Orphan Drug Report - 627 Words

Orphan Drug Report An orphan drug is a drug designed to treat a rare disease or condition. For a drug to qualify for orphan status both the drug and disease or condition must meet certain criteria specified in the Orphan Drug Act (ODA). This designation would qualify the sponsor of the drug for developmental incentives which includes waiving of regulator fees, faster or simpler clinical trial and approval process, tax credit or grants to offset research and development, and a period of marketing exclusivity which means no competition for many years. it does not mean that the FDA is less strict when it comes to safety and effectiveness. All of this is specified within the FDA’s implementing regulations at 21 CFR Part 316. It’s†¦show more content†¦There are a great many side effects the most common include fear, increased cough, lack or loss of strength, night sweats and throat irritations The primary means of dosage is through intravenous injection and the strength de pends on the condition it is being used for. These conditions include non-Hodgkins Lymphoma, Rheumatoid Arthritis, Chronic Lymphocytic Leukemia, Wegeners Granulomatosus, Microscopic, Polyangiitis. The life threatening side effects of using Rituxan include abnormal heart rhythm and liver damage. Another example is Gleevec from Novartis Oncology this drug went on to have non-orphan indications; it had sales of $2.4 billion in 2010. The brand name of the drug is Gleevec and the generic name is Imatinib. This drug interferes with the growth of some cancer cells. Gleevec is used to treat certain types of leukemia (blood cancer), bone marrow disorders, and skin cancer, or certain tumors of the stomach and digestive system. The most common side effects include Acid or sour stomach, belching, difficulty having a bowel movement (stool), difficulty with moving, discouragement,excess air or gas in the stomach or intestines, fear or nervousness, feeling sad or empty, feeling unusually cold, full or bloated feeling, increased bowel movements, irritability, lack or loss of strength, loose stools, loss of interest or pleasure, muscle stiffness, night sweats, passing gas,Show MoreRelatedWhy Usa Based Abc Company864 Words   |  4 Pagesgathered in one region to be used for the evi dence of product registrations in another region following the effect of ethnic factors (FDA, 1998).The regulatory attentions for planning a multinational pivotal phase III clinical trial for drug used for rare disease (Orphan drug by the FDA) which is touching less than 200,000 US population comprise the subjects enrolled in the clinical trials, clinical trial investigators, regulatory agencies, institutional review boards (IRB), and ethics committees, recommendingRead MoreThe Issue Of Orphan Drugs1352 Words   |  6 PagesOrphan drugs are drugs that treat rare diseases that are life-threatening, seriously debilitating, or cause serious and chronic conditions that affects only a relatively small number of patients. Rare or orphan diseases in Canada are those which affect less than 5 in 10, 000 people. About 6,000 to 8,000 rare diseases have been identified worldwide. Most (~80%) are genetic disorders and the remainder are from either viral or bacterial infections or are caused by environmental factors. orphanRead MoreDifferent Applications For Small And Large Molecules1623 Words   |  7 Pagesand BLA. Why are there different applications for small and large molecules? A small molecule drug approval required a New Drug Application (NDA) because it covered under Federal Food Drug and cosmetic Act Section 505 while large molecule drug required a Biologics License Application (BLA) because it covered under Public Health Service Act Section 351. 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