pre-diagnosis, to diagnosis and beyond. Foundations are also aware of the patient journey, clinical and/or other issues impacting their care.

UBC’s site feasibility team utilizes the foundation’s feedback to help us narrow our patient and site selection approach. We also offer an extensive, proprietary investigator database for site identification to administer feasibility assessments and support direct outreach to rare disease foundations, patient advocacy groups and treatment centers of excellence.

Patient & Physician Services

To help our Sponsors overcome recruitment challenges, our Patient & Physician Services team utilizes data-driven solutions. Our deep scientific understanding, epidemiological approach, and data-driven solutions allow us to deliver our pharmaceutical Sponsors valuable insights and rapid results for patient, physician, and site recruitment.

Ensuring Your Orphan Product is Safe to Use

Due to sparse or incomplete rare-disease clinical trial data, Sponsors encounter many challenges when trying to assess orphan drug risk and safety. Also, these patients often tolerate higher uncertainty because an alternative drug is usually unavailable. Our Risk Management & Pharmacovigilance services help pharmaceutical and biotech manufacturers maximize orphan product performance by managing risk before, during, and after launch.

UBC has developed, implemented, and/or evaluated more risk management programs than any other provider, including for more than 15 rare diseases.
Over the past 10 years, UBC's Reimbursement Hubs have coordinated services for more than 44,000 patients with rare diseases and handled more than 866,800 high-touch calls.

Navigating a Complex Therapeutic Journey

UBC knows that seamless coordination is the key to ensuring a successful therapeutic journey. For rare-disease patients, this journey is as unique as they are. Our Reimbursement services provide unique solutions that ensure patient journeys are navigated successfully.

Building relationships with trusted Healthcare Professionals helps rare-disease patients fully engage in their treatment. Year after year, nurses rank as the most trusted professionals in the country. Our Nursing & Adherence solutions provide patient-centric care, therapy education, and product administration — frequently, where the patient is most comfortable: A UBC-trained clinician is within driving distance of 90% of the U.S. population.

UBC’s Expanded Access Programs have provided more than 3,900 patients with rare diseases access to various investigational orphan products.

Expanded Access Programs

Over the years, pharmaceutical manufacturers and Sponsors have worked hard to find breakthrough therapy products to treat rare diseases. However, currently, only 5% of the roughly 7,000 rare diseases in the world have approved treatment options. While patients wait for a therapy to be approved to treat their disease, many pharmaceutical manufacturers and Sponsors are utilizing Expanded Access Programs (EAPs) for their orphan products.

UBC is the only company that combines all of the necessary services to provide a seamless patient journey. Engaging experts in Clinical Development, Reimbursement Hubs, Nursing, and Specialty Pharmacy at the outset of an EAP allows UBC to offer a true start-to-finish solution and a panoramic view of patient care throughout the process and across the globe.

Experts

UBC thought leaders have a long history of meeting the needs of Sponsors focused on orphan diseases. Our work, performed globally, has ranged from providing strategic planning support, to designing multiple types of research engagements and high-touch programs, to communicating our findings in publications and conference presentations. In certain cases, our research efforts have been the first-ever studies in a specific disease.

Annette Stemhagen, DrPH, FISPE

Sr. Vice President & Chief Scientific Officer

Shazia Ahmad

Director, Patient & Physician Services

Nicole Hebbert

Sr. Vice President, Patient Support Services