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Orphan Drug Report

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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 important for these incentives to be available because there may only be a few thousand who suffer from a rare condition or disease. For the pharmaceutical industry to develop any drug it must see a certain amount of profit potential.
Most of these disease are genetic, many appear early in life, and 30 percent of children with orphan diseases die before age of five. Although the market is small for orphan drugs, there has been notable growth in recent years. Currently, 350 orphan drugs are approved for sale in the U.S.
A good example is Rituxan from Genentech, the second most profitable drug in the world, given orphan status to treat B-cell Non-Hogkin’s lymphoma. In 2010, it yielded $5.24 billion in sales for its use as an orphan drug and for extended usage for other types of cancer and rheumatoid arthritis. It is quite possible to more than compensate for the smaller number of patients an orphan drug may help, through the increased market share, lower marketing costs, higher pricing, longer exclusivity period, and faster returns. The drug Rituxan is the brand name and Rituximab is the generic equivalent. It is a cancer medication that interferes with the growth and spread of cancer cells in the body. Rituxan is used to treat non-Hodgkin's lymphoma or chronic lymphocytic leukemia. It is also used in combination with another drug called methotrexate to treat symptoms of adult rheumatoid. 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 depends on the condition it is being used for. These conditions include non-Hodgkin's Lymphoma, Rheumatoid Arthritis, Chronic Lymphocytic Leukemia, Wegener's 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, sleeplessness, stomach discomfort, upset, or pain, swollen joints, trouble concentrating, unable to sleep, weight loss. The normal means of by way of table with its strength determined by the condition it is treating. The normal dosage is 400 mg.…...

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