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Paper Example on Guideline and Reasons for FDA Regulation

6 pages
1435 words
Wesleyan University
Type of paper: 
Case study
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The Food and Drug Administration in the US developed a regulatory initiative in 2004 on the development of new pharmaceutical products as a modernization effort to improve quality in processing and clinical outcome of the products. Apart from instilling quality in development process and quality of the final product, the legislation also promotes general public safety on the use of drugs, medical devices, and biological products (Leuenberger & Leuenberger, 2016). It is imperative to note that FDA regulation aims at creating a balance between public safety in consuming medical products and promotion of safe scientific inventions. Modern drug development needs to be scientifically proven on merit to cure and prevent health conditions; this has led to significant improvement on determining long-term effects of using the drugs compared to the development that took place in the 19th century. Before the new pharmaceutical product is introduced on the market, a chain of clinical research is conducted incorporating patient exposures to ascertain the effectiveness of the product. Despite conducting preliminary tests on new drugs, the latest technology and the trial population does not represent the general population. Hence, disastrous side effects are realized after the products are already on the market.

The general public believes that a drug advertised on media or online medical websites are safe and accredited for use. Such deductive reasoning has led to extensive clinical trials and extended patient exposure tests before the product is released on the market. It is a high calling therefore for FDA to ensure mitigation of risks associated with the new medical product to maximally harness its therapeutic value. The FDA report in 2006, provided a guideline on the requirements for biomedical makers. The gives the biomedical makers a priority because they have a central role in genomics, metabolomics technologies and proteomics at the initial molecular development stage (Leuenberger & Leuenberger, 2016). For safety purposes, animal toxicology and vitro assay experiments are conducted before launching of the product on the market. FDA has provisions on assay experiments using drug-metabolizing catalysts in genetic polymorphism. The purpose of this regulations is to improve efficacy in patient exposure trails. Additional, a biomarker qualifies to have mass production of drugs if relevant data is provided on the intent of the innovation and subset of the disease. The consortia are also created to benefit the majority of the biomarkers. The oversight role of FDA in clinical trials aims at promoting standardization in every stage by optimizing quality, competency, and reliability of clinical outcome. In fact, FDA is working in collaboration with NIC and CDISC to improve its oversight role. The partnership has created a standard code on bioinformatics known as Electrocardiogram (ECD). All bio-information is recorded in ECD format.

Initially, compounded drugs were customized for individual use without any legislation on their distribution and safety use. The public was vulnerable to catastrophic effects of these steroids. For instance, in 2012, there were over 25,000 drug compounders in US, 7,500 specializing in sterile compounding, this is according to the report compiled by International Academy of Compounding Pharmacists (Pinkerton & Pickar, 2016). Over 2.5 million US citizens used the Custom-compounded Bioidentical Hormone Therapy (CBHT). The customized drug compounding business was at its peak leading to safety concerns. For instance, there were 64 incidences of fatalities in 2012 after injection of methylprednisolone acetate. Therefore, the FDA controls drug compounding activities under the Federal Food, Drug and Cosmetics Act (FDCA) and the Compounding Quality Act (CQA) of 2013 (Pinkerton & Pickar, 2016). The guideline on the new drug approval requires biomarkers to conduct animal tests to determine the likely effect of an agent on the human body, later, an Investigational New Drug (IND) is filed outlining preclinical results at the same time request for authorization to carry out human scientific trials. The human trials facilitate discovery of acute effects of pharmacokinetic properties of the new drug, Random Controlled Trials (RCTs) are later conducted to make a comparison with the existing standard treatment. Enrolment of the participants in RCT must meet the threshold of the FDA-registered set.

The specific provision on drug classes showcase the guidelines in the analysis of Hormone Therapy (HT) to indicate vasomotor signs and Vulvar Vaginal Atrophy (VVA). The FDA recommends a one year period constituting three phases in Estrogen and Progestogen Therapy (EPT) to minimize injury of endometrial caused by hot flushes. Apart from the need to conduct a clinical trial, filing a New Drug Application (NDA) is necessary by compiling a report on pharmacokinetic data, safety and efficacy strategies, manufacturing processes, potency, quality and purity of the product (Pinkerton & Pickar, 2016). Other additional information on that should be included in the report are the drug labeling, packaging, and prescription language. The FDA regulates the labeling of drugs to promote full disclosure of drug-associated risks. It is also an effort towards Good Manufacturing Practice (GMP). Once FDA approves the product, the sponsor of new drug carries out a post-marketing investigation and report any safety issues that arise.

Role of Money and Grants in Scientific Advances

New scientific inventions require funding to improve the global health. The global economy has grown massively over the past years, but there is still a gap between the health and wealth. Developing countries are still grappling with chronic diseases due to poor lifestyle. Provision of government grants and money facilitates increases access and quality of global health despite the global economic crisis in developing countries (Jahnke, 2015). Grants promote sustainable health in both developed and developing countries. Terminating money allocation and grants to scientific projects derails medical discoveries on a long-term basis. In 2011, American Association for Advancement of Science (AAAS), federal funding for research and development amounted to 140 billion dollars which is a lower value compared to 2010 budget of 160 billion dollars (Jahnke, 2015). Scientific researchers have little hope in increasing government grants. Hence, other avenues for funding are being exploited as a top priority.

The role of Family in Decision Making

Family participation in decision making provides consent for proceeding with critical medical operations such as surgery and treatment of colorectal cancer (Kim et al., 2017). Including families promotes patient-centered approach hence increases patient satisfaction. Studies show cancer care has improved tremendously due to family involvement in the treatment process.

Prevalence and Incidence of Alport Syndrome

Alport syndrome is a rare and inheritable disease globally. The syndrome is characterized as an end-stage renal illness that leads to deafness of the sensorineural membrane. It is inherited as at childhood as a result of autosomal dominance and autosomal recessive type of AS. Additionally, the disease also affects type four collagen, an element present in the glomerular basal membrane. The incidence of the syndrome is approximated at 1:50000 (Chan & Gale, 2015). The severity of the disease is equally the same in both male and female patients. Adulthood victims of syndrome might have the capacity to hear but with the help of hearing devices. The management of AS patients involve kidney transplant. The renal defect is common in X-Linked AS males (XLAS).

Laboratory Test

The analysis of the previous medical record is necessary to ascertain the medical background of the patient. Urinalysis and blood testing can be carried out in the laboratory to determine kidney infection. Kidney biopsy, genetic testing and skin biopsy analysis can be conducted to ascertain changes in glomeruli, determine the absence or presence of type IV collagen, COL4A5, COL4A4 and COL4A3 genes (Chan & Gale, 2015). The purpose of biopsy is to diagnosis and genetic nature of the disease. Skin autopsy reveals the X-Linked type of Alport Syndrome. Genetic testing is proven method that diagnoses female patients, in prenatal testing, the amniotic fluid is tested to determine if the embryo is affected.


The purpose of FDA and guideline on the development of new drugs has been helpful in eliminating counterfeit medical products from the market. However, its oversight role should promote medical innovation but not derail it. The funding of medical research and participation of family members in decision making supports quality of care, patient-centered approach and patient satisfaction.


Chan, M. M., & Gale, D. P. (2015). Isolated microscopic haematuria of glomerular origin: clinical significance and diagnosis in the 21st century. Clinical Medicine, 15(6), 576-580.

Jahnke, A. (2015). Who picks up the tab for science. BU Today.

Kim, K., Heinze, K., Xu, J., Kurtz, M., Park, H., Foradori, M., & Nolan, M. T. (2017). Theories of Health Care Decision Making at the End of Life: A Meta-Ethnography. Western journal of nursing research, 0193945917723010.

Leuenberger, H., & Leuenberger, M. N. (2016). Impact of the digital revolution on the future of pharmaceutical formulation science. European Journal of Pharmaceutical Sciences, 87, 100-111.

Pinkerton, J. V., & Pickar, J. H. (2016). Update on medical and regulatory issues pertaining to compounded and FDA-approved drugs, including hormone therapy. Menopause (New York, NY), 23(2), 215.

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