Pharmaceutical Regulatory Affairs: Mega Pharmaceutical Limited - Case Study Example

Pharmaceutical Regulatory Affairs By + Introduction Company Profile Mega Pharmaceutical Limited is a company that deals majorly in the importation and sale of prescription drugs of high quality all over the world. The company produces drugs after undertaking intense research on the possibility of the drug to cure a unique disease. Upon determination of the ability of the drug to offer solutions to the problem under study, Mega Pharmaceuticals Limited then begins the production of the drug in its various manufacturing plants for distribution and sale to the parts of the world that are highly affected by the problem under study (Mortimer, 2013, p. 338). The company also works with other research institutions with the aim of coming up with accurate research findings ascribed to the problem (Dudding, 2013, p 34). After the production of the drugs have begun, Mega Pharmaceuticals looks for strategic partners who help in the distribution of the drug to the most affected areas, as indicated above. in working with strategic partners, the company works with international organizations such as the United Nations, who purchase the drugs for donation to the adversely affected areas and various governments which buy the drugs in order to either donate them to the citizens or offer them for sale to the affected citizens. The Global Orphan Drug Strategy In the present case, the Board of Mega Pharmaceuticals approached my office, the Office of the Director of Worldwide Regulatory Affairs at the company, to come up with a global orphan drug strategy. The global orphan drug strategy would serve the purpose of seeking the consideration of the senior management of the company to apply for an approval of a new orphan drug (Dudding, 2013, p 34). The disease that my department selected for examination was the Farh’s Syndrome, which is a rare disorder that degenerates individual’s brain cells of a person, with a high probability of ending up in paralysis. Farh’s syndrome was chosen for research because it qualifies as an orphan disease. An orphan disease is one that is rare among the different human populations of the world. Orphan diseases have a medical urgency attached to them since they do not have a pre-existing form of treatment (Aronson, 2006, p 127). In that sense, they need urgent research into medical solutions to the problem in order to mitigate the effects of the disease (Badyal, 2006, p 43). The cost of drug development keeps going higher, mainly because of the increase in the cost of the raw materials and chemicals required for research and production (Kelly, 2013, p 125). Therefore, the production of orphan drugs to cure rare conditions such as Farh’s syndrome has become more complicated further, the strict rules and regulations that govern the development of drugs has made the cost of developing drugs to spiral. This, coupled with the low returns on the investment made on the drug have had the effect of discouraging the researchers from engaging themselves in the development of drugs, especially orphan drugs, since they cater for the needs of a few affected patients. In this regard, the development of drugs has become a less attractive venture (Murphy, 2002, p 143). However, there are incentives that come with the development of drugs. Such incentives include the provision of exclusive incentives by the government and other organizations such as the European Union Commission and the Federal Drug Agency (FDA) in instances of special protocols. The developments made include the formation of the Orphan Drug Act and the National Organization for Rare Diseases, which are particularly instrumental in the recognition of some rare conditions and the classification of some diseases as rare after research was performed on them. Another boost in the development of orphan drugs is the setting up of the national Institutes that are under the Health Office, that particularly deal with rare diseases. In addition to that, many countries have been instrumental in supporting the development of research on rare diseases by passing legislation on orphan drugs through their parliaments (Badyal, 2006, p 21). In as much as there is considerable progress as far as development of orphan drugs is concerned, there is still a lot more that is yet to be done. The Farh’s syndrome The Fahr’s Disease is also known as the Familial Idiopathic Basal Ganglia Calcifications. This disease is very rare, and is characterized by a person having too much calcium deposits in some parts of the brain, causing a degeneration of the brain cells, majorly causes it. The reason for such calcification is not yet determined, and is still the subject of research by scientists all over the world (Aronson, 2006, p 127). When a person has the Farh’s syndrome, they exhibit symptoms such as a deterioration of their cognitive abilities and a loss of the individual’s motor abilities. If left untreated, the patient can suffer paralysis at the advanced stages of the condition. The disease is a hereditary disease, handed down from a family member to another. The mode of transmission is widely believed to be during pregnancy, where the mother may carry the disease and hand it over to the foetus. The Fahr B Medication In order to provide a medical solution to the problem of Farh’s syndrome, Mega Pharmaceuticals came up with the Fahr B, which is a drug that is designed to mitigate the effects the disease has on the body of a human being. In this regard, the medication is essential in restoring thee lost cognitive abilities that the patient may have lost during the initial stages of the disease. In situations where the patient has already been severely affected by the disease, the Fahr B drug will only help in slowing down the rate at which the disease affects the motor abilities of the individual. Regulation on the approval of orphan drugs (Fahr B Orphan Drug) Regulation on the application for approval of orphan diseases is done by the Medicines and Healthcare Products and Regulatory Authority (MHRA). In the United Kingdom, this kind of regulation is also done in collaboration with the European Medicines Agency (EMA). Licensing, a function performed by these bodies, is undertaken with the aim of making sure that the processes that are followed in the development of the drug can be monitored closely, aside from seeking to make sure that swift action can be taken in the event that the syndrome becomes harmful for use. Developments have been made in recent history in the regulations put in place to guide in the approval of orphan drugs (Kelly, 2013, p 33). In the European Union, the regulation of the European Union Commission Number 141/2000 that focuses on orphan medication spells out the criteria to be used in determining the eligibility of an orphan drug within the countries making up the European Union. Aside from that, the MTRA has the determined the effect of the drug on sample patients The first criterion is the diagnosis of a condition. This is where the condition has to be established to be genuine and existing among a few people within the population (Mortimer, 2013, p. 337). The existence of Farh’s syndrome was discovered many years ago, and the matter of the population it affects has been established. Further, research into the syndrome has discovered the mode of transmission. For the condition to be classified as an orphan disease, it has to affect less than five people in every 10000 in a population (Field, 2010, p 22). The condition has to be able to threaten the life of the individual that has it. The disease should also have a way to prevent its spread to other people. Research into the syndrome has also posited methods of preventing the spread of the condition, such as the examination of mothers during the prenatal period to protect the foetus from infection, since the transmission has been determined to be through inheritance. Further, for the disease to qualify as an orphan disease, despite it affecting more than five people in every 10000 people it has to have an indication that the return on investment does not outweigh the initial investment on the drug. This is to mean that the development off the drug should not be aligned to profit as an incentive for development (Murphy, 2002, p 143). The main aim for the development of the orphan drug is to help in the alleviation of the effects of Farh’s syndrome on the patients. The drug has been developed with the aim of reducing the rate at which an individual suffering the disease loses their cognitive and motor abilities, depending on the extent to which the disease has affected them before they begin taking Fahr B orphan Drug. A final indicator that ought to be held by the organ drug is that it has to be better than the drugs that currently exist in the market (Kelly, 2013, p 56). The benefits that the patients that suffer the disease will derive from using the orphan drug should be better than the benefits that they have been receiving from the use of the drugs that were existing in the market (Badyal, 2006, p 18). The Fahr B drug holds much promise as the drug has been structured to alleviate the effects of Farh’s syndrome. Prior to the development of the drug, there was no other drug that could effectively slow down the rate at which the brain cells were degenerating. The development of Fahr B drug will come with benefits to the patients as they will be able to reduce the subsequent loss of cognitive and motor abilities that comes with the calcium in their brains killing their brain cells. In terms of the procedure to be followed in the approval of a drug, one important factor that is commonly regarded is the designation that the drug is likely to bear (Dudding, 2013, p 34). Developers of orphan drugs can apply for the designation of their drugs at any point during the development of the orphan drug. The application for the designation of the Farh B orphan drug was done at the beginning of the development process. This application was done in order to maintain the focus on the development of the drug, taking focus away from the legal aspects of the development of Farh B drug. After the application was made, the coordinators that would help in the designation of the drug were selected from the COMP and the EMA (Field, 2010, p 37). These institutions are then expected wrote a report on the Fahr B drug, which had an effect on the decision made to grant the drug a designation. The applicant can withdraw an application of such kind at any time. Upon withdrawal, a re-evaluation can only be instigated after five years have elapsed (Bali, 2014, p 4). It is a rule in EU legislation and the provisions of the EMA and MHRA that in applying for approval of an orphan drug, the applicant has to provide the active ingredients that make up the orphan drugs he seeks approval for (Haffner, 2005, p 48). Mega Pharmaceuticals, after the completion of the research and experiments of Fahr B, shall provide the regulator with information on the ingredients used in the creation of the drug along with the conditions under which Fahr B was created. Evidence to prove the plausibility of the drug to mitigate the effects of Farh’s syndrome shall also be submitted. The benefits that the orphan drug has over the existing drugs will be exhibited as per the requirements (Murphy, 2002, p 143).. Mega Pharmaceuticals shall also submit an application for permission to market the drug with its true qualities and according to the provisions of European Union legislation on marketing authorization. Since the market for orphan drugs is not a vibrant market similar to the market for the other drugs, Mega Pharmaceuticals shall apply for a reduction or a waiver on the fees chargeable for protocol assistance (Campos- Castella, et al., 2000, p 143). This shall go a long way in reducing the costs of developing Farh B, which is very costly. Further, it shall reduce the chances of Mega Pharmaceuticals undergoing losses from the development of the product, since the people targeted by the drug are very few, and the company does not aim at charging exorbitant prices for the product for profit maximization. It is a mandatory requirement under European Union legislation that the developer of the orphan drug has to apply for community authorization (Haffner, 2005, p 51). Community authorization allows the applicant to introduce his product to the community for use. This is in accordance with the regulatory framework as posited in the Medicines Regulations Act of 1994, which deals with advertising. The amendments to the Medicines Regulation Act changed its name to the Advertising regulation. These agencies work with the government agencies in ensuring that the provisions of marketing are within the law as stipulated in Title Eight of the European Directive on marketing, which is the Directive 2001/83/EC. Mega Pharmaceuticals will apply for community authorization, since it guarantees the protection of Farh B from being interfered by in the market by the introduction of another drug. The MAA gives protection to the patent of the orphan drug for a period of ten years. Further protection is offered to any data pertaining to the orphan drug from being accessed and used by another researcher (Campos- Castella, et al., 2000, p 145). 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