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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 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.
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.
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.
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 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.
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.
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.
Dr. Stemhagen provides strategic consultative services to pharmaceutical and biotechnology clients in epidemiology, safety surveillance, and risk management. Dr. Stemhagen has extensive oncology experience, having served as a principal investigator and collaborator on multiple national cancer studies through the National Cancer Institute, FDA, and EPA. As part of the National Cancer Institute’s Surveillance, Epidemiology, and End Results Program (SEER), Dr. Stemhagen helped establish the New Jersey State Cancer Registry, which collects data on all cancer cases diagnosed and/or treated in the state.
Sr. Vice President & Chief Scientific Officer
Shazia Ahmad provides our pharmaceutical Sponsors with extensive rare-disease expertise, insight, and direction on the design and implementation of study recruitment and retention plans. Before joining our organization, Shazia served as a study coordinator at the National Institutes of Health (NIH), where she was involved in the coordination of various rare-disease studies. Throughout her career, she has worked with many rare diseases, including cystic fibrosis, pulmonary arterial hypertension, Kawasaki disease, sickle cell disease, Tourette syndrome, Lyme disease, restless legs syndrome, and pediatric multiple sclerosis.
Senior Director, Patient & Physician Services
Nicole Hebbert leads UBC’s Patient Access and Engagement teams, providing orphan drug manufacturers with best-in-class services in patient access programs, including reimbursement hubs, clinical contact centers, patient assistance programs, field nursing, and administration of risk evaluation & mitigation (REMS) programs. Soon after her son was born in 1995, Nicole was diagnosed with Lupus. Living with Lupus, a rare disease, has given her a unique perspective into patient interaction, as well as knowing the details of a physician’s practice. Nicole understands the challenges of frontline responsibilities for small patient populations. She often tells her team — and other leaders — that behind every phone call, fax, or email is a patient in need of care.
Sr. Vice President, Patient Support Services