Rare disease/orphan drug trials have unique challenges with respect to clinical trial management, investigator/site selection, and patient recruitment/retention. Ensuring the implementation of Risk Mitigation strategies throughout the lifecycle of the trial is key in assisting with the identification of several critical factors: Understanding and maintaining strong collaborations with patient advocacy groups and key opinion leaders to create deeper study awareness and support. Ensuring that trial protocols are realistic given any limitations of the target patient population. Engaging physicians with pediatric experience to understand the study design and complexity involved. Remaining patient focused to boost trial enrollment and retention by providing quality subject support materials to demonstrate the benefits of participation. Creating a set of study branded tools to help sites identify potential patients as well as educate them about the study and its requirements. Regulatory understanding and consultation surrounding the unique environment for rare disease studies. Site relationships with access to patient registries to drive patient enrollment. Our experience includes: Multiple Sclerosis Rare Cancers (such as Adrenocortical Carcinoma (ACC), Glioblastoma, Esophageal cancer, Chronic Myeloid Leukemia) Rare Eye Diseases (such as Wet AMD, Diabetic Retinopathy, Retinitis Pigmentosa) Porphyria Cushing’s Syndrome Cystic Fibrosis Familial Hypercholesterolemia Fibrodysplasia Gastroparesis Glomerulonephritis Hidradenitis Supurattiva (HS) Renal Vasculitis Tinnitus Vantage BioTrials will remain focused and dedicated to finding solutions for your study that will realize efficiency and productivity, all the while maintaining quality and keeping your study within cost and timeline. For more information or to request the proposal, please email us at info@vantagebiotrials.com.