The Drug Approval Of The Disease Essay - 2495 Words

There are over 7,000 rare diseases affecting between 25 and 30 million Americans (Global Genes 2016). A disease or disorder is defined as rare in the U.S when it affects fewer than 200,000 Americans at any given time (Eurordis 2016). Some of these rare diseases come with even more rare and controversial decisions. â€Å"The FDA just made its most controversial drug approval of the year† (Fortune 2016). This injectable drug is called eteplirsen by Sarepta Therapeutics and it is an exon-skipping drug targeting a section of genetic code called â€Å"exon 51† in the dystrophin gene. Exon skipping is a type of strategy that is being developed for the rare disease DMD (Duchenne Muscular Dystrophy). The exon-skipping process skips sections of genetic codes during protein manufacturing, allowing cells to create shortened but partially functional dystrophin protein which is the muscle protein that is missing in DMD. This rare X-linked disease was first described in the 1850†™s by French neurologists Guillaume Duchenne but was not known to inhibit the production of protein dystrophin and result in the loss of motor functions and muscular degeneration until the 1980’s (MDA 2016). 1 in 3600-6000 live male births has resulted in DMD (Bushby 2010). Most patients are diagnosed around 5 years of age when their physical abilities are no longer similar to their peers (Bushby 2010). If left untreated, signs of deterioration in muscle strength increase, the chance of using a wheelchair before their teensShow MoreRelatedEvaluation Of A New Drug1167 Words   |  5 Pagesfor a new cancer drug to go from concept to market it must first go through the following FDA approval process to get there. This process can take up to 12 years and cost a drug manufacturer well over 350 million dollars.2 The steps are Preclinical research, the Investigational New Drug (IND) application, Phase I Trials, Phase II Trials, Phase III Trials, the New Drug Application (NDA), Ap proval, and Phase IV Clinical Trials.1 The FDA regulates this process to ensure that drugs are safe and effectiveRead MoreWhy Usa Based Abc Company864 Words   |  4 Pagesthem before conducting clinical trial for the rare disease and follow ICH E5 guidance to provide references for regulatory and development strategies to authorize clinical data gathered in one region to be used for the evidence 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 USRead MoreThe Medicine Of The Pharmaceutical Industry Essay1599 Words   |  7 Pagesdeaths from diseases lower. About 253 people die per minute, 6,322 people die per hour, about 151,729 people die every day and a total of approximately 372,000,000 people per year. Not all of this death are due to different diseases bu t the 60% of the death is because of it and because there is no appropriate medicine to cure it. Every year a lot of medicines are discovered by the pharmaceuticals but it is not enough to reduce those large numbers. Before, the discovery of the drugs was a casualtyRead MoreA Research Study On Biotechnology Essay1502 Words   |  7 Pagesevidence that biotechnology carriages additional risk, but there are uncertainties that concern the public. Biotechnology product put the same risk on subjects as the airplane put on travellers. The cost of new biologic product development through the approval is approximately $2.6 Billion, means $2600 million plus the cost of post-marketing surveillance is $312 million, which is many times higher than one Airbus A380-800 that cost $428 million. The regulatory program may differ for different field ofRead MoreOrphan Drug Market Development And Manufacturing Of Products1622 Words   |  7 Pagesof rare diseases. The Allston Landing Facility (ALF) currently produces a single enzyme replacement therapy which treats patients with Type 1 Gaucher disease. This treatment falls within the classification of an Orphan drug which is a drug or biologic that treats a patient population of less than 200,000 within the United States. There is an entire segment of the pharmaceutical industry comprised of companies that develop and manufacture treatments for rare diseases. The Orphan Drug market hasRead MoreA Research Study On Biotechnology Essay1538 Words   |  7 Pagestechnology users. There is no evidence that biotechnology carriages additional risk, but there are uncertainties that concern the public. We put same risk on subjects as the pilot put on his passengers. The cost of new biologic development to the approval is approximat ely $2.6 Billion, means $2600 million plus the cost of post-marketing surveillance is $312 million, which is many times higher than one Airbus A380-800 that cost $428 million. The regulatory program may differ for different field ofRead MoreFda Approval Process Of Drugs Essay1681 Words   |  7 PagesJoston Toney Nicole Thompson English 2010 Online 03 December 2016 The FDA Approval Process of Drugs When I was a kid, I always wondered why it took so long for an ill person to become well again. I always thought that if the ill person went to the doctor they would be back to normal the next day, but that’s not the case. For some people it took several days, weeks, months, and even years to conquer an illness but as a child I never could understand that. I don’t know how many times I’ve asked myRead MoreVaccines Are Safe And Safe Today1231 Words   |  5 PagesIn the world today, we are exposed to all kinds of germs and disease, these germs and disease can cause all kind of harm on to humans. Throughout history there has been epidemic of deadly viruses all around the world. As a species, we have developed a way to prevent disease and viruses though vaccines or immunizations. The Idea of Vaccine have bee a major challenged to public health over the last century. But like any type of medication, there are going to be both pros and cons. Recently there hasRead Mo reDrug And Drug Testing In The Roles Of The New Drugs831 Words   |  4 Pagesbroad review in order to get the approval process by the Unites States Food and Drug Administration before any drugs can be prescribed to any of it patients. By the end of the year 2016, according to the 3.2 billion drugs were ordered from many physician practice for there patients (CDC, 2016). Many of these good and some are harmful drugs have bombarded numerous of pill bottles. Currently, the drugs supposed to go through a process of development, and next approval before being introduce/marketingRead MoreThreat Of New Entrants : Low848 Words   |  4 Pagesfunding coming from heavy expenditures in RD, along with the risk of little to no returns or even heavy losses if the drug fails to reach the market. Regulatory environment partly contributes to the barrier as the new drug approval process can be time-consuming with relatively 89% of failure to pass through. The second barrier is specialization. Companies with knowledge in obscure diseases will enjoy low threat of new entrant for there are few experts in the field. Power of Suppliers: Low Biotech companies’