Global Impact and Prevalence of Rare Diseases
Rare diseases, also known as orphan diseases, are conditions that affect a small percentage of the population. They are characterized by their rarity, with most countries defining a rare disease as one that affects fewer than 1 in 2,000 people. Despite their individual rarity, when considered collectively, rare diseases are not uncommon. It is estimated that there are over 300 million people worldwide living with one of the approximately 7,000 identified rare diseases.
Types of Rare Diseases
Types of rare diseases include genetic disorders, rare cancers, infectious diseases, autoimmune diseases, and others. These conditions vary widely in their severity, with some life-threatening or chronically debilitating. Due to their low prevalence, research and development in rare diseases face unique challenges, including limited knowledge, scarcity of expertise, and difficulties in patient recruitment for clinical studies.
Ultra-rare diseases are a subset of rare diseases that are even less common, often defined as affecting fewer than 1 in 50,000 people. These diseases represent an extreme end of the rarity spectrum and pose additional challenges in research and healthcare due to the extremely small patient populations.
At Bazis Americas, we possess a profound and unique expertise in conducting research related to rare diseases. Our team has successfully navigated the complexities of rare disease studies, both commercial and non-commercial, enabling us to deliver insightful and actionable results to our clients.
Ad hoc and Longitudinal Research
Our experience spans across various rare diseases, including Duchenne muscular dystrophy, Spinal muscular atrophy, Huntington’s disease, hemophilia, paroxysmal nocturnal hemoglobinuria, Hodgkin’s lymphoma, phenylketonuria, and others. Each project is tailored to uncover deep insights, from understanding patient journeys to evaluating the effectiveness of new treatments.
Patient-Centric Approach
Central to our research methodology is a patient-centric approach. We involve patients and their caregivers in the design of our projects to ensure that our research addresses the most pertinent issues they face. This approach not only enriches our studies but also fosters a more empathetic understanding of the patient experience.
Collaboration with Patient Advocacy Groups
Our established relationships with patient advocacy groups and patient communities are invaluable in our research efforts. These collaborations enhance our ability to recruit participants and gain nuanced perspectives on the lived experiences of those with rare diseases.
Commitment to Advancing Rare Disease Research
Our commitment to rare disease research is driven by a passion to contribute to advancements in medical care that can significantly improve the lives of patients worldwide. Through our rigorous academic training, hands-on experience, and patient-centric approach, we are dedicated to uncovering truly inspiring insights that can lead to real-world impacts.
At Bazis Americas, we are partners in the quest to understand and address the challenges faced by those living with rare diseases.
