Journal of Bacteriology & Parasitology

The Past, Present And Future Impact Of Patient Advocacy On The Orphan Drug Act-Through the Eyes Of A Patient Advocate

9^th International Conference on Rare Diseases & Orphan Drugs

April 13, 2022 | Webinar

Deborah Vick

DLV development Seattle University School of Law, United States

Scientific Tracks Abstracts: J Bacteriol Parasito

Abstract:

A rare disease (RD) affects a small number of people. There is no universal definition of an RD, and each country's definition is based on its population, healthcare system, and resources. The most widely used epidemiological metric is disease prevalence: 28 European Union (EU) countries use the figure of five per 10,000, whereas the United States of America (US) and Japan define an RD as one that affects less than 200,000 and 50,000 patients, respectively, at any given point in time. Some definitions include additional criteria such as disease severity and whether it is genetic in nature, resulting in as many as 296 definitions for an RD. RDs affect a large population and pose a significant challenge to any country's public health system. They can also be devastating to the lives of patients and caregivers. Medical genetics and rare disorders have received little attention in medical school curriculum, and as a result, the majority of cases go undiagnosed. The diagnosis was usually made at a tertiary hospital in cases that were correctly identified. Furthermore, the exorbitant prices of orphan drugs, combined with the widespread lack of health insurance, renders even available RD treatment inaccessible to the vast majority of patients. The cost of drugs like Spyrine and Venclext for a year's treatment approaches Rs. 0.5 million, which is more than most Indians' annual income. Because most of these treatments are lifelong, treatment costs can quickly add up to tens of millions of rupees per patient.

Biography :

Deborah Vick is a Director at DLVdevelopment. She has completed her graduation in Seattle University School of Law in San Francisco, California, United States.