Emma Bagshaw

Vitaccess

This white paper discusses the benefits and uses of real-world evidence as well as the power of digital technology in RWE generation.

OBJECTIVES To identify common methods for the estimation of healthcare resource use (HRU) and to assess their strengths and weaknesses. METHODS A targeted search of the MEDLINE database, databases for economic evaluations, and HTA websites was conducted to identify articles reporting on HRU data-collection methods. The search was indication- and t...

Introduction Myasthenia gravis (MG) is a rare, chronic, autoimmune disease, mediated by immunoglobulin G antibodies, which causes debilitating muscle weakness. As with most rare diseases, there is little patient-reported data with which to understand and address patient needs. This study explores the impact of MG in the real world from the patient...

Background and Objective Transfusion-dependent β-thalassemia (TDT) is a rare genetic disease characterized by a deficiency of functional β-globin, ultimately leading to lifelong dependence on blood transfusions. There is little patient- and caregiver-reported data with which to understand the holistic and societal impact of TDT. The objective of th...