Orphan Drug Act Research Paper

The pharmaceutical industry is one of the most powerful and greedy industries in our country, with a goal to make as large a profit as possible, at the expense of the sick.

Although the FDA prolongs this process for safety concerns, I personally feel that they should be able to expedite their approval. The reason for expedition is because more and more people are being diagnosed with multiple health issues. Some people are more desperate then others and feel they can benefit from the drug as it is. Therefore, if a person wants to take a drug that has not been approved yet, then they should be able to

Pharmaceutical companies are provided with temporary monopoly rights on the production of new drugs which result in a higher cost on consumers. If competing companies were allowed to produce generic forms of those drugs, consumers will be able to afford those medications even in cases where those consumers have no insurance coverage. The company responsible for developing and inventing the original medication could be offered incentives to invent in the future by either obtaining tax breaks or NIH funding for future research. They could even be offered a percentage of the sales of the generic drugs. Economist Gary S. Becker advocates dropping many FDA requirements that, in his opinion, provide no additional safety measures but rather delay the development of new drugs.[12] Betamethasone, for example, has been part of the standard prenatal care in Europe since the late 1970’s while it got adopted in the U.S. after 1997. On many occasions, the FDA ignores all scientific evidence concerning certain drugs because the manufacturer did not follow their mandated bureaucratic standards.

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]

The National Institutes of Health found that “manufacturing problems and business decisions had caused twenty-three percent of drug shortages.” The FDA has currently listed sixty-two drugs undergoing a shortage. Forty-three of those are hospital injections for pediatrics.

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

The high prices set by pharmaceutical companies for drugs allows the companies to continue researching, developing, and producing new drugs. As new diseases are discovered, new medications must be discovered in order to treat them.

2. Denosumab has been FDA approved for 2 indications rather than 1 which is true for most of its competition. Both the conditions are prevalent in the population and their incidence is growing. This will allow the drug to tap unmet needs for the available market.

Methaqualone, is also referred to as Disco Biscuits, Down And Dirties, Jekyll-and-Hyde, Joe Fridays, Lemmon 714, Lemons, Lennon's, Lovers, Ludes, Mandies, Mandrake, Q, Qua, Quack, Quad, Quaaludes, Soaper, Supper, Vitamin Q, The Love Drug, Wallbangers, Whore Pills, and Sopor. This list of street names for the drug goes on and on.

In most cases, there is a committee that decides this. The committee typically includes both physicians and pharmacists. They meet periodically to update the drug list in order to keep it current and relevant.

With the huge diversity and changeability of human biology, it is impossible to imagine a reality without some mutations, changes, or issues in the organs and tissues of humans. Thus, it rightly follows that medications and pharmaceuticals have been created in an effort to counteract the various ailments and illnesses that people can experience. However, as time has gone on and these pharmaceuticals have become more and more high-tech, regulated, and trusted, they have also become incredibly commercialized. Worse still, medications have become incredibly expensive and can be unattainable for some people.

12. The FDA would not let unsafe replications of drugs onto the market, prescription or over the counter medications

Firstly that society often blindly trusts big companies based on the belief that they only release drugs that are safe for consumers and are often swayed to believe these companies due to heavy amounts of advertising. Society trusts that all the harmful risks of medicines will be made apparent that were found through extensive research. Secondly, the horrific side affects that Thalidomide caused in pregnant women generations of people in up to 46 countries began to doubt pharmaceutical companies and manufacturers. This tragedy showed the importance of knowing all the risks of new drugs and medical treatments being proposed. Safety regulations for new drugs were improved all over the world to ensure adequate testing of new drugs and preventing the release of unsafe drugs. Finally though bad science allowed Thalidomide to be released onto the marked and deform more than 10,000 living babies, it created a positive change in the way we test our drugs and has prevented another instance like this from happening

In the United States, a drug can only be advertised legally after being approved by the Food and Drug Administration (FDA). Once attaining at least one FDA-approved use, physicians can prescribe a drug for other unapproved uses, based on their clinical judgment; this is referred to as “off-label use” (McCambridge, 2008). In general, marketing drugs for off-label uses is illegal; however, pharmaceutical companies have gone to various lengths within their legal rights to accomplish exactly that.

This enabled numerous “me too” drugs to achieve satisfactory returns on investment. Imitating a known drug reduced R&D risk considerably, while the marketplace was open to products offering minor advantages such as a more convenient dosage with fewer side effects, but with much the same therapeutic outcome. Generics legislation had a major impact on the industry, providing incentives for innovation and a race to market. The time during which R&D costs could be recouped was drastically curtailed, putting upward pressure on prices. By the end of the 1970s generic entrants and more stringent controls on clinical trials had led to substantial increases in R&D spending.