Orphan Drug Act

Another issue involving the Orphan Drug Act that has caused great conflict and controversy is the market exclusivity it provides as an incentive. In 1989 Amgen’s drug erythropoietin was approved by the FDA to treat anemia. Amgen was given exclusive marketing rights for seven years and erythropoietin became one of the most successful drugs in history. In these types of instances, the issue is that these companies have essentially created a monopoly where they have complete control. [6][7] Since there is no competition, companies have the opportunity to drive up the prices forcing people to be left with the ultimatum of either paying high out of pocket prices or leaving their condition untreated. The reduction in competition also severely limits the access to treatments for patients with rare diseases. [6]

P3- explain relevant sections of key legislation associated guidelines with regard to the administration of medicines

In 1906, the Pure Food and Drug Act, that was years in the making was finally passed under President Roosevelt. This law reflected a sea change in medicine-- an unprecedented wave of regulations. No longer could drug companies have a secret formula and hide potentially toxic substances such as heroin under their patent. The law required drug companies to specify the ingredients of medications on the label. It also regulated the purity and dosage of substances. Not by mere coincidence was the law passed only about five years after Bayer, a German based drug company began selling the morphine derivative, heroin. Thought to be a safe, non-habit forming alternative to morphine, heroin quickly became the “cure-all drug” that was used to treat

This act, under the direct administration of the Bureau of Chemistry, forbade the interstate transport of illegal food and drugs, and exposed any responsible parties, that dare to violate the law, to penalty of seizure of the questionable products and/or prosecution. In fact, the foundation of the law relies on the regulation of product labeling rather than pre-market approval. The selling of Drugs, which are defined according to the standards of strength, quality, and purity in the United States Pharmacopoeia and the National Formulary, is denied under any other condition except that the specific variations from the applicable standards were clearly stated on the label. However, there were different standards that defined foods, but the law outlawed the addition of any ingredients that would alternate for the food, hide damage, generate a health danger, or constitute a filthy or decomposed substance. The controversial interpretations of the food provisions in the law engendered many, sometimes prolonged, court battles. In case the manufacturer decided to list the weight or measure of a food, this had to be done accurately. Also, the food or drug label could not be false or misleading in any particular, and the presence and amount of eleven dangerous ingredients, including alcohol, heroin, and cocaine, had to be listed (Nash 276-77).

Americans must wait up to 19 years after a discovered treatment before they can participate in benefits of a new medication (Philipson & Sun, 2008). The regulatory process drug manufacturers need to endure before releasing potentially life-saving medication is an extremely expensive, time-consuming process. The Center for Drug Evaluation and Research (CDER) is the main department of the Food and Drug Administration (FDA) responsible for the safety of drugs (both prescription and over-the-counter) sold in the United States (Food and Drug Administration, 2011). This department scrutinizes the testing of new drugs and

Even though the pharmaceutical industry had been highly profitable and contributed about 40% OF Ciba-Geigy’s revenues in profit, there were some trends, which were worrying. The government had attempted to reduce a cost of healthcare thus; pressure to lower costs was mounting on industrialized countries. There were restrictions to introduce new products, and price control became stricter while limiting the freedom of doctors to prescribe medications. Patent controls were becoming reduced, and the pharmaceutical industry was becoming increasingly criticized. These trends later made the industry to

Under the Ronald Reagan administration the Orphan Drug Act allows the federal government to help out in the making of new drugs for these rare diseases. Incentives are given to the drug manufacturer in order to intise them to find a cure or develop a drug that will help out. To find a cure can become very expensive and time consuming for the drug companies. There are incentives given to the drug manufacturers like grant funding, assistance in the drug development, seven-year patent on the drug post-Food and Drug Administration approval, and so forth. In the end, the drug company has the better end of the deal.

Although the FDA was not known by this title until 1930, the FDA’s regulatory functions first began with the passage of the 1906 Pure Food and Drugs Act, prohibiting interstate commerce in adulterated and incorrect labels on food and drugs (FDA 2). The bill gave the FDA its power to adjust over a wide subject matter (fda 2). This law was the merging of 100 bills over a half of century that was aimed towards abuses in the consumer product marketplace (FDA 2). The meat inspection act was also passed on the same day which set rules for cleanliness and sanitation of meat processing and slaughterhouses

Under this act only treatments for unmet medical needs would be eligible. Some helped to improve outcomes and reduce risk more often than those approved by the FDA. All of these drugs could not already be on the market or they would not be eligible. To do this it is required of sponsors to give up all other IP protections for indications protected under dormant therapy exclusivity (Usdin).

The Food and Drug Administration is a regulation agency within the Department of Health and Human Services. It’s role in our nation is to be responsible for “protecting the public health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nation’s food supply, cosmetics, and products that emit radiation” ("What We Do."). One of the most important responsibilities and the topic I will be discussing throughout this paper is drug regulation. The Food and Drug Administration approves drugs that are intended for use in diagnosis, cure, relief, treatment, or prevention of disease, and is intended to affect the function of the body. In order to do so, The Food and Drug Administration reviews drug manufacturer’s via application to put drugs on the market; therefore, a drug may not be sold or marketed unless it has and remains approved by the FDA. Even though a drug has been approved does not mean it will remain on the market, drugs have the likelihood to be recalled. For example, when you see those late night personal injury lawyer commercials saying, “If you have been prescribed and taken said drug, and experienced any of these side effects such as blood clots, seizures, etc., you may be entitled to compensation.” those drugs have usually been recalled due to adverse effects

The U.S. Food and Drug Administration (FDA) is responsible for protecting and promoting public health through regulating pharmaceutical drugs, biologics and medical devices in the context of granting approvals for marketing authorization, surveillance of the clinical trial study of the drug, and post-marketing surveillance of the medical product. Pharmaceutical companies seek FDA approval for a new drug to be marketed which entails a long process. This process starts with submitting an application known as an investigational new drug application (IND) to start clinical trials to enroll a group of patients believed to benefit from the investigational product, and to approve that the drug is safe and effective.

an NDA/BLA to FDA as they are ready, rather than the standard requirement to submit

Kefauver Harris Amendement or "Drug Efficacy Amendment" is a 1962 amendment according to this drug cannot be brought into united states without proving its efficacy and safety. This act says that manufacturers need to prove the effectiveness of drug products before they go on the market, and report any serious side effects to us (FDA). It also requires that evidence of effectiveness of the unapproved drug be based on adequate and well-controlled clinical studies conducted by qualified experts and all this information to be documented in the appropriate form according to ICH guidelines and submitted for approval. The drug is allowed for marketing after the FDA approves the NDA when all the information is reviewed and is considered safe and appropriate.

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.

The decision of this instance requires the starting of a short report of the Indian pharmaceutical industry, to expose the trip about its recent times of progress. India’s pharmaceutical industry was famous for duplicating the majority of the patented drugs from the organizations of the Western world and Japanese nation. The scheme occurred before passing a contract with the world trade organization. The statement mentioned above procedure result from the U.S. only, between the develop markets. Nonetheless, the ratification of the contract with the world trade organizations in 2005, brought the Indian pharmaceutical industry to compliance of defending the knowledgeable belongings rights