Rare diseases represent some of the most challenging frontiers in healthcare. With small patient populations, complex diagnostic journeys, and fast-moving therapeutic innovation, they require specialized insight and tailored research approaches. At APLUSA, we bring decades of expertise in rare disease market research, delivering clarity through patient-level data, syndicated tracking, and innovative methodologies.

Our goal is to help pharma and biotech teams understand real-world practice, anticipate barriers, and build strategies that truly reflect the unique dynamics of rare disease markets.

Some of the areas we’ve explored

• Waldenström Macroglobulinemia (WM)
• Marginal Zone Lymphoma (MZL)
• Myelodysplastic Syndrome (MDS)
• Myelofibrosis (MF)
• Beta-Thalassemia
• Paroxysmal Nocturnal Hemoglobinuria (PNH)
• Hereditary Angioedema (HAE)
• CIDP (Chronic Inflammatory Demyelinating Polyneuropathy)
• MMN (Multifocal Motor Neuropathy)
• Primary Immunodeficiency (PID, via subcutaneous immunoglobulin use)