The Issue Of Orphan Drugs - 1352 Words

Orphan 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. orphan drugs A patient with a rare disease target for orphan drugs Over half begin early in childhood and are degenerative and life-threatening. These diseases are called ‘orphans’ because the number of people affected is so small that research on treatment would not be profitable for the pharmaceutical industry. The cost of developing an orphan drug is the same as for other pharmaceuticals. This is why incentives were developed to encourage vital and necessary orphan drugs research. Canada and Orphan Drugs Policy Canada adopted an Orphan Drug policy on October 3, 2012. This made it the last developed country to adopt an orphan drug policy. In July 2011 the orphan drug Soliris, now available through restricted access, received funding from through the Pan-Canadian Pharmaceutical Alliance, which negotiates drug prices for all provinces and territories in Canada. Health Canada will launch a regulatory framework for orphan drugs soon. The proposed framework has 6 key features:Show MoreRelatedTaking a Look at BioMarin1603 Words   |  6 Pagesenzymes for rare diseases. Their first candidate drug was Aldurazyme ® which is currently being used to treat patients of Mucopolysaccharidosis (MPS-I). MPS-I patient census consists of about 3,400 patients in developed countries; 1,000 of which reside in the United States of America. Because of the rare nature of this condition and the rareness of other conditions being treated by BioMarin, they are considered an orphan drug company. Receiving orphan drug designation has resulted in BioMarin gainingRead MoreDrug Development Essay1537 Words   |  7 PagesDrug development This entire section on drug development highlights concerns which are very particular to the industry. They therefore lead to the implementation of CSR activities which are also very industry specific, although aspects such as animal testing and ethics in research can be shared by other industries such as the cosmetics industry, or the medical device sector for instance. Need for RD and innovation Today, the need for RD and innovation is great, maybe greater than ever. In theRead 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. Compared with conventional small-molecule drugs, products derived from a biological source are structurally complex, large molecules and involvedRead MoreNature Of The Drug Store1008 Words   |  5 Pagesâ€Å"Nature to the drug store† is an interesting phrase to describe the series of steps a drug undergoes before it is brought to the consumer. When one thinks of the nature aspect of a drug, they might think of natural sources from plants and/or animals. Although this is true, †¦ Professor Akala recalls as a student having to go to the bush to collect plants to extract them in lab. He also recalls that insulin used for diabetes was taken from pigs in his time. Now, he states, â€Å"insulin is being extractedRead MoreFoster Children Attachment Styles1395 Words   |  6 Pagesto become quit tormented individuals. These children as adults may act out and grow have issues with drug abuse, alcohol abuse, and even self-injurious behaviors. In an article Attachment and Adaptation of Orphans the author Pei-Yung Lane studies foster children and gives an example of an orphans outcome who bounces around from home to home. Pei-Yung Laning calls this child orphan B and states that his issues grew the longer he was in the hands of others besides his parents, ‘ His bad everything includeRead MoreResearch Paper. Have You Or Anyone Around You Ever Been1135 Words   |  5 Pagesof, she is also known to have been supposedly treating children with other treatments. She was known to abuse prescription drugs such as amphetamines, anti-convulsants, and Metrazol. She would use these drugs in hopes of treating children, hoping to gain more success that her electroshock experiments. Metrazol is by far one of the worst drugs that she used, as this drug in particular is known to cause seizures and other harmful side-effects. The odd thing about this case is that although many mayRead MoreBackground And Aims Of A Drug Application Process Essay2029 Words   |  9 Pagesa direct result, often highly infectious diseases with little to no marketable potential are not developed or easily available for those with financial instability, particularly those in the developing world. When you take into consideration that a drug application process takes nearly 10 years to develop, millions of dollars to fund and then is often denied acceptance, it is not unforeseen that companies are hesitant to spend valuable time and money on a product with no intentional revenue. NonethelessRead MoreThe Ethics Of Animal Research Essay1588 Words   |  7 PagesBrody’s, â€Å"Defending animal research: An international perspective† and Peter Singer’s, â€Å"Animal Liberation at 30†. I will then come to my own conclusion based on the different perspectives from the above philosophers and by using my own knowledge on the issue. In this first half of the essay I will discuss the different viewpoints from the philosophers Regan, DeGrazia and Singer on their perspectives on why biomedical research is morally unjustified. In Tom Regan s â€Å"The Case for Animal Rights† he arguesRead MoreEssay about Madonna Kolbenschlags Lost in the Land of Oz1749 Words   |  7 Pagesare no longer useful in todays society. The author believes we need to embrace the ego archetype of the orphan, the most influential metaphor for the self, in order to become a whole and complete person. Madonna Kolbenschlag discusses how our society is particularly hostile towards women, resulting in an acute feeling of self-loathing, doubt, loneliness, and guilt. Today, women as the orphan feel a complete sense of powerlessness and abandonment, not only by everyone around her but also by God.Read MoreA Company When Medication And Health Essay1956 Words   |  8 Pagesdelivers a product with the intention to produce profit. As a direct result, often highly infectious diseases with little to no marketable potential will not be developed or available for those who need it most. When you take into consideration that a drug application process takes nearly 10 years to develop, millions of dollars to fund and then is often denied acceptance, it is not surprising that companies are hesitant to spend valuable time and money on a product with absolutely no disposition for