...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...
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...There are many different treatments for Idiopathic thrombocytopenic purpura that depends on the severity of the patient, the age of the patient, and the type of Idiopathic thrombocytopenic purpura the patient has acute or chronic. Children with this disease don’t need much treatment, in most cases children with ITP will improve without any treatment. Close monitoring of the child and keeping them from any serious bleeding, until the body had a chance to correct the disorder by its self is what is recommended for children. The Children’s Hospital of Philadelphia says that “many children with Idiopathic thrombocytopenic purpura are able to spontaneously recover in about 2 to 4 days” (Children’s Hospital). If the child does need treatment the one doctor’s go to first is to put the child on a steroid which...
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...1. How to care for the child with a chronic condition or special needs. Give child choices, some sort of control/independence. Treat them like a normal kid. Mainstreaming: In school in a regular classroom. Do not separate these kids and make them feel different. Don’t focus on things they cannot do but rather focus on what they could do. . Use a more positive approach. Don’t focus on their chronological age because two six year olds will be different. Overall integration into society without stigmas. 2. Phases of reactions that parents go through related to a child with a chronic illness or disability 5 stages 1) Shock 2) Denial 3) Anger 4) Guilt 5) Acceptance. These 5 steps can happen over and over again. i.e., it’s prom night or communion and their child is not like all the other children and the parent may go through these steps again. 3. What is chronic sorrow? A parent dealing with a child with a chronic condition goes through the adjustment process and grief experience it does not happen in one experience. It may happen over and over again when different situations come up. Recurrent adjustment. 4. How to foster an environment of normalcy. Letting them go to a regular school, mainstreaming them, giving them control, treating them overall like a kid, not focusing on chronological age, do not compare to other kids in their age group. 5. Iron deficiency anemia Why does a child get it? We do not see it in the beginning with babies because they have iron stores...
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...Thrombotic Thrombocytopenic Purpura, also acknowledged as TTP, is a rare and serious blood disorder represented by hemolytic anemia and thrombocytopenia coinciding with renal failure and neurological dysfunction (Onundarson, Heal, Rowe, Apr. 1992). The exact reasoning for hemolytic anemia is vague but it may be a consequence of the motorized disintegration of erythrocytes as they pour through partially occluded micro vessels (Onundarson, Heal, Rowe, 1992). TTP is known to cause red blood cells to break apart faster than the body can replace them, conducting hemolytic anemia which is a rare form of anemia. In TTP, small blood vessels begin to clot throughout the body. “These clots can obstruct the flow of oxygen-rich blood to the body’s...
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