A review of orphan drugs and rare diseases
Journal Title: International Journal of Research in Pharmacology & Pharmacotherapeutics (IJRPP) - Year 2016, Vol 5, Issue 2
Abstract
The World Health Organization defines orphan diseases, as all pathological conditions, affecting 0.65-1 out of every 1000 inhabitants. They are usually not studied for their path physiology or for newer therapeutic options, as these are not economically viable. The Orphan Drug Act was passed on January 28, 1983 by USA to stimulate the research, development, and approval of those products that treat orphan diseases. Till date, 11 drugs (4.87%) for tropical infectious diseases has been designated with orphan drug status, and as many drugs for other infectious diseases. Several drugs with orphan status are used in the treatment of diseases that no longer meet orphan status criteria, such as AIDS and end-stage renal disease. Understanding of the human genome, nuclear cloning, rational drug designing, and application of high throughput screening in drug discovery programs, might lead to new drug discoveries for orphan diseases. Hence, there is hope in the future for patients neglected by for-profit drug discovery efforts.
Authors and Affiliations
Venkat Paluri
Keywords
Related Articles
- EP ID EP408374
- DOI -
- Views 102
- Downloads 0
Case report on klatskin tumor
Klatskin Tumor also known as Hilar Cholangiocarcinoma is a cancer that affect Bilary tree predominantly at the bifurcation of the common hepatic duct. Of all diagnosed cancers, Cholangiocarcinoma accounts for approximate...
Introducing an evidence based medicine course to second year medical students - evaluating its effectiveness and student feedback
Objectives: To study the effectiveness of Introducing an Evidence Based Medicine (EBM) course in the curriculum of Second Year Medical Students and to obtain their feedback regarding the same. Methods: 66 students conse...
Observational study on prescription pattern of various antibiotics in a teaching hospital
Introduction Prescription pattern monitoring studies (PPMS) are useful to assess the prescribing, dispensing and distribution of medicines. They promote appropriate use of monitored drugs, reduce the misuse of these val...
Hepatoprotective effects of l-carnitine against cyclosporine A-induced liver injury in white albino rats; a newly proposed mechanism of action
It is well documented that l-carnitine (L-C) has protective effects against various types of injury. Although its antioxidant action has been reported as a major mechanism, other suggested pathways may be implicated in i...
Pharmacist’s interventions in the management of patients with chronic kidney disease
Aim Chronic kidney disease (CKD) is a complex debilitating condition affecting more than 70 million people worldwide. With the increased prevalence in risk factors such as diabetes, hypertension, and cardiovascular dise...