Global and National Patient Registries

Abstract

MD registries—collections of secondary data related to patients with one of the nine types of muscular dystrophy—can vary in sophistication from simple spreadsheets accessible only by a small group of physicians, to complex databases accessed online across multiple institutions. Registries can help identify MD patients for scientific research, clinical trials, and later, as products/drugs are approved for the treatment of MD, in the post-marketing surveillance of pharmaceuticals. Registries can also provide healthcare providers or patients with reminders of the need to undergo certain tests in order to reach quality goals. At present, many registries cover only one geographic area or one or two types of MD. The Muscular Dystrophy Association (MDA) is attempting to remedy this disparity with two initiatives. First it is seeking participation from patients with certain myopathies in a patient registry and world map. This database is designed to allow researchers to better understand certain diseases and locate participants for clinical trials and other research studies. Second, to address the lack of a fully operational central registry database for patients with MD, the MDA and Quintiles, a biopharmaceutical services company, formed a partnership in October 2013 to develop and implement the US, Neuromuscular Disease Registry.