UBC has developed, implemented, and/or evaluated more risk management programs than any other provider, including for more than 15 rare diseases.
UBC has provided clinical development and late stage services for more than 35 rare-disease projects, touching more than 8,900 subjects and 1,500 sites.
UBC’s Expanded Access Programs have provided more than 3,900 rare-disease patients access to various investigtional orphan products.
Over the past 10 years, UBC's Reimbursement Hubs have coordinated services for more than 44,000 rare-disease patients and handled more than 866,800 high-touch calls.
UBC as Your Rare Disease & Orphan Drug 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.
Alpha 1 antitrypsin disease • Growth hormone deficiency • Multiple myeloma • Myelodysplastic Syndrome (MDS) • MPS II/Hunter Syndrome • Achondroplasia • Niemann-Pick Disease • Dystonia • Pompe Disease • Huntington’s Disease • Precocious Puberty • Lipodystrophy • Chronic Lymphocytic Leukemia • Non-small Cell Lung Cancer • Renal Cell Cancer • Thyroid Disorders • Fabry Disease • Castelman’s Disease • Systemic Lupus Erythematosus • Duchenne Muscular Dystrophy • Post-transplant Lymphoproliferative Disorder • Hurler-Scheie Disease • Narcolepsy • Hodgkin's Lymphoma • Restless Legs syndrome • Morquio Syndrome • Nephropathic Cystinosis • Pulmonary Arterial Hypertension • Infantile Spasms • Polycythemia Vera • Factor XII Deficiency • Idiopathic Pulmonary Fibrosis • Hypothyroidism • Short Bowel Syndrome • Acute Lymphocytic Leukemia • Mucopolysaccharidosis VI • Idiopathic Thrombotic Thrombocytopenic Purpura • Crohn's Disease • Carnitine Deficiency • Pyoderma Gangrenosum • Cushing’s Syndrome • Urea Cycle Disorders • Pediatric Crohn's Disease • Heterozygous or Homozygous Familial Hypercholesterolemia • Enzyme Replacement Therapy • Phenylketonuria • Cystic Fibrosis • Thalassemia • Acromegaly • Juvenile Idiopathic Arthritis • Hemophilia • Hurler Disease • Acute Myelogenous Leukemia • Gastrointestinal Stromal Tumor • Cryopyrin (CIAS1)-associated Periodic Syndromes • Chronic Myelogenous Leukemia • Hereditary Angioedema • Gaucher's Disease • Scheie Disease • Pediatric Multiple Sclerosis
Developing Breakthrough Therapies for Rare Diseases
UBC offers global Clinical Development & 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 challenging for all studies, but even more so for small patient populations. UBC’s site feasibility team utilizes an extensive, proprietary investigator database for site identification, to administer feasibility assessments, and for 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, such as ExpressConnectSM. This tool offers our pharma partners many unique advantages, such as deep patient targeting, real-time data, and higher response rates. 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.
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. For uninsured and underinsured rare-disease patients, UBC can design a Patient Assistance Program using our industry-leading experience and innovative technology solutions.
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 options1. 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 is part of the nation’s largest supply chain. Working with our sister companies, we have the unique ability to follow your orphan product through every step of its lifecycle to maximize access.
Accredo® Specialty Pharmacy
Accredo® Specialty Pharmacy has cared for more patients living with rare diseases than any other pharmacy in the United States. UBC can count on Accredo for additional orphan and ultra-orphan support and expertise.
CuraScript® SD builds specialized distribution strategies that enhance in-office care for rare-disease patients while ensuring our manufacturer partners stay abreast of the current landscape and trends.
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
Safety, Epidemiology, Registries & Risk Management
Dr. Stemhagen has more than 25 years of public health epidemiological research experience, including 15 years in safety surveillance of pharmaceutical, biotech, and vaccine products. Knowing that sponsors have limited information on patients with rare diseases, Dr. Stemhagen develops programs with these populations in mind. She and her team work hand-in-hand with our sponsors by providing them with the appropriate methods to minimize risk without impeding patient access to life-changing medications.
Patient & Physician Services
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.
Patient Access & Engagement
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.